Sickle Cell Program

Our innovative approach

Researchers at Dana-Farber/Children's Hospital Cancer Center conduct clinical and laboratory research to increase and enhance knowledge of sickle cell disorders and their treatment. Keeping abreast of current research, its application to clinical care and advising families and referring physicians about available clinical trials are high priorities as well.

We are a part of the Boston Comprehensive Sickle Cell Center, one of ten previously National Institute of Health-funded centers in the United States, and a site for the Sickle Cell Disease Clinical Research Network. Regionally, we are also a member of the New England Pediatric Sickle Cell Consortium, and recently led a project aimed at improving newborn screen interpretation by primary care pediatricians.

Through these affiliations and independent projects, our specialists offer clinical trials investigating the efficacy of new medications and therapies to treat sickle cell disease. Some current clinical research initiatives include:

• Hydroxyurea therapy - Many of our patients participated in early clinical research programs related to the use of hydroxyurea, including the HUG-Kids trial. Our sickle cell clinicians are experienced in the use of hydroxyurea in selected patients for the prevention of acute and chronic complications. This medication has changed the landscape for patients and families affected sickle cell disease.
• Oral iron chlelator - Sickle cell anemia patients who require chronic red blood cell transfusion to prevent stroke gradually become iron overloaded. Without removal of this iron by through a process known as chelation, it can become toxic to vital organs. In the past, chelation required medication to be administered by a needle nightly. we recently completed an exciting trial of a new oral chelator called deferasirox.
• Stem cell transplantation - Through Dana-Farber/Children's Hospital Cancer Center, sickle cell disease patients have access to one of the largest pediatric transplantation programs in the United States. The program is actively pursuing protocols for transplantation of hematological diagnoses. There is interest in the use of reduced intensity conditioning and minimizing the occurrence graft-versus-host disease, two potentially life-threatening complications of transplantation.
  
  Click here to learn more about stem cell transplantation and stem cell research.

Our Sickle Cell Program is currently conducting a study to gather medical history and quality of life information from a large number of sickle cell patients treated here. We hope this information will lead to greater insight into the disease and help us to improve quality of life for sickle cell patients.

Our researchers Stuart Orkin, MD, and Vijay Sankaran identified a gene which may help scientists develop new therapies for sickle cell disease. Click here to read more about their innovative research.