• Research & Innovation

    You can say our faculty 'wrote the book' on pediatric blood disorders. Because of our long track record of innovation, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center is considered a world leader in laboratory and clinical research on blood disorders. We discovered two white blood cell disorders, Shwachman-Diamond syndrome and Diamond-Blackfan syndrome, and we developed the first test to detect prenatal thalassemia. We are a world leader in research on platelet disorders and immune thrombocytopenia purpura.

    Members of our Blood Disorders Center: 

    • Demonstrated effective iron chelation (the removal of excess iron from the blood) using the intravenous drug deferoxamine
    • Recognized the important role of hypertransfusion to treat thalassemia 
    • Discovered a gene for a rare form of inherited iron deficiency
    • Established the use of hydroxyurea as an effective treatment for sickle cell disease 
    • Discovered a gene that plays a central role in the transition from fetal hemoglobin to adult hemoglobin at birth and which has the potential to revolutionize treatment of hemoglobinopathies.

    Currently, our Blood Disorders Center is investigating the possible use of genetic therapy to cure sickle cell disease, anemia, hemophilia, and other conditions, studying the molecular basis of Fanconi anemia, and conducting research to improve the quality of life of children with blood disorders. Learn how tomorrow's breakthroughs are being developed today through our ongoing clot disorder clinical trials.

  • Contact Us

    Our specialized new patient coordinators can answer your questions about treatment options and becoming a patient.
  • Clinical Trials

    Through clinical trials and research, our Blood Disorders Center is continuously advancing the treatment of blood disorders in children. callout bg
  • Ranked #1 for Cancer Care

    US News

    Rated the top pediatric
    cancer center by U.S. News & World Report