Faculty Collaboration Database

Bone marrow transplantation for Diamond-Blackfan anemia. Bone Marrow Transplant 1995 Jan;15(1):55-8

Date

01/01/1995

Pubmed ID

7742755

Scopus ID

2-s2.0-0028924937 (requires institutional sign-in at Scopus site)   42 Citations

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).

Author List

Mugishima H, Gale RP, Rowlings PA, Horowitz MM, Marmont AM, McCann SR, Sobocinski KA, Bortin MM

Author

Mary M. Horowitz MD, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Bone Marrow Transplantation
Child
Child, Preschool
Fanconi Anemia
Female
Histocompatibility
Humans
Infant
Male
Retrospective Studies
Survival Rate