• Bone Marrow Failure Treatment Program

    The Bone Marrow Failure Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center is recognized as one of the nation’s best pediatric treatment and research programs for bone marrow failure and related conditions. Our patients have access to advanced treatments and diagnosis, including stem cell transplantation, DNA mutation identification, and ongoing clinical trials that are investigating new treatments.

    Located in Boston, Mass., our Bone Marrow Failure Program has the expertise to diagnose and treat a wide range of disorders, including:

    How We Diagnose & Treat Bone Marrow Failure

    A quick and accurate diagnosis of bone marrow failure means that treatment can begin sooner rather than later. Our pediatric hematologists perform a complete blood test and bone marrow exam to confirm the diagnosis, and other blood and genetic tests to rule out other inherited types of anemia or myelodysplastic syndrome. We also provide DEB testing (also called chromosome breakage testing), the standard, definitive test for Fanconi anemia.

    Depending on the type of bone marrow failure a child has, we offer a range of comprehensive treatments, including:

    • Hematopoietic stem cell transplant 
    • Immunosuppressive drug therapy
    • Blood transfusions and iron chelation therapy
    • Androgen therapy
    • Steroid therapy
    • Growth factor therapy
    • Preventive antibiotic therapy

    Our Treatment Team

    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.

    At the core of our treatment teams are renowned hematologists and bone marrow failure specialists. Many of these physicians are also active researchers, so our patients have access to the most advanced treatments as they become available. From there, we build a team that is best suited to carry out the patient’s treatment plan, including cardiologists, dental surgeons, endocrinologists, gastroenterologists, geneticists, hand surgeons, nephrologists, orthopedists, and otolaryngologists – all with expertise with pediatric bone marrow failure.

    Research & Clinical Trials

    Dana-Farber/Boston Children's has a long history of research and innovation in conditions associated with bone marrow failure. Our clinical research program offers unique access to clinical trials in which children can receive the newest bone marrow failure treatments. A major focus of our research program is the rapid translation of scientific discoveries to the bedside to benefit patients.

    Our physicians and scientists are working on a number of initiatives, including collecting blood and bone marrow from patients to better understand the genetic contributions and molecular aspects of bone marrow failure syndromes; exploring possible genetic factors that put children at risk; collecting blood and bone marrow from patients with Diamond-Blackfan anemia to pinpoint specific proteins that are faulty and how these defects relate to clinical outcomes; and clinical trials to determine if an androgen called danazol is a safe treatment for Fanconi anemia and dyskeratosis congenita. Search for current clinical trials.

  • Contact Us

    Call 1-855-320-2091. We can answer questions about treatment or new patient scheduling.
  • Clinical Trials

    Through clinical trials and research, our Blood Disorders Center is continuously advancing the treatment of blood disorders in children. callout bg
  • Stem Cell Transplant

    Dana-Farber/Boston Children’s has one of the largest and most experienced pediatric stem cell transplant programs in the U.S. Stem cell transplants can provide effective treatment for cancers, blood disorders, and other conditions.