The Pediatric Comprehensive Bone Marrow Failure Center at Children's Hospital of Philadelphia (CHOP) offers complete care for children with complex bone marrow diseases. Our team:

• Excels in caring for children by creating personalized treatment plans
• Diagnoses bone marrow failure conditions using the latest technology
• Partners with adult treatment programs so patients receive lifelong care
• Makes research discoveries that lead to better treatment options

Collaborative care

Our 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 CHOP specialists that may be needed by our patients, including:

• Endocrinology and Diabetes
• Gastroenterology, Hepatology and Nutrition
• Pulmonary and Sleep Medicine
• Otolaryngology (Ear, Nose and Throat)
• Oncology

We also partner with local, national and international colleagues to meet our patients' needs.

Advanced diagnostics and treatments

Bone marrow failure in children is often the result of an undiagnosed genetic condition. We conduct in-depth evaluations of each patient to arrive at a diagnosis that guides treatment and helps us understand if your child needs additional care from other specialists. Key to our success is our collaboration with the Division of Genomic Diagnostics, which performs genome-scale testing to detect the underlying genetic roots of bone marrow failure.

Our highly advanced treatment options include potentially curative therapies like stem cell transplants. We work closely with CHOP's Research Institute to make groundbreaking advances in stem cell gene therapy available to patients quickly.

Ease of scheduling across all specialties

If your child needs to be evaluated at the Pediatric Comprehensive Bone Marrow Failure Center, our nurse care coordinator will ensure your visit goes smoothly. The care coordinator will collect in-depth patient information and help you navigate the scheduling process across specialties. Our physicians will thoroughly review your child's information before your initial visit to determine the most appropriate plan.

If additional specialists will see your child, this can happen during a single visit to make your schedule as streamlined as possible.

Psychosocial support

Our child psychologist works with your child and the whole family to identify and support your emotional and behavioral needs. Through consultations and community education, our psychosocial team can help your family develop healthy strategies to cope with the challenges of pediatric bone marrow failure.

In addition, your family receives ongoing education and support from a licensed clinical social worker who helps navigate issues related to insurance coverage, 504 plans for your child's school and more.

Transition to adult care

For many patients, complex bone marrow failure disorders require lifelong care from multiple specialists. As your child reaches young adulthood, our team helps with the transition into adult care at Penn Medicine. Our partnership with specialists at Penn makes us one of the few centers worldwide to provide comprehensive lifelong care.

Research

Our researchers and physician-scientists actively lead or collaborate on ongoing research projects to discover new treatments for bone marrow failure patients. Some examples of what we are investigating include:

• The biology of inherited neutropenia syndromes
• The biologic basis of bone marrow failure in patients with Fanconi anemia
• The immune system's role in losing hematopoietic stem cells in bone marrow failure
• Gene therapy for Fanconi anemia
• The relationship between aplastic anemia and the outgrowth of myelodysplasia

The CHOP/Penn Bone Marrow Failure Data Registry and Sample Repository are essential resources for our research. Our patients can be a part of research discoveries that drive the next generation of cures. Since its inception, our repository has grown to include data from over 650 unique patients/families and over 10,000 samples that will help us make the subsequent game-changing discovery.