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The "much anticipated" guideline to help primary care and emergency clinicians improve the management of sickle cell disease includes a consensus treatment protocol for implementing hydroxyurea therapy and more detailed guidance regarding long-term transfusion therapy, according to a summary report published online September 9 in Journal of the American Medical Association.
Sickle cell disease (SCD), a life-threatening genetically transmitted disorder affecting 70,000-100,000 Americans, is associated with a wide array of complex acute and chronic complications that require immediate medical attention. But high-quality data on which to base management decisions are sorely lacking, and clinicians get little in the way of guidance from existing recommendations. One result is that "the two most widely available disease-modifying therapies, hydroxyurea and long-term transfusions, are underused, and hematopoietic stem cell transplantation, the only curative approach, has been used in only a small proportion of affected individuals," said Dr. Barbara P. Yawn and her associates on the National Heart, Lung, and Blood Institute expert panel that issued the summary report.
Even this guideline is somewhat rudimentary due to the dearth of good data "in virtually every area related to SCD management," and cannot help but leave "many uncertainties for health professionals caring for individuals with SCD." But it is hoped that this guideline will furnish a critical foundation for future research and will now begin "to facilitate improved and more accessible care for all affected individuals," said Dr. Yawn, director of research at Olmsted Medical Center, Rochester, Minn., and her associates.
The guideline is based on an extensive literature review of more than 13,000 abstracts and articles, which was winnowed to 1,583 original studies regarding SCD. From this, a team of health care professionals in family medicine, internal medicine, pediatric and adult hematology, psychiatry and mental health, transfusion medicine, obstetrics and gynecology, maternal/fetal medicine, and emergency department nursing compiled the guideline as well as the summary, entitled Evidence-Based Management of Sickle Cell Disease: Expert Panel Report 2014 (JAMA 2014 September 9 [doi:10.1001/jama.2014.10517]).
In addition to establishing a protocol for implementing hydroxyurea therapy, the guideline addresses changes in pneumococcal vaccination recommendations for adults and children; annual transcranial Doppler screening coupled with long-term transfusion therapy when necessary to prevent stroke in children aged 2-16 years; rapid initiation of opioids for severe pain during vasoocclusive crises; analgesics and physical therapy for avascular necrosis; ACE inhibitor treatment for adults with microalbuminuria; referral to specialists for screening and treatment of proliferative retinopathy; echocardiography to assess signs of pulmonary hypertension; and monitoring for iron overload in patients receiving transfusion therapy.
Both the summary report and the full guideline are available at http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines/.
Yawn et al. have made a monumental effort to produce practical, evidence-based guidelines, but they were hampered at every turn by a pervasive lack of good quality evidence on which to base their recommendations. Still missing from this guideline are suggestions for how often and when to screen for kidney disease, how to screen for and treat the common clinical problem of asthma-like symptoms (when standard therapies are contraindicated in SCD), how to advocate for patients with the common sequelae of silent cerebral infarcts, or when to consider hematopoietic stem-cell transplantation.
The expert panel also failed to include representatives from the people most affected by SCD: patients and their families. Failure to listen to the perspective of the families, understand which of these recommendations are important to them, and deal with the obstacles families face in implementing the recommendations is a critically important omission.
Dr. Michael R. DeBaun is in the department of pediatrics at the Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Nashville. He made his remarks in an editorial accompanying Dr. Yawn’s report (JAMA 2014:312;1004-5). Dr. DeBaun reported no financial conflicts of interest.
Yawn et al. have made a monumental effort to produce practical, evidence-based guidelines, but they were hampered at every turn by a pervasive lack of good quality evidence on which to base their recommendations. Still missing from this guideline are suggestions for how often and when to screen for kidney disease, how to screen for and treat the common clinical problem of asthma-like symptoms (when standard therapies are contraindicated in SCD), how to advocate for patients with the common sequelae of silent cerebral infarcts, or when to consider hematopoietic stem-cell transplantation.
The expert panel also failed to include representatives from the people most affected by SCD: patients and their families. Failure to listen to the perspective of the families, understand which of these recommendations are important to them, and deal with the obstacles families face in implementing the recommendations is a critically important omission.
Dr. Michael R. DeBaun is in the department of pediatrics at the Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Nashville. He made his remarks in an editorial accompanying Dr. Yawn’s report (JAMA 2014:312;1004-5). Dr. DeBaun reported no financial conflicts of interest.
Yawn et al. have made a monumental effort to produce practical, evidence-based guidelines, but they were hampered at every turn by a pervasive lack of good quality evidence on which to base their recommendations. Still missing from this guideline are suggestions for how often and when to screen for kidney disease, how to screen for and treat the common clinical problem of asthma-like symptoms (when standard therapies are contraindicated in SCD), how to advocate for patients with the common sequelae of silent cerebral infarcts, or when to consider hematopoietic stem-cell transplantation.
The expert panel also failed to include representatives from the people most affected by SCD: patients and their families. Failure to listen to the perspective of the families, understand which of these recommendations are important to them, and deal with the obstacles families face in implementing the recommendations is a critically important omission.
Dr. Michael R. DeBaun is in the department of pediatrics at the Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Nashville. He made his remarks in an editorial accompanying Dr. Yawn’s report (JAMA 2014:312;1004-5). Dr. DeBaun reported no financial conflicts of interest.
The "much anticipated" guideline to help primary care and emergency clinicians improve the management of sickle cell disease includes a consensus treatment protocol for implementing hydroxyurea therapy and more detailed guidance regarding long-term transfusion therapy, according to a summary report published online September 9 in Journal of the American Medical Association.
Sickle cell disease (SCD), a life-threatening genetically transmitted disorder affecting 70,000-100,000 Americans, is associated with a wide array of complex acute and chronic complications that require immediate medical attention. But high-quality data on which to base management decisions are sorely lacking, and clinicians get little in the way of guidance from existing recommendations. One result is that "the two most widely available disease-modifying therapies, hydroxyurea and long-term transfusions, are underused, and hematopoietic stem cell transplantation, the only curative approach, has been used in only a small proportion of affected individuals," said Dr. Barbara P. Yawn and her associates on the National Heart, Lung, and Blood Institute expert panel that issued the summary report.
Even this guideline is somewhat rudimentary due to the dearth of good data "in virtually every area related to SCD management," and cannot help but leave "many uncertainties for health professionals caring for individuals with SCD." But it is hoped that this guideline will furnish a critical foundation for future research and will now begin "to facilitate improved and more accessible care for all affected individuals," said Dr. Yawn, director of research at Olmsted Medical Center, Rochester, Minn., and her associates.
The guideline is based on an extensive literature review of more than 13,000 abstracts and articles, which was winnowed to 1,583 original studies regarding SCD. From this, a team of health care professionals in family medicine, internal medicine, pediatric and adult hematology, psychiatry and mental health, transfusion medicine, obstetrics and gynecology, maternal/fetal medicine, and emergency department nursing compiled the guideline as well as the summary, entitled Evidence-Based Management of Sickle Cell Disease: Expert Panel Report 2014 (JAMA 2014 September 9 [doi:10.1001/jama.2014.10517]).
In addition to establishing a protocol for implementing hydroxyurea therapy, the guideline addresses changes in pneumococcal vaccination recommendations for adults and children; annual transcranial Doppler screening coupled with long-term transfusion therapy when necessary to prevent stroke in children aged 2-16 years; rapid initiation of opioids for severe pain during vasoocclusive crises; analgesics and physical therapy for avascular necrosis; ACE inhibitor treatment for adults with microalbuminuria; referral to specialists for screening and treatment of proliferative retinopathy; echocardiography to assess signs of pulmonary hypertension; and monitoring for iron overload in patients receiving transfusion therapy.
Both the summary report and the full guideline are available at http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines/.
The "much anticipated" guideline to help primary care and emergency clinicians improve the management of sickle cell disease includes a consensus treatment protocol for implementing hydroxyurea therapy and more detailed guidance regarding long-term transfusion therapy, according to a summary report published online September 9 in Journal of the American Medical Association.
Sickle cell disease (SCD), a life-threatening genetically transmitted disorder affecting 70,000-100,000 Americans, is associated with a wide array of complex acute and chronic complications that require immediate medical attention. But high-quality data on which to base management decisions are sorely lacking, and clinicians get little in the way of guidance from existing recommendations. One result is that "the two most widely available disease-modifying therapies, hydroxyurea and long-term transfusions, are underused, and hematopoietic stem cell transplantation, the only curative approach, has been used in only a small proportion of affected individuals," said Dr. Barbara P. Yawn and her associates on the National Heart, Lung, and Blood Institute expert panel that issued the summary report.
Even this guideline is somewhat rudimentary due to the dearth of good data "in virtually every area related to SCD management," and cannot help but leave "many uncertainties for health professionals caring for individuals with SCD." But it is hoped that this guideline will furnish a critical foundation for future research and will now begin "to facilitate improved and more accessible care for all affected individuals," said Dr. Yawn, director of research at Olmsted Medical Center, Rochester, Minn., and her associates.
The guideline is based on an extensive literature review of more than 13,000 abstracts and articles, which was winnowed to 1,583 original studies regarding SCD. From this, a team of health care professionals in family medicine, internal medicine, pediatric and adult hematology, psychiatry and mental health, transfusion medicine, obstetrics and gynecology, maternal/fetal medicine, and emergency department nursing compiled the guideline as well as the summary, entitled Evidence-Based Management of Sickle Cell Disease: Expert Panel Report 2014 (JAMA 2014 September 9 [doi:10.1001/jama.2014.10517]).
In addition to establishing a protocol for implementing hydroxyurea therapy, the guideline addresses changes in pneumococcal vaccination recommendations for adults and children; annual transcranial Doppler screening coupled with long-term transfusion therapy when necessary to prevent stroke in children aged 2-16 years; rapid initiation of opioids for severe pain during vasoocclusive crises; analgesics and physical therapy for avascular necrosis; ACE inhibitor treatment for adults with microalbuminuria; referral to specialists for screening and treatment of proliferative retinopathy; echocardiography to assess signs of pulmonary hypertension; and monitoring for iron overload in patients receiving transfusion therapy.
Both the summary report and the full guideline are available at http://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines/.
FROM JAMA
Major finding: The two most widely available disease-modifying therapies for SCD, hydroxyurea and long-term transfusions, are underused, in large part because no evidence-based treatment protocols have been devised until now.
Data source: A review of the literature and compilation of management guidelines "to assist health care professionals in the management of common issues of sickle cell disease."
Disclosures: The National Heart, Lung, and Blood Institute sponsored the development of this guideline. All expert panel members served voluntarily. Many reported numerous ties to industry sources.
