• Bone Marrow Failure and Myelodysplastic Syndrome Program

    Bone Marrow Failure Program

    The Bone Marrow Failure and Myelodysplastic Syndrome (MDS) Program in the 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, MDS, and related conditions.

    Our patients have access to advanced diagnostic evaluations and treatments, including individually tailored stem cell transplantation and ongoing clinical trials that are investigating new treatments. Our program also has been designated a “Center of Excellence” from the MDS Foundation – the only large pediatric center in the country awarded this designation.

    Our program has the expertise to diagnose and treat a wide range of disorders including:

    We also specialize in the evaluation of undefined bone marrow failure and MDS disorders and offer cutting edge diagnostic testing and research studies.

    Why Choose Us

    Dana-Farber/Boston Children’s offers one of the largest and most renowned pediatric bone marrow failure and MDS programs in the world. Here are a few of the reasons to choose Dana-Farber/Boston Children’s:

    • About 30% of bone marrow failure cases are as yet undiagnosed sub-types. Our team specializes in working with these patients to determine the best forms of treatment – and to continually work toward a diagnosis and cure.
    • Our program offers expertise in individually tailored medical management and treatment of bone marrow failure and MDS. We provide expert genetic evaluation for diagnosis and treatment.
      • Our bone marrow failure experts partner with the Dana-Farber/Boston Children’s Pedatric Cancer Genetic Risk Program to help families assess their risk for complications of bone marrow failure or MDS and to inform medical management. Our genetic counselors have expertise in bone marrow failure and MDS and understand the overlap between bone marrow failure and cancer predisposition syndromes.
      • We also partner with the Dana-Farber/Boston Children’s precision medicine program to provide somatic genetic testing (marrow profiling) to determine specific mutations within your child’s marrow that may be causing the disease. As targeted therapies become available for specific mutations, these therapies could become part of your child’s treatment.
    • Because bone marrow failure and MDS affect organs throughout the body, we have a specialized team of experts in endocrinology, cardiology, gastroenterology, pulmonology, and other specialties who are integral partners within our bone marrow failure/MDS treatment team. These sub-specialists have expertise in treating bone marrow failure syndromes and MDS, and understand the medical complications of these disorders.
    • We have one of the largest and most experienced pediatric stem cell transplant programs in the U.S. Many children with bone marrow failure or MDS need a stem cell transplant to treat their disease.
    • Dana-Farber/Boston Children’s is ranked the #1 pediatric cancer center in the U.S. by U.S. News & World Report. If your child has high-grade MDS, our world-renowned oncologists will be able to assist in your child’s care as needed.
    • We offer a range of clinical trials and research studies that may not be available at other centers. These include studies for aplastic anemia, telomere biology disorders, Fanconi anemia, Shwachman Diamond Syndrome, Diamond Blackfan anemia, MDS, and other bone marrow failures. (Learn more about our research and clinical trials below.)
    • Dana-Farber/Boston Children’s is a leading member of several national consortia focused on research and treatment of bone marrow failures and MDS. These include:
    • Through Dana-Farber and the Brigham and Women’s Hospital, we work in partnership with the adult Bone Marrow Failure Program. As your child enters adulthood, he/she will be able to transition care to this team of adult physicians with expertise in these disorders.
    • Dana-Farber/Boston Children’s offers MMC/DEB testing (also called chromosome breakage testing), the standard, definitive test for Fanconi anemia.

    How We Diagnose and Treat Bone Marrow Failure and MDS

    Early and accurate diagnosis of bone marrow failure or MDS allows optimal medical management and treatment to improve the health of children, adolescents, and young adults with these disorders. Dana-Farber/Boston Children's is one of only a few centers in the country with hematopathologists specifically trained to evaluate these disorders in children.

    Our doctors use a variety of diagnostic tests, including blood tests, bone marrow aspiration and biopsy, chromosomal analysis, and the newest genetic tests available. We also provide MMC/DEB testing (also called chromosome breakage testing), which tests for mitomycin C (MMC) and diepoxybutane (DEB). This is the standard, definitive test for Fanconi anemia, a blood disorder that is characterized by an inability to repair DNA damage caused by agents like MMC and DEB.

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

    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 child.

    At the core of our treatment teams are pediatric hematologists and bone marrow failure specialists, as well as pathologists with specialized training in pediatric hematopathology, pediatric oncologists, geneticists, and nurse practitioners. Many of our physicians are also active researchers, so our patients have access to the very best and up-to-date diagnostic tests and treatments available.

    From there, we build a team that is best suited to carry out your child’s treatment plan. Sub-specialists may include cardiologists, dental surgeons, endocrinologists, gastroenterologists, pulmonologists, immunologists, hand surgeons, nephrologists, orthopedists, and otolaryngologists – all with expertise with pediatric bone marrow failure and MDS.

    Research and Clinical Trials

    Dana-Farber/Boston Children's has a long history of research and innovation in conditions associated with bone marrow failure. Our basic science and clinical research program offers unique access to clinical trials in which children can receive the newest bone marrow failure diagnostic evaluations and treatments.

    Our physicians and scientists are working on a number of initiatives, including collecting blood and bone marrow from patients to better understand the causes of bone marrow failure syndromes and MDS; exploring possible genetic factors that guide medical care; collecting blood and bone marrow to identify specific proteins that are faulty and how these defects relate to clinical complications and outcomes; and clinical trials to improve stem cell transplants for disorders such as dyskeratosis congenita. Another study is testing whether unrelated donor stem cell transplants can provide better long-term outcomes than traditional immunosuppressive therapy (IST) for children with severe aplastic anemia.

    With the support of funding from the National Institutes of Health, we established a nationwide Pediatric Bone Marrow Failure and MDS Registry. This allows researchers to collect clinical information and tissue samples to help better understand and ultimately identify new therapies for these conditions. The registry also offers second opinion pathology consultation as a resource for the hematology community.

    We are also leading members of the North American Pediatric Aplastic Anemia Consortium (NAPAAC) and the Shwachman-Diamond Syndrome (SDS) Registry.

    More about clinical trials
    For many children with rare or hard-to-treat conditions, clinical trials provide new options.

  • Appointments, Referrals, and Second Opinions

    Phone: 1-844-222-9991
    Email: bmf_pedi@dfci.harvard.edu
  • 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 treatment for cancers, blood disorders, and other conditions.