About the Comprehensive Bone Marrow Failure Center

The Pediatric Comprehensive Bone Marrow Failure Center (CBMFC) at Children’s Hospital of Philadelphia (CHOP) was established in 2010 as a comprehensive, multidisciplinary center dedicated to providing world-class care for children and adults with inherited and acquired forms of bone marrow failure (BMF). The CBMFC at CHOP collaborates with partners at the University of Pennsylvania (Penn) for both clinical and research efforts.

The CBMFC’s commitment to ongoing basic, translational and clinical research bridges the gap from bench to bedside, getting advanced treatments to patients faster. Offering cutting-edge diagnostics, clinical collaboration with experts spanning the globe, and individualized curative therapies, the CBMFC is one of the world’s leading centers for comprehensive, lifelong care of BMF patients.

Collaborative Care

Our CBMFC experts — including physicians, physician-scientists, registered nurses, social workers, genetic counselors, psychologists and laboratory and clinical research staff — have diverse and extensive training backgrounds. Our team works closely with multiple CHOP specialties, including:

  • The Division of Endocrinology and Diabetes
  • The Division of Gastroenterology, Hepatology and Nutrition
  • The Division of Pulmonary Medicine
  • The Division of Otolaryngology (Ear, Nose and Throat)
  • The Cancer Center

We also collaborate with local, national and international colleagues in order to provide our patients the highest-quality care.

Advanced Diagnostics and Treatments

BMF in children is often the consequence of an undiagnosed genetic condition. At the CBMFC, we conduct in-depth evaluations of each patient, with the goal of arriving at a diagnosis that can guide treatment and inform the need for additional care. Key to our success is our longstanding collaboration with the Division of Genomic Diagnostics, which performs genome-scale testing to detect the underlying genetic roots of BMF.

Our physician-scientists have a breadth of experience in basic, translational and clinical research, with active lab projects funded by the National Institutes of Health. We work closely with CHOP’s Research Institute to move groundbreaking advances in stem cell gene therapy to patient-specific clinical care. Our highly individualized treatment plans include potentially curative therapeutics — such as stem cell transplants — as well as ongoing treatment to manage symptoms.


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