Bone marrow transplantation for Diamond-Blackfan anemia

Bone Marrow Transplant. 1995 Jan;15(1):55-8.

Abstract

Diamond-Blackfan anemia (DBA) is a rare disorder usually diagnosed in the first year of life. Although most cases respond to corticosteroids, > 20% are, or become, steroid-resistant. We report 10 children with DBA who received a bone marrow transplant from an HLA-identical sibling (n = 8), maternal (n = 1) or unrelated (n = 1) donor and reported to the International Bone Marrow Transplant Registry. Among eight recipients of HLA-identical sibling transplants, six are alive 5-87 months after transplant with no evidence of DBA and with Karnofsky performance scores of 90-100%. The two recipients of non-HLA-identical sibling transplants died < 2 weeks after transplant. The actuarial 2-year probability of survival for the eight sibling transplants was 72 (37-92)% (95% confidence interval).

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Fanconi Anemia / mortality
  • Fanconi Anemia / therapy*
  • Female
  • Histocompatibility
  • Humans
  • Infant
  • Male
  • Retrospective Studies
  • Survival Rate