Bone Marrow Failure Disorders Program

Bone marrow failure syndromes are rare diseases in which the body can’t make enough red cells, which carry oxygen; white cells, which fight infection; or platelets, which help the blood clot and stop bleeding. Bone marrow failure disorders may be either inherited or acquired, and understanding their cause can help guide important treatments.

We bring together a coordinated, comprehensive team with deep expertise in hematology, genetics and stem cell transplantation to deliver exceptional care and support for your child. We will provide your child with a rapid, precise diagnosis and the best available treatments for these complex, rare conditions, including:

• Amegakaryocytic thrombocytopenia
• Diamond-Blackfan anemia
• Dyskeratosis congenita
• Fanconi anemia
• Idiopathic aplastic anemia
• Paroxysmal nocturnal hemoglobinuria
• Pearson syndrome
• Severe congenital neutropenia (Kostmann syndrome)
• Shwachman-Diamond anemia
• Thrombocytopenia absent radii

Stanford doctors are long-time pioneers in the field of bone marrow failure. We have extensive experience using the latest genetic and phenotypic tests to give your child an accurate diagnosis. Our coordinated team of hematologists, stem cell transplant physicians, pathologists, geneticists, genetic counselors, care coordinators, nurses and social workers can help your family understand these often confusing disorders and provide you with the best integrated care possible. Our doctors and researchers also conduct research to discover new treatments for bone marrow failure disorders.