BONE-MARROW TRANSPLANTATION FOR DIAMOND-BLACKFAN ANEMIA

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 siblin g (n = 8), maternal (n = 1) or unrelated (n = 1) donor and reported to the International Bone Marrow Transplant Registry. Among eight recipi ents of HLA-identical sibling transplants, six are alive 5-87 months a fter transplant with no evidence of DBA and with Karnofsky performance scores of 90-100%. The two recipients of non-HLA-identical sibling tr ansplants died < 2 weeks after transplant. The actuarial 2-year probab ility of survival for the eight sibling transplants was 72 (37-92)% (9 5% confidence interval).