Mugishima H, Gale R P, Rowlings P A, Horowitz M M, Marmont A M, McCann S R, Sobocinski K A, Bortin M M
International Bone Marrow Transplant Registry, Health Policy Institute, Medical College of Wisconsin, Milwaukee 53226, USA.
Bone Marrow Transplant. 1995 Jan;15(1):55-8.
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).
