Fanconi anemia (FA) is a rare, inherited blood disorder caused by mutations in any number of at least 14 genes. These mutations make it hard for blood stem cells (and other cells in the body) to detect and repair damage to their DNA, causing the bone marrow to fail.
Children with the disorder may have several physical abnormalities in addition to bone marrow failure and an increased risk of developing cancer. FA is a severe lifelong condition that requires ongoing medical treatment.
How Dana-Farber/Boston Children’s Cancer and Blood Disorders Center approaches Fanconi anemia
Experts in our Bone Marrow Failure Program provide compassionate, patient- and family-centered care to young patients with FA and bone marrow failure syndromes. Our unique services include:
- Two experienced pediatric hematologists, Colin Sieff, MBBCh, and David A. Williams, MD, oversee the Program.
- Our team includes doctors from other departments within the hospital, each of whom is familiar with the implications of FA in their specialty area.
- Our research is focused on increasing our molecular understanding of FA, as well as improvements in diagnosis and treatment.
- We offer multiple clinical trials for FA patients.
- We work as part of one team with the Dana-Farber/Boston Children’s Stem Cell Transplant Center, which is familiar with and has experience performing hematopoietic stem cell transplants as a way of treating Fanconi anemia in children.
“Fanconi anemia is a complex disease. At our multidisciplinary clinic, children are not only seen by hematologists but also can be seen by geneticists, cardiologists, kidney experts and hand surgeons. Essentially, any kind of expertise that's needed, we provide in our clinic”. -David A. Williams, MD, co-director of the Bone Marrow Failure Program