Comprehensive Bone Marrow Failure Center

The Comprehensive Bone Marrow Failure Center (CBMFC) at Children’s Hospital of Philadelphia serves patients with inherited or acquired bone marrow failure (BMF). It provides comprehensive consultation and treatment across a range of BMF disorders. The Center brings together CHOP experts from all relevant pediatric subspecialties and is closely affiliated with colleagues at the Hospital of the University of Pennsylvania caring for adult patients with BMF. The team has extensive experience in caring for patients with aplastic anemia (acquired and familial), Diamond-Blackfan anemia, dyskeratosis congenital, Fanconi anemia, myelodysplastic syndrome, paroxysmal nocturnal hemoglobinuria, severe congenital neutropenia, Shwachman Diamond syndrome, and other inherited BMF syndromes.

Research Project Highlights

Researchers at the Comprehensive Bone Marrow Failure Center are currently engaged in the following efforts:

• Kurre Laboratory: Understanding the cause of stem cell losses in Fanconi Anemia (FA); the role of FA proteins during hematopoietic development; and the role of cell-derived extracellular vesicles in stem cell regulation.
• Olson Laboratory: The role of germline genetic predisposition syndromes in the development of pediatric myelodysplastic syndrome and acute myeloid leukemia; patterns of HLA allele expression and clonal evolution in acquired aplastic anemia; investigation of how intrinsic pathways governing hematopoietic stem cell niche function can be harnessed to improve the efficiency and durability of donor engraftment after hematopoietic stem cell transplantation; clinical trials of novel conditioning and T cell depletion strategies to improve survival and decrease allo-immune complications for patients with non-malignant hematologic disorders undergoing hematopoietic stem cell transplantation.  
• Babushok Laboratory: Clonal evolution in acquired and inherited BMF syndromes; mechanisms of autoimmunity in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria.