Bone Marrow Failure Program

Led by nationally recognized experts in pediatric hematology, the program manages ongoing treatment plans for children with congenital and acquired bone marrow failure syndromes, including Diamond-Blackfan anemia, Fanconi anemia, Shwachman-Diamond syndrome, idiopathic aplastic anemia and others. Approximately 100 patients are enrolled in the program with referrals coming from across Texas and surrounding states.

Our program is counted among the country’s strongest because of the extent of care we provide. We rely on a comprehensive panel of subspecialists with an express interest in bone marrow failure syndromes. These include endocrinologists, gastroenterologists, orthopedists, transplant physicians, surgeons (hand, oral and maxillofacial), pathologists, neurologists, cardiologists, radiologists and more – each of whom has acquired an expertise in bone marrow failure disorders. 

Ongoing Care

Children and their families affected by these syndromes require years of comprehensive and qualified care, beginning with a clear diagnosis.

• Diagnosis: Diagnoses are made based on clinical criteria and/or genetic testing. 

• Education: At every stage we provide reassuring education to families on all aspects of their child’s disorder, from physical and emotional to psychological and social. 

• Screening for abnormalities: These patients require numerous screenings on an ongoing basis – echocardiograms and abdominal ultrasounds for patients with Fanconi anemia, for example. We manage these with an eye toward reducing the toxicity to which these patients are particularly susceptible (e.g., X-ray). 

• Screening family members: We counsel families on the underlying genetic causes of bone marrow failure syndromes, and offer them carrier testing when appropriate. 

• Coordination of care: We help refer patients to subspecialists at  Children’s who have a particular interest and expertise in these disorders

When Transplant is Required

For children whose bone marrow fails or who develop pre-malignant chromosomal changes, a stem cell transplant may be required. Patients are referred for pre-transplant consultation to stem cell transplant physicians who are also integral members of the Bone Marrow Failure Program team. These patients are cared for by Stem Cell Transplant Program physicians for approximately two years during and after the transplant before returning to the care of hematologists in the Bone Marrow Failure Program. Given the increased risk of future cancers that occur in many bone marrow failure syndromes post-transplant, our team provides disease-specific recommendations for screening of at-risk patients and offers ongoing follow-up throughout childhood and adolescence. 

Program Highlights

• A leading contributor to bone marrow failure disorder registries, including the International Fanconi Anemia Registry, Diamond-Blackfan Registry, Shwachman- Diamond Registry and North American Pediatric Aplastic Anemia Consortium (NAPAAC). 

• Our program regularly partners with the National Institutes of Health on developing and reviewing treatment protocols.