The Firearm Safety Among Children and Teens (FACTS) Consortium issued a research agenda with the goal of preventing pediatric firearm injuries and death.

Kateywhat/ThinkStock

The 26 research agenda items from FACTS, which span across the broad topic areas of epidemiology, surveillance, and risk and protective factors; primary, secondary, and cross-cutting protective factors; policy-related issues; and data-enhancement priorities, were published in JAMA Pediatrics.

“Firearms are the second leading cause of death among children and adolescents aged 1 to 18 years in the United States and responsible for more than 2,570 deaths and nearly 12,000 nonfatal injuries requiring emergency department treatment in 2017,” said Rebecca Cunningham, MD, of the University of Michigan, Ann Arbor, and colleagues who are members of the FACTS Consortium.

“Pediatric firearm injuries result from a range of causes, including the unintentional discharge of a firearm, self-inflicted wounds, or the escalation of interpersonal violence,” they continued. “Nearly 265 million firearms are in civilian hands in the United States, and a 44% increase in pediatric firearm mortality rate has been documented during the past 5 years.”

The FACTS Consortium defined an agenda “to serve as a guide for future research efforts to decrease pediatric death and injury.”

Some of the research agenda items include the following:

• Understanding epidemiologic trends and how demographic factors are associated with fatal and nonfatal outcomes.
• The long-term cost associated with pediatric firearm outcomes.
• The effectiveness of health care–focused primary prevention strategies for children, adolescents, and their families to reduce firearm outcomes.
• Examination of health care–based interventions for children and adolescents who have experienced or witnessed a firearm injury to prevent (or reduce) subsequent firearm outcomes including firearm injury recidivism and mental health, socioemotional, and educational outcomes.

“It is an excellent list, and I think it was very thoughtful to come out with a research agenda for firearm safety for kids,” Marlene Melzer-Lange, MD, chair of the American Academy of Pediatrics subcommittee on violence prevention, said in an interview. “The thing that’s been lacking nationally has been funding for firearm injury prevention, both for children and adults, and to have something focused specifically on children – because children have the biggest chance of living the longest – is really important.”

She described the research agenda as being “comprehensive” and didn’t see anything that stood out as missing from the list, although the big issue that remains is getting funding for the research now that an agenda has been outlined.

And getting that is not going to be easy.

“I think it’s going to take some political will of the people and particularly of our legislators ... to allow the [Centers for Disease Control and Prevention] to provide funding” said Dr. Melzer-Lange, an attending emergency department physician at Children’s Hospital of Wisconsin, Milwaukee, adding that AAP has long been asking for such funding. “It’s going [to come down to] the legislators [having] enough guts to actually put the funding in and not be afraid of external groups that might ask them not to do that.”

She remains optimistic and hopeful that the legislators are going to look at it and will eventually say that this is an crisis and will take more action beyond just talking about it.

Dr. Melzer-Lange also emphasized that this is about firearm safety and not firearm control, especially for children and teenagers.

All authors reported receiving grants from National Institutes of Health/National Institute of Child Health and Human Development.

gtwachtman@mdedge.com

SOURCE: Cunningham RM et al. JAMA Pediatrics. 2019. doi: 10.1001/jamapediatrics.2019.1494.

The Firearm Safety Among Children and Teens (FACTS) Consortium issued a research agenda with the goal of preventing pediatric firearm injuries and death.

Kateywhat/ThinkStock

The 26 research agenda items from FACTS, which span across the broad topic areas of epidemiology, surveillance, and risk and protective factors; primary, secondary, and cross-cutting protective factors; policy-related issues; and data-enhancement priorities, were published in JAMA Pediatrics.

“Firearms are the second leading cause of death among children and adolescents aged 1 to 18 years in the United States and responsible for more than 2,570 deaths and nearly 12,000 nonfatal injuries requiring emergency department treatment in 2017,” said Rebecca Cunningham, MD, of the University of Michigan, Ann Arbor, and colleagues who are members of the FACTS Consortium.

“Pediatric firearm injuries result from a range of causes, including the unintentional discharge of a firearm, self-inflicted wounds, or the escalation of interpersonal violence,” they continued. “Nearly 265 million firearms are in civilian hands in the United States, and a 44% increase in pediatric firearm mortality rate has been documented during the past 5 years.”

The FACTS Consortium defined an agenda “to serve as a guide for future research efforts to decrease pediatric death and injury.”

Some of the research agenda items include the following:

• Understanding epidemiologic trends and how demographic factors are associated with fatal and nonfatal outcomes.
• The long-term cost associated with pediatric firearm outcomes.
• The effectiveness of health care–focused primary prevention strategies for children, adolescents, and their families to reduce firearm outcomes.
• Examination of health care–based interventions for children and adolescents who have experienced or witnessed a firearm injury to prevent (or reduce) subsequent firearm outcomes including firearm injury recidivism and mental health, socioemotional, and educational outcomes.

“It is an excellent list, and I think it was very thoughtful to come out with a research agenda for firearm safety for kids,” Marlene Melzer-Lange, MD, chair of the American Academy of Pediatrics subcommittee on violence prevention, said in an interview. “The thing that’s been lacking nationally has been funding for firearm injury prevention, both for children and adults, and to have something focused specifically on children – because children have the biggest chance of living the longest – is really important.”

She described the research agenda as being “comprehensive” and didn’t see anything that stood out as missing from the list, although the big issue that remains is getting funding for the research now that an agenda has been outlined.

And getting that is not going to be easy.

“I think it’s going to take some political will of the people and particularly of our legislators ... to allow the [Centers for Disease Control and Prevention] to provide funding” said Dr. Melzer-Lange, an attending emergency department physician at Children’s Hospital of Wisconsin, Milwaukee, adding that AAP has long been asking for such funding. “It’s going [to come down to] the legislators [having] enough guts to actually put the funding in and not be afraid of external groups that might ask them not to do that.”

She remains optimistic and hopeful that the legislators are going to look at it and will eventually say that this is an crisis and will take more action beyond just talking about it.

Dr. Melzer-Lange also emphasized that this is about firearm safety and not firearm control, especially for children and teenagers.

All authors reported receiving grants from National Institutes of Health/National Institute of Child Health and Human Development.

gtwachtman@mdedge.com

SOURCE: Cunningham RM et al. JAMA Pediatrics. 2019. doi: 10.1001/jamapediatrics.2019.1494.

The Firearm Safety Among Children and Teens (FACTS) Consortium issued a research agenda with the goal of preventing pediatric firearm injuries and death.

Kateywhat/ThinkStock

The 26 research agenda items from FACTS, which span across the broad topic areas of epidemiology, surveillance, and risk and protective factors; primary, secondary, and cross-cutting protective factors; policy-related issues; and data-enhancement priorities, were published in JAMA Pediatrics.

“Firearms are the second leading cause of death among children and adolescents aged 1 to 18 years in the United States and responsible for more than 2,570 deaths and nearly 12,000 nonfatal injuries requiring emergency department treatment in 2017,” said Rebecca Cunningham, MD, of the University of Michigan, Ann Arbor, and colleagues who are members of the FACTS Consortium.

“Pediatric firearm injuries result from a range of causes, including the unintentional discharge of a firearm, self-inflicted wounds, or the escalation of interpersonal violence,” they continued. “Nearly 265 million firearms are in civilian hands in the United States, and a 44% increase in pediatric firearm mortality rate has been documented during the past 5 years.”

The FACTS Consortium defined an agenda “to serve as a guide for future research efforts to decrease pediatric death and injury.”

Some of the research agenda items include the following:

• Understanding epidemiologic trends and how demographic factors are associated with fatal and nonfatal outcomes.
• The long-term cost associated with pediatric firearm outcomes.
• The effectiveness of health care–focused primary prevention strategies for children, adolescents, and their families to reduce firearm outcomes.
• Examination of health care–based interventions for children and adolescents who have experienced or witnessed a firearm injury to prevent (or reduce) subsequent firearm outcomes including firearm injury recidivism and mental health, socioemotional, and educational outcomes.

“It is an excellent list, and I think it was very thoughtful to come out with a research agenda for firearm safety for kids,” Marlene Melzer-Lange, MD, chair of the American Academy of Pediatrics subcommittee on violence prevention, said in an interview. “The thing that’s been lacking nationally has been funding for firearm injury prevention, both for children and adults, and to have something focused specifically on children – because children have the biggest chance of living the longest – is really important.”

She described the research agenda as being “comprehensive” and didn’t see anything that stood out as missing from the list, although the big issue that remains is getting funding for the research now that an agenda has been outlined.

And getting that is not going to be easy.

“I think it’s going to take some political will of the people and particularly of our legislators ... to allow the [Centers for Disease Control and Prevention] to provide funding” said Dr. Melzer-Lange, an attending emergency department physician at Children’s Hospital of Wisconsin, Milwaukee, adding that AAP has long been asking for such funding. “It’s going [to come down to] the legislators [having] enough guts to actually put the funding in and not be afraid of external groups that might ask them not to do that.”

She remains optimistic and hopeful that the legislators are going to look at it and will eventually say that this is an crisis and will take more action beyond just talking about it.

Dr. Melzer-Lange also emphasized that this is about firearm safety and not firearm control, especially for children and teenagers.

All authors reported receiving grants from National Institutes of Health/National Institute of Child Health and Human Development.

gtwachtman@mdedge.com

SOURCE: Cunningham RM et al. JAMA Pediatrics. 2019. doi: 10.1001/jamapediatrics.2019.1494.

SAN ANTONIO – Key determinants of no-show rates to a sleep clinic include being a new patient and lacking health insurance coverage, results from a single-center study showed.

Doug Brunk/MDedge News

“There are lots of people with sleep problems,” one of the study’s authors, Alvan Nzewuihe, MD, said in an interview at the annual meeting of the Associated Professional Sleep Societies. “However, we have to identify these patients to be able to help them. If they don’t come to the sleep clinic [for assessment], we are going nowhere.”

In an effort to better understand determinants of no-show rates among sleep clinics, Dr. Nzewuihe and colleagues performed a 10-month, retrospective chart review of 2,532 patients scheduled at Saint Louis University’s SLUCare Sleep Disorders Center during the months of July and October 2017 and April 2018. A no-show was determined if the patient failed to show up at their scheduled appointment on time or if they canceled their appointment. The researchers used multivariable logistic regression to determine which factors were independently associated with no-show rates.

Dr. Nzewuihe, a sleep medicine physician at the university, reported that the overall no-show rate during the study period was 21.2% and did not change with age, sex, or appointment factors such as time of day, day of week, or season of the year. Significant determinants of no-show rates included being a new patient (39.1% vs. 28.8% among established patients; P less than .0001) and having no health insurance (47.5% vs. public 28.3% vs private 24.2%; P less than .0001). Multivariate logistic regression confirmed the associations. New patients were 1.96 times more likely to not show up to the sleep clinic, compared with established patients, while patients with no health insurance coverage were 1.55 times more likely to not show up, compared with those with public health insurance.

The researchers wrote in their poster abstract, “Patients who are new to the clinic or have no insurance coverage have a higher odds of not showing up to their appointment and delaying their care. Efforts to prioritize high-risk patients of nonadherence will help contribute to better care and outcomes. Further studies are needed to develop methods to decrease no-show rates once high-risk appointments have been identified.”

Dr. Nzewuihe acknowledged certain limitations of the study, including its retrospective design and the fact that other possible contributing factors were not evaluated such as literacy level, employment status, and length of time between appointment booking and appointment date. The study’s first author was Julie Sahrmann, DO. The researchers reported having no financial disclosures.

dbrunk@mdedge.com

SOURCE: Sahrmann J et al. Sleep 2019, Abstract 0965.

SAN ANTONIO – Key determinants of no-show rates to a sleep clinic include being a new patient and lacking health insurance coverage, results from a single-center study showed.

Doug Brunk/MDedge News

“There are lots of people with sleep problems,” one of the study’s authors, Alvan Nzewuihe, MD, said in an interview at the annual meeting of the Associated Professional Sleep Societies. “However, we have to identify these patients to be able to help them. If they don’t come to the sleep clinic [for assessment], we are going nowhere.”

In an effort to better understand determinants of no-show rates among sleep clinics, Dr. Nzewuihe and colleagues performed a 10-month, retrospective chart review of 2,532 patients scheduled at Saint Louis University’s SLUCare Sleep Disorders Center during the months of July and October 2017 and April 2018. A no-show was determined if the patient failed to show up at their scheduled appointment on time or if they canceled their appointment. The researchers used multivariable logistic regression to determine which factors were independently associated with no-show rates.

Dr. Nzewuihe, a sleep medicine physician at the university, reported that the overall no-show rate during the study period was 21.2% and did not change with age, sex, or appointment factors such as time of day, day of week, or season of the year. Significant determinants of no-show rates included being a new patient (39.1% vs. 28.8% among established patients; P less than .0001) and having no health insurance (47.5% vs. public 28.3% vs private 24.2%; P less than .0001). Multivariate logistic regression confirmed the associations. New patients were 1.96 times more likely to not show up to the sleep clinic, compared with established patients, while patients with no health insurance coverage were 1.55 times more likely to not show up, compared with those with public health insurance.

The researchers wrote in their poster abstract, “Patients who are new to the clinic or have no insurance coverage have a higher odds of not showing up to their appointment and delaying their care. Efforts to prioritize high-risk patients of nonadherence will help contribute to better care and outcomes. Further studies are needed to develop methods to decrease no-show rates once high-risk appointments have been identified.”

Dr. Nzewuihe acknowledged certain limitations of the study, including its retrospective design and the fact that other possible contributing factors were not evaluated such as literacy level, employment status, and length of time between appointment booking and appointment date. The study’s first author was Julie Sahrmann, DO. The researchers reported having no financial disclosures.

dbrunk@mdedge.com

SOURCE: Sahrmann J et al. Sleep 2019, Abstract 0965.

SAN ANTONIO – Key determinants of no-show rates to a sleep clinic include being a new patient and lacking health insurance coverage, results from a single-center study showed.

Doug Brunk/MDedge News

“There are lots of people with sleep problems,” one of the study’s authors, Alvan Nzewuihe, MD, said in an interview at the annual meeting of the Associated Professional Sleep Societies. “However, we have to identify these patients to be able to help them. If they don’t come to the sleep clinic [for assessment], we are going nowhere.”

In an effort to better understand determinants of no-show rates among sleep clinics, Dr. Nzewuihe and colleagues performed a 10-month, retrospective chart review of 2,532 patients scheduled at Saint Louis University’s SLUCare Sleep Disorders Center during the months of July and October 2017 and April 2018. A no-show was determined if the patient failed to show up at their scheduled appointment on time or if they canceled their appointment. The researchers used multivariable logistic regression to determine which factors were independently associated with no-show rates.

Dr. Nzewuihe, a sleep medicine physician at the university, reported that the overall no-show rate during the study period was 21.2% and did not change with age, sex, or appointment factors such as time of day, day of week, or season of the year. Significant determinants of no-show rates included being a new patient (39.1% vs. 28.8% among established patients; P less than .0001) and having no health insurance (47.5% vs. public 28.3% vs private 24.2%; P less than .0001). Multivariate logistic regression confirmed the associations. New patients were 1.96 times more likely to not show up to the sleep clinic, compared with established patients, while patients with no health insurance coverage were 1.55 times more likely to not show up, compared with those with public health insurance.

The researchers wrote in their poster abstract, “Patients who are new to the clinic or have no insurance coverage have a higher odds of not showing up to their appointment and delaying their care. Efforts to prioritize high-risk patients of nonadherence will help contribute to better care and outcomes. Further studies are needed to develop methods to decrease no-show rates once high-risk appointments have been identified.”

Dr. Nzewuihe acknowledged certain limitations of the study, including its retrospective design and the fact that other possible contributing factors were not evaluated such as literacy level, employment status, and length of time between appointment booking and appointment date. The study’s first author was Julie Sahrmann, DO. The researchers reported having no financial disclosures.

dbrunk@mdedge.com

SOURCE: Sahrmann J et al. Sleep 2019, Abstract 0965.

SEATTLE – The one constant in multiple sclerosis (MS) is its lack of constancy. “As our lives are dynamic and change over time, MS is also dynamic and changes over time.” This is especially the case in women, noted Mitzi Joi Williams, MD.

bowdenimages/iStock/Getty Images

About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.

In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.

Pay attention to sexual dysfunction

Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”

She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.

In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.

Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”

Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.

Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”

Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”

“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.

To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.

• Fatigue – stimulants, diet, exercise.
• Spasticity – muscle relaxants, exercise.
• Bladder dysfunction – fluid restriction, medication.
• Paresthesia – antidepressants, anticonvulsants.
• Numbness – vibrators, devices to increase stimulation.

Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.

Be aware of special needs during prepregnancy and pregnancy

“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”

In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.

Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”

What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”

Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.

Menopause and MS symptoms may overlap

Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”

A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.

Dr. Williams reported no relevant financial disclosures.

SEATTLE – The one constant in multiple sclerosis (MS) is its lack of constancy. “As our lives are dynamic and change over time, MS is also dynamic and changes over time.” This is especially the case in women, noted Mitzi Joi Williams, MD.

bowdenimages/iStock/Getty Images

About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.

In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.

Pay attention to sexual dysfunction

Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”

She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.

In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.

Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”

Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.

Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”

Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”

“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.

To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.

• Fatigue – stimulants, diet, exercise.
• Spasticity – muscle relaxants, exercise.
• Bladder dysfunction – fluid restriction, medication.
• Paresthesia – antidepressants, anticonvulsants.
• Numbness – vibrators, devices to increase stimulation.

Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.

Be aware of special needs during prepregnancy and pregnancy

“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”

In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.

Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”

What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”

Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.

Menopause and MS symptoms may overlap

Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”

A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.

Dr. Williams reported no relevant financial disclosures.

SEATTLE – The one constant in multiple sclerosis (MS) is its lack of constancy. “As our lives are dynamic and change over time, MS is also dynamic and changes over time.” This is especially the case in women, noted Mitzi Joi Williams, MD.

bowdenimages/iStock/Getty Images

About three in four people with MS are female – about 750,000 in the United States. And the risk and incidence may be highest in African American women.

In a presentation about the unique needs of women with MS, Dr. Williams, an assistant professor of internal medicine at the Morehouse School of Medicine in Atlanta, offered these tips at the annual meeting of the Consortium of Multiple Sclerosis Centers.

Pay attention to sexual dysfunction

Patients with MS often are ashamed to talk about sexual dysfunction, Dr. Williams said, but it is on many minds. “If I have a program on intimacy in MS, people are out the door.”

She urged colleagues to understand that MS can affect sexuality through three routes: primary, secondary, and tertiary dysfunction.

In primary sexual dysfunction, brain and spinal lesions directly related to MS can cause problems such as lack of sensation or abnormal sensations, decreased libido, vaginal dryness, and difficult orgasm.

Secondary sexual dysfunction refers to problems caused by symptoms of MS such as fatigue, which can worsen as the day progresses and affect nighttime intimacy, she said. Bladder dysfunction is another sensitive area in sexuality, with patients – especially women – “concerned that they will lose control of their bladder or they have already lost control.”

Cognitive dysfunction also can disrupt sexual function. “It is important to focus, and certain things cannot happen if you do not. If you are not able to focus and concentrate, it can affect interest,” Dr. Williams said.

Additionally, medications can improve some symptoms while making others worse. For example, a drug may relieve spasticity but boost fatigue. “We have to walk this tightrope,” she said. “But if we are not asking our patients, they may not volunteer this information.”

Finally, she said, MS can spark tertiary sexual dysfunction – poor body image, depression, anxiety, and disruptive changes in familial roles. For example, one partner may become a caregiver, and “it is hard to go from caregiving to sexy time.”

“It is something we have to acknowledge and find ways to deal with,” Dr. Williams said.

To address these issues, she pointed to strategies for symptomatic relief and disease-modifying therapy (DMT) and pinpointed several treatment options.

• Fatigue – stimulants, diet, exercise.
• Spasticity – muscle relaxants, exercise.
• Bladder dysfunction – fluid restriction, medication.
• Paresthesia – antidepressants, anticonvulsants.
• Numbness – vibrators, devices to increase stimulation.

Sexual therapy, couples therapy, and pelvic floor physical therapy also can be helpful.

Be aware of special needs during prepregnancy and pregnancy

“MS itself does not have a lot of effects on fertility, pregnancy, or pregnancy outcomes,” Dr. Williams said. However, “medications cause concern about how we manage pregnancy and fertility.”

In vitro fertilization may increase the risk of relapse, she added, and patients on dimethyl fumarate who experience vomiting or diarrhea may not be able to properly absorb oral contraceptives.

Women with MS may not need to go off DMT when they are trying to conceive, she said. “If patients have very aggressive disease, they may need to be on DMT through conception, through the first trimester, and even the entire pregnancy to prevent long-term disability.”

What about pregnancy itself? “An MS diagnosis alone does not mean that a pregnancy is high risk,” she said. “There are not necessarily additional tests and ultrasounds that are recommended for our patients based on MS diagnosis alone.”

Treatment discontinuation may be warranted during pregnancy, when MS generally improves. However, some MS symptoms – fatigue, bladder dysfunction, and balance – may increase. Corticosteroids can be appropriate if relapses occur during pregnancy.

Menopause and MS symptoms may overlap

Symptoms such as hot flashes, mood changes, sleep disturbance, bladder dysfunction, and decreased energy may be signs of MS, or they could indicate menopause, Dr. Williams said. “Sometimes patients come in and they are getting worse, and we look into it and discover they are premenopausal.”

A decline in estrogen during menopause may worsen MS symptoms, she added, and hormone therapy may be appropriate. A phase 2 study found a benefit in menopausal patients with MS for estriol in conjunction with a DMT, but more studies are needed.

Dr. Williams reported no relevant financial disclosures.

The other day, I saw a patient who really brought home the importance of considering culture in psychiatry. The patient’s chief complaint was that he had been hearing the voice of an “invisible man.” I noticed he had an accent I was familiar with, and it sounded like he was from Haiti. Indeed, he was born there.

Accordingly, I asked him about voodoo. He said he is not a voodoo worshiper but he believes in voodoo – and he thought that that was what was happening to him. He reported this was the second time he heard the voices – the last time was less than a year ago. He said he came to the hospital because he was trying to wash dishes when he felt some invisible force holding him down. The patient got upset, and he broke the dishes he was washing. Of course, a big melee ensued, and the police were called. They brought the patient to my hospital.

When I spoke with him, he said he was doing pretty well with his Parkinson’s disease but he was a little stiff. The patient was on carbidopa-levodopa 25-100 mg 1.5 t.i.d. for his Parkinson’s, quetiapine 50 mg b.i.d. for his psychotic symptoms, amantadine 100 mg b.i.d. to stimulate his dopamine, ropinirole 1 mg t.i.d. for restless legs, and baclofen 10 mg t.i.d. for muscle spasms.

This is a 66-year-old male who was appropriately groomed and who was cooperative with the interview. He was not hyperactive or lethargic. His mood was euthymic, and he had a wide range of affect as he was able to smile, get serious, and be sad (about his problems). His speech was relevant, linear, and goal directed. His thought processes did not show any signs of loose associations, tangentiality or circumstantiality, but he did have delusions, and current auditory and visual hallucinations. His thought content was surrounding his problems, which because of the culture he is from, were attributed by him to voodoo. He was attentive, and his recent and remote memory were intact. Clinical estimate of his intelligence was average. Despite my explaining to him that his psychotic symptoms were caused by the medication he was taking, his judgment and insight were fair as he explained to me the things that were happening to him were so tangible they had to be real. He had no suicidal or homicidal ideation.

I decided to leave his meds as is, and I gave him 25 mg loxapine at h.s.

When I saw him a few days later, I asked him how he was doing, and he reported that the invisible man and all of his shenanigans were gone. I again explained that the medication he was taking for his Parkinson’s was causing his psychotic symptoms, and now I had proof. Had the voices been tied to voodoo, the medication would not have stopped the symptoms. He looked skeptical.

This struck me as a perfect example of the importance of culture in psychiatry, and I thought it instructive to share.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit; clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago; former president/CEO of the Community Mental Health Council; and former director of the Institute for Juvenile Research (birthplace of child psychiatry), all in Chicago. He is recipient of the American Psychiatric Association’s 2019 Adolph Meyer Award for Lifetime Achievement in Psychiatric Research. Check out Dr. Bell’s new book, Fetal Alcohol Exposure in the African-American Community, at https://thirdworldpressfoundation.org/product/pre-order-fetal-alcohol-exposure-in-the-african-american-community.

The other day, I saw a patient who really brought home the importance of considering culture in psychiatry. The patient’s chief complaint was that he had been hearing the voice of an “invisible man.” I noticed he had an accent I was familiar with, and it sounded like he was from Haiti. Indeed, he was born there.

Accordingly, I asked him about voodoo. He said he is not a voodoo worshiper but he believes in voodoo – and he thought that that was what was happening to him. He reported this was the second time he heard the voices – the last time was less than a year ago. He said he came to the hospital because he was trying to wash dishes when he felt some invisible force holding him down. The patient got upset, and he broke the dishes he was washing. Of course, a big melee ensued, and the police were called. They brought the patient to my hospital.

When I spoke with him, he said he was doing pretty well with his Parkinson’s disease but he was a little stiff. The patient was on carbidopa-levodopa 25-100 mg 1.5 t.i.d. for his Parkinson’s, quetiapine 50 mg b.i.d. for his psychotic symptoms, amantadine 100 mg b.i.d. to stimulate his dopamine, ropinirole 1 mg t.i.d. for restless legs, and baclofen 10 mg t.i.d. for muscle spasms.

This is a 66-year-old male who was appropriately groomed and who was cooperative with the interview. He was not hyperactive or lethargic. His mood was euthymic, and he had a wide range of affect as he was able to smile, get serious, and be sad (about his problems). His speech was relevant, linear, and goal directed. His thought processes did not show any signs of loose associations, tangentiality or circumstantiality, but he did have delusions, and current auditory and visual hallucinations. His thought content was surrounding his problems, which because of the culture he is from, were attributed by him to voodoo. He was attentive, and his recent and remote memory were intact. Clinical estimate of his intelligence was average. Despite my explaining to him that his psychotic symptoms were caused by the medication he was taking, his judgment and insight were fair as he explained to me the things that were happening to him were so tangible they had to be real. He had no suicidal or homicidal ideation.

I decided to leave his meds as is, and I gave him 25 mg loxapine at h.s.

When I saw him a few days later, I asked him how he was doing, and he reported that the invisible man and all of his shenanigans were gone. I again explained that the medication he was taking for his Parkinson’s was causing his psychotic symptoms, and now I had proof. Had the voices been tied to voodoo, the medication would not have stopped the symptoms. He looked skeptical.

This struck me as a perfect example of the importance of culture in psychiatry, and I thought it instructive to share.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit; clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago; former president/CEO of the Community Mental Health Council; and former director of the Institute for Juvenile Research (birthplace of child psychiatry), all in Chicago. He is recipient of the American Psychiatric Association’s 2019 Adolph Meyer Award for Lifetime Achievement in Psychiatric Research. Check out Dr. Bell’s new book, Fetal Alcohol Exposure in the African-American Community, at https://thirdworldpressfoundation.org/product/pre-order-fetal-alcohol-exposure-in-the-african-american-community.

The other day, I saw a patient who really brought home the importance of considering culture in psychiatry. The patient’s chief complaint was that he had been hearing the voice of an “invisible man.” I noticed he had an accent I was familiar with, and it sounded like he was from Haiti. Indeed, he was born there.

Accordingly, I asked him about voodoo. He said he is not a voodoo worshiper but he believes in voodoo – and he thought that that was what was happening to him. He reported this was the second time he heard the voices – the last time was less than a year ago. He said he came to the hospital because he was trying to wash dishes when he felt some invisible force holding him down. The patient got upset, and he broke the dishes he was washing. Of course, a big melee ensued, and the police were called. They brought the patient to my hospital.

When I spoke with him, he said he was doing pretty well with his Parkinson’s disease but he was a little stiff. The patient was on carbidopa-levodopa 25-100 mg 1.5 t.i.d. for his Parkinson’s, quetiapine 50 mg b.i.d. for his psychotic symptoms, amantadine 100 mg b.i.d. to stimulate his dopamine, ropinirole 1 mg t.i.d. for restless legs, and baclofen 10 mg t.i.d. for muscle spasms.

This is a 66-year-old male who was appropriately groomed and who was cooperative with the interview. He was not hyperactive or lethargic. His mood was euthymic, and he had a wide range of affect as he was able to smile, get serious, and be sad (about his problems). His speech was relevant, linear, and goal directed. His thought processes did not show any signs of loose associations, tangentiality or circumstantiality, but he did have delusions, and current auditory and visual hallucinations. His thought content was surrounding his problems, which because of the culture he is from, were attributed by him to voodoo. He was attentive, and his recent and remote memory were intact. Clinical estimate of his intelligence was average. Despite my explaining to him that his psychotic symptoms were caused by the medication he was taking, his judgment and insight were fair as he explained to me the things that were happening to him were so tangible they had to be real. He had no suicidal or homicidal ideation.

I decided to leave his meds as is, and I gave him 25 mg loxapine at h.s.

When I saw him a few days later, I asked him how he was doing, and he reported that the invisible man and all of his shenanigans were gone. I again explained that the medication he was taking for his Parkinson’s was causing his psychotic symptoms, and now I had proof. Had the voices been tied to voodoo, the medication would not have stopped the symptoms. He looked skeptical.

This struck me as a perfect example of the importance of culture in psychiatry, and I thought it instructive to share.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit; clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago; former president/CEO of the Community Mental Health Council; and former director of the Institute for Juvenile Research (birthplace of child psychiatry), all in Chicago. He is recipient of the American Psychiatric Association’s 2019 Adolph Meyer Award for Lifetime Achievement in Psychiatric Research. Check out Dr. Bell’s new book, Fetal Alcohol Exposure in the African-American Community, at https://thirdworldpressfoundation.org/product/pre-order-fetal-alcohol-exposure-in-the-african-american-community.

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

Only a limited number of patients with von Willebrand disease (VWD) are likely to require treatment with von Willebrand factor (VWF) concentrates, according to results from a retrospective analysis.

The retrospective, single-center study sought to describe the need for VWF concentrates in a “real-life setting,” wrote Ana Boban, MD, PhD, of the University of Zagreb (Croatia) and colleagues in a letter to the editor published in Haemophilia. The study was conducted at the Saint‐Luc University Hospital in Brussels and included all the VWD patients listed in the hospital registry from 2000 to 2015.

The researchers evaluated the necessity for VWF concentrate therapy based on VWD severity (mild, moderate, severe) and type (1, 2A, 2B, 2M, 2N, or 3), in addition to therapeutic indication. Bleeding scores were not included in the analysis.

A total of 174 patients with VWD were included in the study, which consisted of 116 females and 61 males aged 3-81 years. The majority of study participants had type 1 VWD (n = 118, 67%).

Data collected included patient demographic information, disease type and severity, responses to specific tests, and type of treatment received.

The researchers found that just 18% (n = 32) of patients within the cohort required therapy with VWF concentrates over the study period. Additionally, the team reported that a large number of patients did not require any therapy (n = 79; 45%) or were or managed with DDAVP (desmopressin) (n = 61; 64%).

“When assessing the results according to disease severity, it clearly occurred that most patients with severe disease required VWF concentrates [100%, 75%, and 100% for type 1, type 2, and type 3 VWD, respectively],” the authors wrote.

With respect to therapeutic indication, apart from prophylaxis, contraindications to DDAVP (n = 5) and unresponsiveness to DDAVP (n = 7) were absolute indications for the use of VWF concentrates among patients within the cohort.

“Our study has confirmed that the absolute indications for using VWF concentrates in VWD patients are prophylaxis, major surgeries, and nonresponsiveness/contraindications to DDAVP,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Boban A et al. Haemophilia. 2019 May 20. doi: 10.1111/hae.13779.

LJUBLJANA, SLOVENIA – An effective strategy in helping vaccine skeptics to come around to accepting immunizations for their children is to pivot the conversation away from vaccine safety and focus instead on the disease itself and its potential consequences, Saad B. Omer, MBBS, PhD, asserted at the annual meeting of the European Society for Paediatric Infectious Diseases.

Bruce Jancin/MDedge News

“Why do we cede ground by focusing too much on the vaccine itself? At the end of the day, vaccination is a means to prevent disease. So I suggest that we talk about the disease. I call it the disease salience approach,” said Dr. Omer, professor of global health, epidemiology, and pediatrics at Emory University in Atlanta.

It’s a strategy guided by developments in social psychology, persuasion theory, and communication theory. But if applied incorrectly, the disease salience approach can backfire, causing behavioral paralysis and an inability to act, he cautioned.

Dr. Omer explained that it’s a matter of framing.

“Always include a solution to promote self-efficacy and response-efficacy. After you inform parents of disease risks, provide them with actions they can take. Now readdress the vaccine, pointing out that this is the single best way to protect yourself and your baby,” he said. “The lesson is that since vaccines are a social norm, reframe nonvaccination as an active act, rather than vaccination as an active act.”

Don’t attempt to wow parents with statistics on how vaccine complication rates are dwarfed by the disease risk if left unvaccinated, he advised. Studies have shown that‘s generally not effective. What actually works is to provide narratives of disease severity.

“We are excellent linguists, but really, really poor statisticians,” Dr. Omer observed.

Is it ethical to talk to parents about disease risks to influence their behavior? Absolutely, in his view.

“We’re not selling toothpaste. We are in the business of life-saving vaccines. And I would submit that if it’s done correctly it’s entirely ethical to talk about the disease, and sometimes even the severe risks of the disease, instead of the vaccine,” said Dr. Omer.

If parents cite a myth about vaccines, it’s necessary to address it head on without lingering on it. But debunking a myth is tricky because people tend to remember negative information they received earlier.

“If you’re going to debunk a myth, clearly label it as a myth in the headline as you introduce it. State why it’s not true. Replace the myth with the best alternative explanation. Think of it like a blank space where the myth used to reside. That space needs to be filled with an alternative explanation or the myth will come back,” Dr. Omer said.

He is a coauthor of a book titled, ‘The Clinician’s Vaccine Safety Resource Guide: Optimizing Prevention of Vaccine-Preventable Diseases Across the Lifespan.’

bjancin@mdedge.com

LJUBLJANA, SLOVENIA – An effective strategy in helping vaccine skeptics to come around to accepting immunizations for their children is to pivot the conversation away from vaccine safety and focus instead on the disease itself and its potential consequences, Saad B. Omer, MBBS, PhD, asserted at the annual meeting of the European Society for Paediatric Infectious Diseases.

Bruce Jancin/MDedge News

“Why do we cede ground by focusing too much on the vaccine itself? At the end of the day, vaccination is a means to prevent disease. So I suggest that we talk about the disease. I call it the disease salience approach,” said Dr. Omer, professor of global health, epidemiology, and pediatrics at Emory University in Atlanta.

It’s a strategy guided by developments in social psychology, persuasion theory, and communication theory. But if applied incorrectly, the disease salience approach can backfire, causing behavioral paralysis and an inability to act, he cautioned.

Dr. Omer explained that it’s a matter of framing.

“Always include a solution to promote self-efficacy and response-efficacy. After you inform parents of disease risks, provide them with actions they can take. Now readdress the vaccine, pointing out that this is the single best way to protect yourself and your baby,” he said. “The lesson is that since vaccines are a social norm, reframe nonvaccination as an active act, rather than vaccination as an active act.”

Don’t attempt to wow parents with statistics on how vaccine complication rates are dwarfed by the disease risk if left unvaccinated, he advised. Studies have shown that‘s generally not effective. What actually works is to provide narratives of disease severity.

“We are excellent linguists, but really, really poor statisticians,” Dr. Omer observed.

Is it ethical to talk to parents about disease risks to influence their behavior? Absolutely, in his view.

“We’re not selling toothpaste. We are in the business of life-saving vaccines. And I would submit that if it’s done correctly it’s entirely ethical to talk about the disease, and sometimes even the severe risks of the disease, instead of the vaccine,” said Dr. Omer.

If parents cite a myth about vaccines, it’s necessary to address it head on without lingering on it. But debunking a myth is tricky because people tend to remember negative information they received earlier.

“If you’re going to debunk a myth, clearly label it as a myth in the headline as you introduce it. State why it’s not true. Replace the myth with the best alternative explanation. Think of it like a blank space where the myth used to reside. That space needs to be filled with an alternative explanation or the myth will come back,” Dr. Omer said.

He is a coauthor of a book titled, ‘The Clinician’s Vaccine Safety Resource Guide: Optimizing Prevention of Vaccine-Preventable Diseases Across the Lifespan.’

bjancin@mdedge.com

LJUBLJANA, SLOVENIA – An effective strategy in helping vaccine skeptics to come around to accepting immunizations for their children is to pivot the conversation away from vaccine safety and focus instead on the disease itself and its potential consequences, Saad B. Omer, MBBS, PhD, asserted at the annual meeting of the European Society for Paediatric Infectious Diseases.

Bruce Jancin/MDedge News

“Why do we cede ground by focusing too much on the vaccine itself? At the end of the day, vaccination is a means to prevent disease. So I suggest that we talk about the disease. I call it the disease salience approach,” said Dr. Omer, professor of global health, epidemiology, and pediatrics at Emory University in Atlanta.

It’s a strategy guided by developments in social psychology, persuasion theory, and communication theory. But if applied incorrectly, the disease salience approach can backfire, causing behavioral paralysis and an inability to act, he cautioned.

Dr. Omer explained that it’s a matter of framing.

“Always include a solution to promote self-efficacy and response-efficacy. After you inform parents of disease risks, provide them with actions they can take. Now readdress the vaccine, pointing out that this is the single best way to protect yourself and your baby,” he said. “The lesson is that since vaccines are a social norm, reframe nonvaccination as an active act, rather than vaccination as an active act.”

Don’t attempt to wow parents with statistics on how vaccine complication rates are dwarfed by the disease risk if left unvaccinated, he advised. Studies have shown that‘s generally not effective. What actually works is to provide narratives of disease severity.

“We are excellent linguists, but really, really poor statisticians,” Dr. Omer observed.

Is it ethical to talk to parents about disease risks to influence their behavior? Absolutely, in his view.

“We’re not selling toothpaste. We are in the business of life-saving vaccines. And I would submit that if it’s done correctly it’s entirely ethical to talk about the disease, and sometimes even the severe risks of the disease, instead of the vaccine,” said Dr. Omer.

If parents cite a myth about vaccines, it’s necessary to address it head on without lingering on it. But debunking a myth is tricky because people tend to remember negative information they received earlier.

“If you’re going to debunk a myth, clearly label it as a myth in the headline as you introduce it. State why it’s not true. Replace the myth with the best alternative explanation. Think of it like a blank space where the myth used to reside. That space needs to be filled with an alternative explanation or the myth will come back,” Dr. Omer said.

He is a coauthor of a book titled, ‘The Clinician’s Vaccine Safety Resource Guide: Optimizing Prevention of Vaccine-Preventable Diseases Across the Lifespan.’

bjancin@mdedge.com

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

Undergoing hepatectomy for the treatment of hepatocellular carcinoma in patients with von Willebrand disease or hemophilia A was found to be safe through the use of appropriate von Willebrand factor concentrate or recombinant factor VIII therapy, according to findings from a small study.

“The aim of the present study was to evaluate the safety of hepatectomy in patients with inherited blood coagulation disorders through appropriate coagulation factor replacement,” wrote Kosuke Kobayashi, MD, PhD, of the University of Tokyo and colleagues. The findings were published in Haemophilia.

The researchers retrospectively studied seven patients with hemophilia A and three patients with von Willebrand disease who underwent hepatectomy for the treatment of hepatocellular carcinoma. Specific regimens of von Willebrand factor concentrate or recombinant factor VIII therapy were administered perioperatively in these patients.

Study participants were matched in a 2:1 ratio to 20 patients without a bleeding disorder who also underwent hepatectomy. Various intraoperative and postoperative outcomes were compared between the two groups.

The researchers found no significant differences in estimated blood loss (P = .748), operative time (P = .359), or red blood cell transfusion rate (P = .605) between the bleeding disorder and nonbleeding disorder groups.

Additionally, there were no significant differences seen for mortality rate (P greater than .999) or major complication rate (P = .605).

“Even repeated hepatectomy can be safely performed in these patients, similar to patients without coagulation disorders,” the authors wrote.

Dr. Kobayashi and colleagues acknowledged two key limitations of the study were the small sample size and retrospective design.

“The administration protocol reported in the present study would certainly help surgeons when planning hepatectomy in patients with coagulation disorders,” they concluded.

No funding sources were reported. The authors reported having no conflicts of interest.

SOURCE: Kobayashi K et al. Haemophilia. 2019 May 29. doi: 10.1111/hae.13759.

MILAN – In a region where scabies is endemic, a strategy of mass drug treatment for scabies resulted in durable reduction of both scabies and impetigo, findings that may have implications for future treatment of scabies or other infestations in other regions, dermatologist Margot Whitfield, MD, said at the World Congress of Dermatology.

“Mass drug administration is highly effective and safe in the treatment of endemic scabies,” she said.

Using a strategy of directly observed treatment (DOT) with oral ivermectin or topical permethrin for all residents of two separate island groups in Fiji, Dr. Whitfield, together with epidemiologist Lucia Romani, PhD, both of the University of New South Wales, Sydney, and coinvestigators, demonstrated large and sustained decreases in the rates of scabies and impetigo (N Engl J Med. 2015 Dec 10;373[24]:2305-13).

Across study arms, which included a usual care arm, the baseline rate for scabies ranged from 30% to 40%. With usual care, the rate dropped from 36.6% to 18.8% at the end of 12 months, a relative reduction of 49%. However, the 15.8% prevalence rate 12 months after permethrin DOT (from 41.7%), and the 1.9% rate 12 months after ivermectin DOT (from 32.1%) – reductions of 62% and 94%, respectively – represented much larger decreases, “especially since these reductions were seen without any further interventions,” Dr. Whitfield said. “This was extremely exciting, and a game-changer as far as the management of endemic scabies is concerned.”

At baseline, impetigo rates hovered around 20%-25%, and usual care resulted in a 32% reduction at 12 months. With permethrin DOT, the impetigo rate dropped by 54%; with ivermectin DOT, the impetigo rate dropped by 67%. “The community level of impetigo went down, purely as a result of treating the scabies,” Dr. Whitfield said.

The outcomes of this study, she noted, “have contributed to the global discussion of the treatment of scabies.”

Two years after the mass drug administration (MDA) campaign, scabies prevalence remained much lower than at baseline, with clinical scabies diagnosed in 15.2% of the usual care group, 13.5% of the permethrin group, and just 3.6% of the ivermectin group. “The exciting thing for us was that these levels ... were able to be sustained at 2 years,” Dr. Whitfield noted.

The islands that had received ivermectin saw a continued decline in impetigo prevalence as well: By 24 months, impetigo was seen in 2.6% of participants in that arm.


Scabies is a neglected – but highly treatable – tropical disease, she noted. It is associated with intense pruritus, which results in reduced quality of life, and excoriations predispose those affected to bacterial superinfections, commonly impetigo in the young.

In Fiji, the scabies mite infests nearly 40% of those aged 5-9 years, and over one-third of those younger than 5 years. Rates drop steeply with increasing age and then climb again for the elderly; still, prevalence tops 10% for all Fijian age groups, Dr. Whitfield pointed out. Overall, scabies prevalence is 23% in Fiji, with resultant impetigo affecting 19% of the population.

Providing more details about the study, she said that she and her collaborators – working in conjunction with the Fijian Ministry of Health – took advantage of the geography of the island country, whose 850,000 residents live on 300 islands, to compare mass drug treatment with either ivermectin or permethrin with usual care. “We actually didn’t look for ‘infected scabies,’ ” she explained. “We looked for scabies as one outcome, and infection as another.”

The study was designed to take advantage of lessons from previous public health work addressing filariasis and soil-transmitted helminths, and addressed the following question: In Fiji, could a single round of MDA for scabies control lead to sustained reductions in scabies and impetigo prevalence 12 months later, compared with standard care?

For one MDA arm, island residents received oral ivermectin via DOT. A second DOT dose was administered for those with clinically diagnosed scabies. For pregnant and breastfeeding women, children weighing less than 15 kg, and those with ivermectin hypersensitivity, permethrin was used, Dr. Whitfield said.

The individuals in the permethrin MDA arm received one topical dose via DOT, with a second round of topical permethrin for those with topical scabies.

In all, 803 Fijians were assigned to receive standard of care, 532 permethrin MDA, and 716 ivermectin MDA. Of these, 623 received ivermectin DOT, and 93 received permethrin. In all, DOT was achieved for 96% of those receiving the first dose. At baseline, 230 patients had scabies, with 200 receiving ivermectin and 30 permethrin; the DOT rate was 100% for the second dose.

For the permethrin arm, just 307 of 532 participants (58%) had DOT, though all were given permethrin. Scabies was present at baseline for 222 participants, and of these, 181 had DOT. “It’s much easier to do the direct observed therapy with an oral medication than with a cream,” Dr. Whitfield said. Data were not collected for the Fijians who received usual care at community health centers.

Outcomes were clinically determined via the child skin assessment algorithm of the World Health Organization’s International Management of Childhood Illness (IMCI) guidelines.

Dr. Whitfield acknowledged that the study was not a true cluster-randomized trial, and differences existed between the communities studies. Also, “dermatoscopy was not a practical option” for this real-world trial in a resource-limited setting, but validated clinical criteria were used, she said.

Going forward, she and her colleagues are continuing to track durability of reduced scabies rates, as well as downstream sequelae such as impetigo and septicemia. Also, “we need to see whether this community- and island-based project could be scaled up to a national or regional level,” she said.

The burden of disease from scabies globally is probably underestimated, and changing migration patterns may bring endemic scabies to the doorsteps of more developed nations, prompting consideration of MDA as a strategy in expanded circumstances.

Dr. Whitfield reported that she had no relevant conflicts of interest.

koakes@mdedge.com

MILAN – In a region where scabies is endemic, a strategy of mass drug treatment for scabies resulted in durable reduction of both scabies and impetigo, findings that may have implications for future treatment of scabies or other infestations in other regions, dermatologist Margot Whitfield, MD, said at the World Congress of Dermatology.

“Mass drug administration is highly effective and safe in the treatment of endemic scabies,” she said.

Using a strategy of directly observed treatment (DOT) with oral ivermectin or topical permethrin for all residents of two separate island groups in Fiji, Dr. Whitfield, together with epidemiologist Lucia Romani, PhD, both of the University of New South Wales, Sydney, and coinvestigators, demonstrated large and sustained decreases in the rates of scabies and impetigo (N Engl J Med. 2015 Dec 10;373[24]:2305-13).

Across study arms, which included a usual care arm, the baseline rate for scabies ranged from 30% to 40%. With usual care, the rate dropped from 36.6% to 18.8% at the end of 12 months, a relative reduction of 49%. However, the 15.8% prevalence rate 12 months after permethrin DOT (from 41.7%), and the 1.9% rate 12 months after ivermectin DOT (from 32.1%) – reductions of 62% and 94%, respectively – represented much larger decreases, “especially since these reductions were seen without any further interventions,” Dr. Whitfield said. “This was extremely exciting, and a game-changer as far as the management of endemic scabies is concerned.”

At baseline, impetigo rates hovered around 20%-25%, and usual care resulted in a 32% reduction at 12 months. With permethrin DOT, the impetigo rate dropped by 54%; with ivermectin DOT, the impetigo rate dropped by 67%. “The community level of impetigo went down, purely as a result of treating the scabies,” Dr. Whitfield said.

The outcomes of this study, she noted, “have contributed to the global discussion of the treatment of scabies.”

Two years after the mass drug administration (MDA) campaign, scabies prevalence remained much lower than at baseline, with clinical scabies diagnosed in 15.2% of the usual care group, 13.5% of the permethrin group, and just 3.6% of the ivermectin group. “The exciting thing for us was that these levels ... were able to be sustained at 2 years,” Dr. Whitfield noted.

The islands that had received ivermectin saw a continued decline in impetigo prevalence as well: By 24 months, impetigo was seen in 2.6% of participants in that arm.


Scabies is a neglected – but highly treatable – tropical disease, she noted. It is associated with intense pruritus, which results in reduced quality of life, and excoriations predispose those affected to bacterial superinfections, commonly impetigo in the young.

In Fiji, the scabies mite infests nearly 40% of those aged 5-9 years, and over one-third of those younger than 5 years. Rates drop steeply with increasing age and then climb again for the elderly; still, prevalence tops 10% for all Fijian age groups, Dr. Whitfield pointed out. Overall, scabies prevalence is 23% in Fiji, with resultant impetigo affecting 19% of the population.

Providing more details about the study, she said that she and her collaborators – working in conjunction with the Fijian Ministry of Health – took advantage of the geography of the island country, whose 850,000 residents live on 300 islands, to compare mass drug treatment with either ivermectin or permethrin with usual care. “We actually didn’t look for ‘infected scabies,’ ” she explained. “We looked for scabies as one outcome, and infection as another.”

The study was designed to take advantage of lessons from previous public health work addressing filariasis and soil-transmitted helminths, and addressed the following question: In Fiji, could a single round of MDA for scabies control lead to sustained reductions in scabies and impetigo prevalence 12 months later, compared with standard care?

For one MDA arm, island residents received oral ivermectin via DOT. A second DOT dose was administered for those with clinically diagnosed scabies. For pregnant and breastfeeding women, children weighing less than 15 kg, and those with ivermectin hypersensitivity, permethrin was used, Dr. Whitfield said.

The individuals in the permethrin MDA arm received one topical dose via DOT, with a second round of topical permethrin for those with topical scabies.

In all, 803 Fijians were assigned to receive standard of care, 532 permethrin MDA, and 716 ivermectin MDA. Of these, 623 received ivermectin DOT, and 93 received permethrin. In all, DOT was achieved for 96% of those receiving the first dose. At baseline, 230 patients had scabies, with 200 receiving ivermectin and 30 permethrin; the DOT rate was 100% for the second dose.

For the permethrin arm, just 307 of 532 participants (58%) had DOT, though all were given permethrin. Scabies was present at baseline for 222 participants, and of these, 181 had DOT. “It’s much easier to do the direct observed therapy with an oral medication than with a cream,” Dr. Whitfield said. Data were not collected for the Fijians who received usual care at community health centers.

Outcomes were clinically determined via the child skin assessment algorithm of the World Health Organization’s International Management of Childhood Illness (IMCI) guidelines.

Dr. Whitfield acknowledged that the study was not a true cluster-randomized trial, and differences existed between the communities studies. Also, “dermatoscopy was not a practical option” for this real-world trial in a resource-limited setting, but validated clinical criteria were used, she said.

Going forward, she and her colleagues are continuing to track durability of reduced scabies rates, as well as downstream sequelae such as impetigo and septicemia. Also, “we need to see whether this community- and island-based project could be scaled up to a national or regional level,” she said.

The burden of disease from scabies globally is probably underestimated, and changing migration patterns may bring endemic scabies to the doorsteps of more developed nations, prompting consideration of MDA as a strategy in expanded circumstances.

Dr. Whitfield reported that she had no relevant conflicts of interest.

koakes@mdedge.com

MILAN – In a region where scabies is endemic, a strategy of mass drug treatment for scabies resulted in durable reduction of both scabies and impetigo, findings that may have implications for future treatment of scabies or other infestations in other regions, dermatologist Margot Whitfield, MD, said at the World Congress of Dermatology.

“Mass drug administration is highly effective and safe in the treatment of endemic scabies,” she said.

Using a strategy of directly observed treatment (DOT) with oral ivermectin or topical permethrin for all residents of two separate island groups in Fiji, Dr. Whitfield, together with epidemiologist Lucia Romani, PhD, both of the University of New South Wales, Sydney, and coinvestigators, demonstrated large and sustained decreases in the rates of scabies and impetigo (N Engl J Med. 2015 Dec 10;373[24]:2305-13).

Across study arms, which included a usual care arm, the baseline rate for scabies ranged from 30% to 40%. With usual care, the rate dropped from 36.6% to 18.8% at the end of 12 months, a relative reduction of 49%. However, the 15.8% prevalence rate 12 months after permethrin DOT (from 41.7%), and the 1.9% rate 12 months after ivermectin DOT (from 32.1%) – reductions of 62% and 94%, respectively – represented much larger decreases, “especially since these reductions were seen without any further interventions,” Dr. Whitfield said. “This was extremely exciting, and a game-changer as far as the management of endemic scabies is concerned.”

At baseline, impetigo rates hovered around 20%-25%, and usual care resulted in a 32% reduction at 12 months. With permethrin DOT, the impetigo rate dropped by 54%; with ivermectin DOT, the impetigo rate dropped by 67%. “The community level of impetigo went down, purely as a result of treating the scabies,” Dr. Whitfield said.

The outcomes of this study, she noted, “have contributed to the global discussion of the treatment of scabies.”

Two years after the mass drug administration (MDA) campaign, scabies prevalence remained much lower than at baseline, with clinical scabies diagnosed in 15.2% of the usual care group, 13.5% of the permethrin group, and just 3.6% of the ivermectin group. “The exciting thing for us was that these levels ... were able to be sustained at 2 years,” Dr. Whitfield noted.

The islands that had received ivermectin saw a continued decline in impetigo prevalence as well: By 24 months, impetigo was seen in 2.6% of participants in that arm.


Scabies is a neglected – but highly treatable – tropical disease, she noted. It is associated with intense pruritus, which results in reduced quality of life, and excoriations predispose those affected to bacterial superinfections, commonly impetigo in the young.

In Fiji, the scabies mite infests nearly 40% of those aged 5-9 years, and over one-third of those younger than 5 years. Rates drop steeply with increasing age and then climb again for the elderly; still, prevalence tops 10% for all Fijian age groups, Dr. Whitfield pointed out. Overall, scabies prevalence is 23% in Fiji, with resultant impetigo affecting 19% of the population.

Providing more details about the study, she said that she and her collaborators – working in conjunction with the Fijian Ministry of Health – took advantage of the geography of the island country, whose 850,000 residents live on 300 islands, to compare mass drug treatment with either ivermectin or permethrin with usual care. “We actually didn’t look for ‘infected scabies,’ ” she explained. “We looked for scabies as one outcome, and infection as another.”

The study was designed to take advantage of lessons from previous public health work addressing filariasis and soil-transmitted helminths, and addressed the following question: In Fiji, could a single round of MDA for scabies control lead to sustained reductions in scabies and impetigo prevalence 12 months later, compared with standard care?

For one MDA arm, island residents received oral ivermectin via DOT. A second DOT dose was administered for those with clinically diagnosed scabies. For pregnant and breastfeeding women, children weighing less than 15 kg, and those with ivermectin hypersensitivity, permethrin was used, Dr. Whitfield said.

The individuals in the permethrin MDA arm received one topical dose via DOT, with a second round of topical permethrin for those with topical scabies.

In all, 803 Fijians were assigned to receive standard of care, 532 permethrin MDA, and 716 ivermectin MDA. Of these, 623 received ivermectin DOT, and 93 received permethrin. In all, DOT was achieved for 96% of those receiving the first dose. At baseline, 230 patients had scabies, with 200 receiving ivermectin and 30 permethrin; the DOT rate was 100% for the second dose.

For the permethrin arm, just 307 of 532 participants (58%) had DOT, though all were given permethrin. Scabies was present at baseline for 222 participants, and of these, 181 had DOT. “It’s much easier to do the direct observed therapy with an oral medication than with a cream,” Dr. Whitfield said. Data were not collected for the Fijians who received usual care at community health centers.

Outcomes were clinically determined via the child skin assessment algorithm of the World Health Organization’s International Management of Childhood Illness (IMCI) guidelines.

Dr. Whitfield acknowledged that the study was not a true cluster-randomized trial, and differences existed between the communities studies. Also, “dermatoscopy was not a practical option” for this real-world trial in a resource-limited setting, but validated clinical criteria were used, she said.

Going forward, she and her colleagues are continuing to track durability of reduced scabies rates, as well as downstream sequelae such as impetigo and septicemia. Also, “we need to see whether this community- and island-based project could be scaled up to a national or regional level,” she said.

The burden of disease from scabies globally is probably underestimated, and changing migration patterns may bring endemic scabies to the doorsteps of more developed nations, prompting consideration of MDA as a strategy in expanded circumstances.

Dr. Whitfield reported that she had no relevant conflicts of interest.

koakes@mdedge.com

MADRID – To achieve positive trials with new agents in osteoarthritis, patient selection should be considered in the context of the primary endpoints, according to Philip G. Conaghan, MBBS, PhD, chair of musculoskeletal medicine at the University of Leeds (England).

In an interview, Dr. Conaghan explained that the issue has arisen with emerging agents that are designed for structural improvements with the expectation that symptom improvements will follow. Recapping a presentation he made at the European Congress of Rheumatology, he cautioned that the key aspects of trial design for these novel agents, including patient and endpoint selection, are particularly challenging.

As an example, Dr. Conaghan referred to the experience so far with the ongoing phase 2 FORWARD trial with sprifermin, a recombinant form of human fibroblast growth factor. In this study, sprifermin has already shown promise for growing cartilage, but the benefit accrues slowly, and there is no symptomatic improvement early in the course of treatment.


Based on the experience with FORWARD, much has been learned about a potential tension between structural and symptomatic endpoints in osteoarthritis, according to Dr. Conaghan. For one, it appears to be important to select patients most likely to achieve measurable structural improvements quickly to achieve a positive result in a reasonable period of time.

For another, it may be necessary to select symptom endpoints that reflect structural change while cautioning patients about the potential for a long delay before a clinical benefit is experienced.

In osteoarthritis, clinical benefit has been traditionally captured with relief of pain. Although an improvement in joint structure might be the best way to produce this result, this has to be proved. Reasonable and achievable endpoints are needed for emerging drugs with the potential to rebuild the joint not just to control pain, he said.

SOURCE: Gühring H et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):70-1. Abstract OP0010. doi: 10.1136/annrheumdis-2019-eular.1216.

MADRID – To achieve positive trials with new agents in osteoarthritis, patient selection should be considered in the context of the primary endpoints, according to Philip G. Conaghan, MBBS, PhD, chair of musculoskeletal medicine at the University of Leeds (England).

In an interview, Dr. Conaghan explained that the issue has arisen with emerging agents that are designed for structural improvements with the expectation that symptom improvements will follow. Recapping a presentation he made at the European Congress of Rheumatology, he cautioned that the key aspects of trial design for these novel agents, including patient and endpoint selection, are particularly challenging.

As an example, Dr. Conaghan referred to the experience so far with the ongoing phase 2 FORWARD trial with sprifermin, a recombinant form of human fibroblast growth factor. In this study, sprifermin has already shown promise for growing cartilage, but the benefit accrues slowly, and there is no symptomatic improvement early in the course of treatment.


Based on the experience with FORWARD, much has been learned about a potential tension between structural and symptomatic endpoints in osteoarthritis, according to Dr. Conaghan. For one, it appears to be important to select patients most likely to achieve measurable structural improvements quickly to achieve a positive result in a reasonable period of time.

For another, it may be necessary to select symptom endpoints that reflect structural change while cautioning patients about the potential for a long delay before a clinical benefit is experienced.

In osteoarthritis, clinical benefit has been traditionally captured with relief of pain. Although an improvement in joint structure might be the best way to produce this result, this has to be proved. Reasonable and achievable endpoints are needed for emerging drugs with the potential to rebuild the joint not just to control pain, he said.

SOURCE: Gühring H et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):70-1. Abstract OP0010. doi: 10.1136/annrheumdis-2019-eular.1216.

MADRID – To achieve positive trials with new agents in osteoarthritis, patient selection should be considered in the context of the primary endpoints, according to Philip G. Conaghan, MBBS, PhD, chair of musculoskeletal medicine at the University of Leeds (England).

In an interview, Dr. Conaghan explained that the issue has arisen with emerging agents that are designed for structural improvements with the expectation that symptom improvements will follow. Recapping a presentation he made at the European Congress of Rheumatology, he cautioned that the key aspects of trial design for these novel agents, including patient and endpoint selection, are particularly challenging.

As an example, Dr. Conaghan referred to the experience so far with the ongoing phase 2 FORWARD trial with sprifermin, a recombinant form of human fibroblast growth factor. In this study, sprifermin has already shown promise for growing cartilage, but the benefit accrues slowly, and there is no symptomatic improvement early in the course of treatment.


Based on the experience with FORWARD, much has been learned about a potential tension between structural and symptomatic endpoints in osteoarthritis, according to Dr. Conaghan. For one, it appears to be important to select patients most likely to achieve measurable structural improvements quickly to achieve a positive result in a reasonable period of time.

For another, it may be necessary to select symptom endpoints that reflect structural change while cautioning patients about the potential for a long delay before a clinical benefit is experienced.

In osteoarthritis, clinical benefit has been traditionally captured with relief of pain. Although an improvement in joint structure might be the best way to produce this result, this has to be proved. Reasonable and achievable endpoints are needed for emerging drugs with the potential to rebuild the joint not just to control pain, he said.

SOURCE: Gühring H et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):70-1. Abstract OP0010. doi: 10.1136/annrheumdis-2019-eular.1216.

MADRID – Treating patients with axial spondyloarthritis (axSpA) until a specific target is reached is an emerging concept that has gained a lot of traction in the past few years, Pedro Machado, MD, said at the European Congress of Rheumatology.

Vidyard Video

“The availability of biologic therapies has improved the clinical outcomes for our patients with axial spondyloarthritis and targeting clinical remission or inactive disease is now an achievable treatment goal in clinical practice,” he observed. “This has trigged the question: Is there a role for ‘treat-to-target’ in axial spondyloarthritis?”

Dr. Machado, an honorary consultant in rheumatology and muscle diseases at University College Hospital and the National Hospital for Neurology and Neurosurgery in London, took a critical look at the treat-to-target approach during a clinical science session at the meeting, organized by the European League Against Rheumatism (EULAR).

The concept of treat-to-target is not new, he acknowledged, having been imported from other chronic conditions where there is a very specific target to achieve – such as lowering glycated hemoglobin in diabetes or hypertension or hyperlipidemia in cardiovascular disease.

“The concept involves changing or escalating therapy according to a predefined target under the assumption that this may lead to a better outcome compared to what we call ‘routine care,’ ” Dr. Machado explained.

Treat-to-target is not only well established in nonrheumatic diseases but also has proved to work in patients with rheumatoid arthritis and psoriatic arthritis with evidence from the TICORA (Tight Control of Rheumatoid Arthritis) and TICOPA (Tight Control in Psoriatic Arthritis) trials.

Whether the approach can also work in axSpA is open to debate, and one of the main arguments against using a treat-to-target in axSpA asks, what exactly is the target? While there is no firm agreement yet, Dr. Machado observed that achieving either clinical remission or inactive disease would be the most likely target.

It could be argued this is already being done to some degree, but “we need to be more ambitious,” Dr. Machado said. Indeed, current Assessment of Spondyloarthritis International Society/EULAR recommendations for the treatment of axSpA (Ann Rheum Dis. 2017;76[6]:978–91) note when patients with high disease activity despite sufficient standard treatment should be escalated to treatment with a biologic disease-modifying antirheumatic drug (bDMARD). High disease activity was defined as an Ankylosing Spondylitis Disease Activity Score (ASDAS) of 2.1 or more or a Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) score of 4 or more.

Another argument against using the approach concerns the evidence base. There are no prospective, randomized trials supporting the use of treat-to-target over routine care. However, there is a lot of observational evidence, Dr. Machado said in an interview. Such studies have shown that achieving inactive disease may improve structural outcomes and stop the development of radiographic damage of the spine. Importantly, these observational studies also show that achieving inactive disease may also help to improve patients’ functional outcomes and quality of life.

Evidence backing a treat-to-target approach in axSpA from a randomized, controlled trial may currently be lacking, but the TiCOSPA (Tight Control in Spondyloarthritis) trial is in progress and should help change that, Dr. Machado said.

“The missing bit is a randomized trial, but I would say that the observational evidence is almost enough to advocate a treat-to-target strategy in axial spondyloarthritis.” This was also the view of an international task force that recently published recommendations and overarching principles for a treat-target strategy in spondyloarthritis, including axSpA (Ann Rheum Dis. 2018;77:3-17).

Of course, a treat-to-target approach may not be without its pitfalls. There are a limited number of drugs currently that could be used to “hit the target” of disease activity, Dr. Machado said in his presentation. The approach might also lead to ‘overtreatment,’ and more treatment is not always better as it could not only lead to more adverse events, but it also may mean the approach is not cost-effective.

Depending on the TiCOSPA study results, which are expected next year, Dr. Machado said that “the feasibility and cost-effectiveness of such a strategy in clinical practice also needs to be tested.”

MADRID – Treating patients with axial spondyloarthritis (axSpA) until a specific target is reached is an emerging concept that has gained a lot of traction in the past few years, Pedro Machado, MD, said at the European Congress of Rheumatology.

Vidyard Video

“The availability of biologic therapies has improved the clinical outcomes for our patients with axial spondyloarthritis and targeting clinical remission or inactive disease is now an achievable treatment goal in clinical practice,” he observed. “This has trigged the question: Is there a role for ‘treat-to-target’ in axial spondyloarthritis?”

Dr. Machado, an honorary consultant in rheumatology and muscle diseases at University College Hospital and the National Hospital for Neurology and Neurosurgery in London, took a critical look at the treat-to-target approach during a clinical science session at the meeting, organized by the European League Against Rheumatism (EULAR).

The concept of treat-to-target is not new, he acknowledged, having been imported from other chronic conditions where there is a very specific target to achieve – such as lowering glycated hemoglobin in diabetes or hypertension or hyperlipidemia in cardiovascular disease.

“The concept involves changing or escalating therapy according to a predefined target under the assumption that this may lead to a better outcome compared to what we call ‘routine care,’ ” Dr. Machado explained.

Treat-to-target is not only well established in nonrheumatic diseases but also has proved to work in patients with rheumatoid arthritis and psoriatic arthritis with evidence from the TICORA (Tight Control of Rheumatoid Arthritis) and TICOPA (Tight Control in Psoriatic Arthritis) trials.

Whether the approach can also work in axSpA is open to debate, and one of the main arguments against using a treat-to-target in axSpA asks, what exactly is the target? While there is no firm agreement yet, Dr. Machado observed that achieving either clinical remission or inactive disease would be the most likely target.

It could be argued this is already being done to some degree, but “we need to be more ambitious,” Dr. Machado said. Indeed, current Assessment of Spondyloarthritis International Society/EULAR recommendations for the treatment of axSpA (Ann Rheum Dis. 2017;76[6]:978–91) note when patients with high disease activity despite sufficient standard treatment should be escalated to treatment with a biologic disease-modifying antirheumatic drug (bDMARD). High disease activity was defined as an Ankylosing Spondylitis Disease Activity Score (ASDAS) of 2.1 or more or a Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) score of 4 or more.

Another argument against using the approach concerns the evidence base. There are no prospective, randomized trials supporting the use of treat-to-target over routine care. However, there is a lot of observational evidence, Dr. Machado said in an interview. Such studies have shown that achieving inactive disease may improve structural outcomes and stop the development of radiographic damage of the spine. Importantly, these observational studies also show that achieving inactive disease may also help to improve patients’ functional outcomes and quality of life.

Evidence backing a treat-to-target approach in axSpA from a randomized, controlled trial may currently be lacking, but the TiCOSPA (Tight Control in Spondyloarthritis) trial is in progress and should help change that, Dr. Machado said.

“The missing bit is a randomized trial, but I would say that the observational evidence is almost enough to advocate a treat-to-target strategy in axial spondyloarthritis.” This was also the view of an international task force that recently published recommendations and overarching principles for a treat-target strategy in spondyloarthritis, including axSpA (Ann Rheum Dis. 2018;77:3-17).

Of course, a treat-to-target approach may not be without its pitfalls. There are a limited number of drugs currently that could be used to “hit the target” of disease activity, Dr. Machado said in his presentation. The approach might also lead to ‘overtreatment,’ and more treatment is not always better as it could not only lead to more adverse events, but it also may mean the approach is not cost-effective.

Depending on the TiCOSPA study results, which are expected next year, Dr. Machado said that “the feasibility and cost-effectiveness of such a strategy in clinical practice also needs to be tested.”

MADRID – Treating patients with axial spondyloarthritis (axSpA) until a specific target is reached is an emerging concept that has gained a lot of traction in the past few years, Pedro Machado, MD, said at the European Congress of Rheumatology.

Vidyard Video

“The availability of biologic therapies has improved the clinical outcomes for our patients with axial spondyloarthritis and targeting clinical remission or inactive disease is now an achievable treatment goal in clinical practice,” he observed. “This has trigged the question: Is there a role for ‘treat-to-target’ in axial spondyloarthritis?”

Dr. Machado, an honorary consultant in rheumatology and muscle diseases at University College Hospital and the National Hospital for Neurology and Neurosurgery in London, took a critical look at the treat-to-target approach during a clinical science session at the meeting, organized by the European League Against Rheumatism (EULAR).

The concept of treat-to-target is not new, he acknowledged, having been imported from other chronic conditions where there is a very specific target to achieve – such as lowering glycated hemoglobin in diabetes or hypertension or hyperlipidemia in cardiovascular disease.

“The concept involves changing or escalating therapy according to a predefined target under the assumption that this may lead to a better outcome compared to what we call ‘routine care,’ ” Dr. Machado explained.

Treat-to-target is not only well established in nonrheumatic diseases but also has proved to work in patients with rheumatoid arthritis and psoriatic arthritis with evidence from the TICORA (Tight Control of Rheumatoid Arthritis) and TICOPA (Tight Control in Psoriatic Arthritis) trials.

Whether the approach can also work in axSpA is open to debate, and one of the main arguments against using a treat-to-target in axSpA asks, what exactly is the target? While there is no firm agreement yet, Dr. Machado observed that achieving either clinical remission or inactive disease would be the most likely target.

It could be argued this is already being done to some degree, but “we need to be more ambitious,” Dr. Machado said. Indeed, current Assessment of Spondyloarthritis International Society/EULAR recommendations for the treatment of axSpA (Ann Rheum Dis. 2017;76[6]:978–91) note when patients with high disease activity despite sufficient standard treatment should be escalated to treatment with a biologic disease-modifying antirheumatic drug (bDMARD). High disease activity was defined as an Ankylosing Spondylitis Disease Activity Score (ASDAS) of 2.1 or more or a Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) score of 4 or more.

Another argument against using the approach concerns the evidence base. There are no prospective, randomized trials supporting the use of treat-to-target over routine care. However, there is a lot of observational evidence, Dr. Machado said in an interview. Such studies have shown that achieving inactive disease may improve structural outcomes and stop the development of radiographic damage of the spine. Importantly, these observational studies also show that achieving inactive disease may also help to improve patients’ functional outcomes and quality of life.

Evidence backing a treat-to-target approach in axSpA from a randomized, controlled trial may currently be lacking, but the TiCOSPA (Tight Control in Spondyloarthritis) trial is in progress and should help change that, Dr. Machado said.

“The missing bit is a randomized trial, but I would say that the observational evidence is almost enough to advocate a treat-to-target strategy in axial spondyloarthritis.” This was also the view of an international task force that recently published recommendations and overarching principles for a treat-target strategy in spondyloarthritis, including axSpA (Ann Rheum Dis. 2018;77:3-17).

Of course, a treat-to-target approach may not be without its pitfalls. There are a limited number of drugs currently that could be used to “hit the target” of disease activity, Dr. Machado said in his presentation. The approach might also lead to ‘overtreatment,’ and more treatment is not always better as it could not only lead to more adverse events, but it also may mean the approach is not cost-effective.

Depending on the TiCOSPA study results, which are expected next year, Dr. Machado said that “the feasibility and cost-effectiveness of such a strategy in clinical practice also needs to be tested.”