• Age, overall health, and medical history
• Tolerance for therapeutic medications, procedures, and therapies
• Expectations for the course of the disorder

Treatment options may include:

• Drug therapy - depending on the type and cause of marrow failure, medical therapy may include corticosteroids, androgens, and hematopoietic growth factors.

• Hematopoietic stem cell transplant (HSCT) - although not appropriate or feasible for all patients, this therapy can provide the only cure for some forms of marrow failure. The procedure involves replacing diseased bone marrow with healthy marrow from another person (usually a close relative). Bone marrow transplants are one form of hematopoietic stem cell transplant (HSTC). HSCT is a very complex treatment, and not all children with bone marrow failure are candidates for this therapy. This treatment requires a genetically-matched donor and carries the typical risks associated with stem cell transplantation.

Treatment options involving HSCT are evaluated in collaboration with the Dana-Farber/Children's Hospital Cancer Care Pediatric Stem Cell Transplantation Program. Its stem cell transplantation team, which includes pediatric specialists from Dana-Farber Cancer Institute and Children's Hospital Boston, performs approximately 80 transplants each year.

Stem cell transplantations are performed in the 13-bed pediatric stem cell transplantation unit at Children's Hospital Boston, while new patient, consultative services and post-transplant care appointments take place at Dana-Farber Cancer Institute's Jimmy Fund Clinic.