Bone marrow transplantation for inherited bone marrow failure syndromes

In 1968, bone marrow transplantation (BMT) was shown to provide an approach to the cure of severe combined immune deficiencies(1) and Wiskott-Aldrich syndrome.(2) The effectiveness of marrow transplantation for treatment of inherited marrow failure states was first demonstrated in 1976 in patients with Diamond-Blackfan syndrome(3) and more recently in those with Fanconi's anemia,(4) Kostmann's syndrome,(5) and osteopetrosis.(6) In this article, we will review approaches to the use of BMT as well as the results of studies in treating patients with inherited bone marrow failure syndromes and osteopetrosis. Where possible, we will emphasize the results after the use of mismatched or unrelated marrow transplantations.