HLA-matched sibling bone marrow transplantation for β-thalassemia major.
We describe outcomes after human leukocyte antigen-matched sibling bone marrow transplantation (BMT) for 179 patients with β-thalassemia major. The median age at transplantation was 7 years and the median follow-up was 6 years. The distribution of Pesaro risk class I, II, and III categories was 2%, 42%, and 36%, respectively. The day 30 cumulative incidence of neutrophil recovery and day 100 platelet recovery were 90% and 86%, respectively. Seventeen patients had graft failure, which was fatal in 11. Six of 9 patients with graft failure are alive after a second transplantation. The day 100 probability of acute graft-versus-host disease and 5-year probability of chronic graft-versus-host disease was 38% and 13%, respectively. The 5-year probabilities of overall- and disease-free survival were 91% and 88%, respectively, for patients with Pesaro risk class II, and 64% and 62%, respectively, for Pesaro risk class III. In multivariate analysis, mortality risks were higher in patients 7 years of age and older and those with hepatomegaly before BMT. The leading causes of death were interstitial pneumonitis (n = 7), hemorrhage (n = 8), and veno-occlusive disease (n = 6). Proceeding to BMT in children younger than 7 years before development of end-organ damage, particularly in the liver, should improve results after BMT for β-thalassemia major.
Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Ontario, Canada.
Mitchell
M
Sabloff
Mammen
M
Chandy
Zhiwei
Z
Wang
Brent R
BR
Logan
Ardeshir
A
Ghavamzadeh
Chi-Kong
CK
Li
Syed Mohammad
SM
Irfan
Christopher N
CN
Bredeson
Morton J
MJ
Cowan
Robert Peter
RP
Gale
Gregory A
GA
Hale
John
J
Horan
Suradej
S
Hongeng
Mary
M
Eapen
Mark C
MC
Walters
D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D005260:Female; D006084:Graft Rejection; D006085:Graft Survival; D006086:Graft vs Host Disease; D006680:HLA Antigens / Q000378:metabolism*; D006801:Humans; D007390:International Agencies; D008297:Male; D035781:Siblings; D015996:Survival Rate; D014019:Tissue Donors; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult; D017086:beta-Thalassemia / Q000276:immunology / Q000628:therapy*
D006680:HLA Antigens