Bone Marrow Failure and Myelodysplastic Syndrome Program

Why choose the Bone Marrow Failure and MDS Program?

The Dana Farber/Boston Children's Bone Marrow Failure and MDS Program offers one of the largest and most renowned pediatric bone marrow failure and MDS programs in the world. Our team provides expert care through leading-edge diagnosis and specializes in developing patient-centric treatment plans.

Tailored medical management and treatment

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 Pediatric 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 Boston Children’s Precision Medicine Service 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.

Specialized team

Because bone marrow failure and MDS affect organs throughout the body, we have a specialized team of experts in endocrinology, cardiology, gastroenterology, immunology, 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 conditions.

Expertise in hematopoietic stem cell transplant

Many children with bone marrow failure or MDS need a hematopoietic stem cell transplant to treat their disease. We have one of the largest and most experienced pediatric stem cell transplant programs in the U.S.

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, RUNX1, MDS and other bone marrow failure conditions.

National partnerships

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:

• North American Pediatric Aplastic Anemia Consortium (NAPAAC)
• Pediatric MDS/BMF Registry
• Shwachman-Diamond Syndrome (SDS) Registry

Seamless transition to adult care

We work in partnership with the Adult Bone Marrow Failure Program at Dana-Farber Cancer Institute. As your child enters adulthood, they will be able to transition care to this team of adult physicians with expertise in these conditions.

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:

• stem cell transplant
• immunosuppressive drug therapy
• blood transfusions and iron chelation therapy
• androgen therapy
• corticosteroid therapy
• growth factor therapy