Bone Marrow Failure and Myelodysplastic Syndrome Program | Research & Innovation

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center has a long history of research and innovation associated with bone marrow failure conditions. 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.

Bone Marrow Failure and Myelodysplastic Syndrome Program clinical trials

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