Why Choose the Pediatric Comprehensive Bone Marrow Failure Center

The Pediatric Comprehensive Bone Marrow Failure Center (CBMFC) at Children’s Hospital of Philadelphia (CHOP) has a history of excellence in bench-to-bedside clinical care, providing advanced genomic diagnostics and actionable scientific discovery. Collaborating with a diverse team of clinicians from across CHOP, at the University of Pennsylvania (Penn) and around the world, the CBMFC offers highly-individualized, multidisciplinary care to bone marrow failure (BMF) patients throughout childhood and into ongoing adult care.

Ease of Scheduling Across all Specialties

Families who schedule visits with the CBMFC will work with our Nurse Care Coordinator, who will collect in-depth patient information and help navigate the scheduling process across specialties. Our physicians undertake a comprehensive review of all patient information prior to the initial appointment in order to determine the most appropriate plan for multidisciplinary care.

Patients who are seen by additional specialists may schedule with multiple clinicians during each CBMFC appointment.

Psychosocial Support

Our child psychologist works with patients and families to identify and support the emotional and behavioral needs of our patients. Through consultations, in- and/or out-patient appointments, and community education, our psychosocial team works to support the development of healthy strategies to help your family cope with the challenges of pediatric BMF. 

In addition, patients and families receive ongoing education and support from a licensed clinical social worker, who helps navigate issues related to insurance coverage, 504 plans and more.

Transition to Adult Care

For many patients, complex BMF disorders require lifelong multi-specialty care. As these patients reach early adulthood, the CBMFC provides a seamless transition into adult care at the Hospital of the University of Pennsylvania. Our longstanding collaboration with specialists at Penn maximizes continuity of care and eases the transition into adult care. Our community of BMF specialists collaborate in clinical care and research, making us one of the few centers in the world to provide comprehensive lifelong care.

Research

Our researchers and physician-scientists are actively involved in ongoing research projects aimed at discovering new treatments for BMF patients. Our current projects are investigating:

  • The biologic basis of BMF in patients with Fanconi anemia
  • The immune system’s role in the loss of hemopoietic stem cells in BMF
  • Gene therapy for Fanconi anemia
  • The relationship between aplastic anemia and the outgrowth of myelodysplasia

An important resource for our research is the CHOP/Penn Bone Marrow Failure Data Registry and Sample Repository. Our patients have the opportunity to participate in research discoveries that drive the next generation of cures for patients with BMF disorders. Since its inception, our Repository has grown to include over 650 unique patients/families and over 10,000 samples.


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