Children's Hospital Boston 300 Longwood Avenue
Boston, MA 02115
(617) 355-6000
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Clinical Services (Sickle Cell Disease Program):
Innovations & Research
Researchers at Children's Hospital Boston 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.

Children's is a clinical core for the Boston Comprehensive Sickle Cell Center, one of 10 National Institute of Health-funded centers in the United States. It is also a member of the New England Pediatric Sickle Cell Consortium (NEPSCC), and recently led a project aimed at improving newborn screen interpretation by primary care pediatricians, reinforcing the link between newborn screening diagnosis, hematology assessment and genetic counseling.

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

  • Magnesium Therapy - The use of this mineral to improve the hydration of sickle red blood cells in an attempt to decrease sickling and its clinical consequences.

  • Hydroxyurea Therapy - Many Children's patients have participated in early clinical research programs related to the use of hydroxyurea, including the HUG-Kids trial. Children's sickle cell clinicians are experienced in the use of hydroxyurea in selected patients for the prevention of acute and chronic complications.

  • 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 'chelation,' it can become toxic to several vital organs. Current chelation requires medication to be administered by a needle nightly. Children's sickle patients are participting in an exciting trial of a new oral chelator.

  • Stem Cell Transplantation - Through Dana-Farber/Children's Hospital Cancer Care, sickle cell disease patients have access to one of the largest Pediatric Transplantation Programs in the U.S. 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.
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Related Links: Comprehensive Sickle Cell Center
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which can only be given to you by your personal health care professional.
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