Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children.
We describe long-term disease-free survival (DFS) after unrelated donor bone marrow transplantation (BMT) for myelodysplastic syndrome (MDS) in 118 patients aged ≤18 years. Forty-six patients had refractory cytopenia (RC), 55 refractory anemia with excess blasts (RAEB), and 17 refractory anemia with excess blasts in transformation (RAEB-t). Transplant-related mortality was higher after mismatched BMT (relative risk [RR] 3.29, P = .002). Disease recurrence was more likely with advanced stages of MDS at the time of BMT: RAEB (RR 6.50, P = .01) or RAEB-t (RR 11.00, P = .004). Treatment failure (recurrent disease or death from any cause; inverse of DFS) occurred in 68 patients. Treatment failure was higher after mismatched BMT (RR 2.79, P = .001) and in those with RAEB-t (RR 2.38, P = .02). Secondary MDS or chemotherapy prior to BMT was not associated with recurrence or treatment failure. Similarly, cytogenetic abnormalities were not associated with transplant outcomes. Eight-year DFS for patients with RC after matched and mismatched unrelated donor BMT was 65% and 40%, respectively. Corresponding DFS for patients with RAEB and RAEB-t was 48% and 28%, respectively. When a matched adult unrelated donor is available, BMT should be offered as first-line therapy, and children with RC can be expected to have the best outcome.
Woodard
Paul
P
Amgen, Thousand Oaks, California, USA.
Carpenter
Paul A
PA
Davies
Stella M
SM
Gross
Thomas G
TG
He
Wensheng
W
Zhang
Mei-Jie
MJ
Horn
Biljana N
BN
Margolis
David A
DA
Perentesis
John P
JP
Sanders
Jean E
JE
Schultz
Kirk R
KR
Seber
Adriana
A
Woods
William G
WG
Eapen
Mary
M
D000293:Adolescent; D000970:Antineoplastic Agents / Q000008:administration & dosage; D001792:Blood Platelets / Q000166:cytology; D016026:Bone Marrow Transplantation / Q000009:adverse effects / Q000276:immunology / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000503:physiopathology; D006680:HLA Antigens / Q000276:immunology; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000276:immunology / Q000401:mortality / Q000503:physiopathology / Q000628:therapy*; D009504:Neutrophils / Q000166:cytology; D012008:Recurrence; D014019:Tissue Donors; D014184:Transplantation, Homologous; D017211:Treatment Failure
D000970:Antineoplastic Agents; D006680:HLA Antigens
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