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 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).
Mugishima
H
H
International Bone Marrow Transplant Registry, Health Policy Institute, Medical College of Wisconsin, Milwaukee 53226, USA.
Gale
R P
RP
Rowlings
P A
PA
Horowitz
M M
MM
Marmont
A M
AM
McCann
S R
SR
Sobocinski
K A
KA
Bortin
M M
MM
D000293:Adolescent; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005199:Fanconi Anemia / Q000401:mortality / Q000628:therapy*; D005260:Female; D006648:Histocompatibility; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D015996:Survival Rate