• Sickle Cell Disease Treatment Program

    Dr. Heeney with sickle cell disease patient

    Specialists in the Sickle Cell Disease Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center provide diagnosis, treatment and long-term health management for children and young adults with all types of sickle cell disease.

    Located in Boston, Massachusetts, we are an international leader in improving treatment and quality of life for children affected by sickle cell diseases, including:

    • Sickle cell anemia (HbSS)
    • Sickle cell with hemoglobin C disease (HbSC)
    • Hemoglobin S-beta-thalassemia
    • Other, less common compound forms, including HbSD, HbSO and HbSE

    At Dana-Farber/Boston Children's, all patients with sickle cell disease and other serious blood disorders are assigned a social worker to help manage the challenges of ongoing treatments that are often required. Our social workers will provide emotional, psychological and logistical support to patients and families from diagnosis to adulthood.

    How We Diagnose & Treat Sickle Cell Disease

    Early diagnosis is essential for providing proper preventive treatment of potentially devastating complications. Sickle cell disease is typically diagnosed at birth by universal newborn screening programs. Newborn screenings, including hemoglobin electrophoresis, can determine if a child is a carrier of sickle cell trait. A complete family history and additional blood tests may also be required for diagnosis. For diagnosing sickle cell disease in an unborn baby, we can sample amniotic fluid surrounding the baby in the mother’s womb to look for the sickle cell gene.

    Emphasizing a prevention-centered treatment plan, our pediatric specialists work to minimize children’s symptoms and avoid complications. A typical plan may include infection prevention with penicillin until age 5, completion of all routine childhood vaccines and additional sickle cell-specific vaccinations, and maintaining vigilance regarding fevers and possible bacterial infections. We also treat sickle cell disease with hydroxyurea therapy, an oral medication that causes the body to produce fetal hemoglobin, which can significantly reduce side effects and complications; blood transfusions to prevent complications such as stroke and to treat severe anemia; iron chelation to prevent iron toxicity-related to chronic or recurrent blood transfusions; and stem cell transplantation, which can cure the disease but also carries significant risks.

    Our Treatment Teams

    Dana-Farber/Boston Children's patients have access to the broadest set of pediatric and hematologic expertise available. The breadth of our expertise allows us to assemble a team of specialists to meet the specific needs of each patient.

    Our staff includes hematologists with extensive expertise in treating sickle cell disease. We also have mid-level providers—nurse practitioners and physician assistants—who specialize in sickle cell disease. Many of our physicians are also active clinical and basic science researchers, so our patients have access to the very best and up-to-date treatments available.

    Because sickle cell disease can affect nearly every major organ in the body, patients may receive evaluation and care from other specialty physicians at Dana-Farber/Boston Children's including radiologists, pulmonologists, nephrologists, ophthalmologists and surgeons.

    Research & Clinical Trials

    In the setting of one of the world’s most extensive pediatric research hospitals, we apply a science-driven approach to sickle cell disease. Dana-Farber/Boston Children's was one of the original National Institutes of Health-funded comprehensive sickle cell centers in the United States, and a site for the Sickle Cell Disease Clinical Research Network. Our researchers conduct clinical and laboratory studies to increase and enhance knowledge of sickle cell disorders and treatment. Keeping abreast of current basic scientific research, its translation to clinical care, and advising families and referring physicians about available clinical trials are among our highest priorities.

    In the 1980s, our physicians were the first to recognize the potential of hydroxyurea to treat sickle cell disease and to use the medication in children. Today, our researchers continue to work on developing new methods of diagnosing and treating sickle cell disease, including stem cell research, stem cell transplantation, and gene therapy. Search for current sickle cell disease clinical trials.

    Learn More About Sickle Cell Disease in Children

    For in-depth information and videos on sickle cell disease symptoms, treatment, research, and more, visit our sickle cell disease in children overview page.

  • Transfusional Iron Overload

    Treatment of transfusion-dependent anemia can result in iron overload. Our expert annual evaluations can help detect potential concerns.
  • Sickle Cell Clinical Trials

    Our sickle cell disease clinical trials may help reduce sickle cell symptoms.callout bg
  • Patient Number One

    esrick-and-jones-thumbManny shares many loves with his little brother but one thing he wishes they didn't share is sickle cell disease (SCD). By becoming the first patient to participate in a new clinical trial, he's hoping to change that.

  • Stem Cell Transplant

    Many pediatric cancers and blood disorders can be treated through autologous or allogenic stem cell transplants (also called bone marrow transplants). Learn more about conditions treated through stem cell transplantation.