Sickle cell disease: What to watch at ASH

Sickle cell disease will take center stage at the annual meeting of the American Society of Hematology.

Top studies to be featured at ASH 2018 include the first in-human presentation of a new approach to gene therapy in sickle cell disease, long-term outcomes of haploidentical transplants, mortality rates in patients prescribed opioids, and aspects of care in lower-resource countries, according to Alexis A. Thompson, MD, the current ASH president.

“These are all quite different aspects of work being done,” said Dr. Thompson, associate director of equity and minority health at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University in Chicago.

In the gene therapy study (abstract 1023), investigators will report initial results of genetic targeting of the fetal-to-adult globin switch in sickle cell patients. The clinical pilot study involves autologous gene therapy utilizing microRNA-adapted shRNAs (shRNA^miR) lentiviral vector targeting BCL11A, a repressor of gamma globin expression, according to the investigators.

“We’re looking forward to seeing this initial presentation on their findings with this first administration to humans,” Dr. Thompson said in a media briefing.

In another study (abstract 162), investigators will report long-term results of familial haploidentical stem cell transplantation (HISCT) showing that high-risk sickle cell patients had significantly improved health-related quality of life in long-term follow-up.

Two years after myeloablative conditioning and familial HISCT using CD34 enrichment and mononuclear cell (CD3) addback, recipients had significant improvements in emotional and physical health-related quality of life, among other outcomes of interest, including neurocognitive outcomes.

Another study, which is interesting in the context of the national opioid epidemic, Dr. Thompson said, will show that opioid use was not associated with in-hospital mortality in patients with sickle cell disease (abstract 315).

Looking at data from the National Inpatient Sample, there was no increase in in-hospital mortality in sickle cell patients versus the general population since the onset of the epidemic, which has been documented since 2000, according to the investigators.

“It should alleviate many concerns about patients with sickle cell who, for legitimate reasons, may require high doses of opioids,” Dr. Thompson said. “While I think we’re being very mindful about opioid use in this patient population, they certainly do need these high doses, but there does not seem to be an extraordinary risk of death associated with their use.”

One final study worth watching, according to Dr. Thompson, is REACH, a prospective, multinational trial of hydroxyurea for sickle cell anemia in sub-Saharan Africa (abstract 3).

The study, which included 635 children, provides the first prospective data showing the feasibility, safety, and benefits of hydroxyurea treatment for children with sickle cell anemia in sub-Saharan Africa, according to the investigators.

The findings are “quite encouraging,” said Dr. Thompson, adding that the results looked “very similar” to the United States experience and demonstrated effective clinical trial design and execution in a lower-resource country.

Sickle cell disease will take center stage at the annual meeting of the American Society of Hematology.

Top studies to be featured at ASH 2018 include the first in-human presentation of a new approach to gene therapy in sickle cell disease, long-term outcomes of haploidentical transplants, mortality rates in patients prescribed opioids, and aspects of care in lower-resource countries, according to Alexis A. Thompson, MD, the current ASH president.

“These are all quite different aspects of work being done,” said Dr. Thompson, associate director of equity and minority health at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University in Chicago.

In the gene therapy study (abstract 1023), investigators will report initial results of genetic targeting of the fetal-to-adult globin switch in sickle cell patients. The clinical pilot study involves autologous gene therapy utilizing microRNA-adapted shRNAs (shRNA^miR) lentiviral vector targeting BCL11A, a repressor of gamma globin expression, according to the investigators.

“We’re looking forward to seeing this initial presentation on their findings with this first administration to humans,” Dr. Thompson said in a media briefing.

In another study (abstract 162), investigators will report long-term results of familial haploidentical stem cell transplantation (HISCT) showing that high-risk sickle cell patients had significantly improved health-related quality of life in long-term follow-up.

Two years after myeloablative conditioning and familial HISCT using CD34 enrichment and mononuclear cell (CD3) addback, recipients had significant improvements in emotional and physical health-related quality of life, among other outcomes of interest, including neurocognitive outcomes.

Another study, which is interesting in the context of the national opioid epidemic, Dr. Thompson said, will show that opioid use was not associated with in-hospital mortality in patients with sickle cell disease (abstract 315).

Looking at data from the National Inpatient Sample, there was no increase in in-hospital mortality in sickle cell patients versus the general population since the onset of the epidemic, which has been documented since 2000, according to the investigators.

“It should alleviate many concerns about patients with sickle cell who, for legitimate reasons, may require high doses of opioids,” Dr. Thompson said. “While I think we’re being very mindful about opioid use in this patient population, they certainly do need these high doses, but there does not seem to be an extraordinary risk of death associated with their use.”

One final study worth watching, according to Dr. Thompson, is REACH, a prospective, multinational trial of hydroxyurea for sickle cell anemia in sub-Saharan Africa (abstract 3).

The study, which included 635 children, provides the first prospective data showing the feasibility, safety, and benefits of hydroxyurea treatment for children with sickle cell anemia in sub-Saharan Africa, according to the investigators.

The findings are “quite encouraging,” said Dr. Thompson, adding that the results looked “very similar” to the United States experience and demonstrated effective clinical trial design and execution in a lower-resource country.

Sickle cell disease will take center stage at the annual meeting of the American Society of Hematology.

Top studies to be featured at ASH 2018 include the first in-human presentation of a new approach to gene therapy in sickle cell disease, long-term outcomes of haploidentical transplants, mortality rates in patients prescribed opioids, and aspects of care in lower-resource countries, according to Alexis A. Thompson, MD, the current ASH president.

“These are all quite different aspects of work being done,” said Dr. Thompson, associate director of equity and minority health at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University in Chicago.

In the gene therapy study (abstract 1023), investigators will report initial results of genetic targeting of the fetal-to-adult globin switch in sickle cell patients. The clinical pilot study involves autologous gene therapy utilizing microRNA-adapted shRNAs (shRNA^miR) lentiviral vector targeting BCL11A, a repressor of gamma globin expression, according to the investigators.

“We’re looking forward to seeing this initial presentation on their findings with this first administration to humans,” Dr. Thompson said in a media briefing.

In another study (abstract 162), investigators will report long-term results of familial haploidentical stem cell transplantation (HISCT) showing that high-risk sickle cell patients had significantly improved health-related quality of life in long-term follow-up.

Two years after myeloablative conditioning and familial HISCT using CD34 enrichment and mononuclear cell (CD3) addback, recipients had significant improvements in emotional and physical health-related quality of life, among other outcomes of interest, including neurocognitive outcomes.

Another study, which is interesting in the context of the national opioid epidemic, Dr. Thompson said, will show that opioid use was not associated with in-hospital mortality in patients with sickle cell disease (abstract 315).

Looking at data from the National Inpatient Sample, there was no increase in in-hospital mortality in sickle cell patients versus the general population since the onset of the epidemic, which has been documented since 2000, according to the investigators.

“It should alleviate many concerns about patients with sickle cell who, for legitimate reasons, may require high doses of opioids,” Dr. Thompson said. “While I think we’re being very mindful about opioid use in this patient population, they certainly do need these high doses, but there does not seem to be an extraordinary risk of death associated with their use.”

One final study worth watching, according to Dr. Thompson, is REACH, a prospective, multinational trial of hydroxyurea for sickle cell anemia in sub-Saharan Africa (abstract 3).

The study, which included 635 children, provides the first prospective data showing the feasibility, safety, and benefits of hydroxyurea treatment for children with sickle cell anemia in sub-Saharan Africa, according to the investigators.

The findings are “quite encouraging,” said Dr. Thompson, adding that the results looked “very similar” to the United States experience and demonstrated effective clinical trial design and execution in a lower-resource country.