Analyzing Treatment Options for Pediatric Sickle Cell Disease
The pilot trial began recruiting participants in January 2015, to assess the safety and toxicity of reduced-intensity conditioning haploidentical hematopoietic stem cell transplantation (HSCT) in children with sickle cell disease and thalassemia. HSCT is used to treat hematologic malignancies, bone marrow disorders, and genetic blood disorders. It is currently only a viable curative option for sickle cell disease and thalassemia if the patient has a sibling with an identical human leukocyte antigen (HLA)—the chances of which are only around 15 percent.
“Most providers reserve transplantation as a last resort when other medical therapies fail,” says Allistair Abraham, MD, Director, Sickle Cell Transplant Program at Children’s National.
Dr. Abraham is the lead researcher for the trial, which will test HSCT using a haploidentical donor—a sibling or parent whose HLA is not a perfect match. If this new transplant approach can sustain high curative rates and avoid prohibitively adverse outcomes, it could greatly increase available donors for sickle cell patients who need a transplant.
“Historically, HSCT has been plagued by serious complications,” says David A. Jacobsohn, MD, Division Chief of Blood and Marrow Transplantation at Children’s National. “We hope our findings will help accelerate progress for sickle cell research and change the standard of treatment for future patients.”
The Sickle Cell Disease Program at Children’s National is one of the largest, most comprehensive programs of its kind in the nation, seeing more than 1,400 patients annually. Along with genetic counseling, pain management, and supportive care, Children’s National offers HLA typing and curative treatment for select patients.