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Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children. Biol Blood Marrow Transplant 2011 May;17(5):723-8

Date

09/04/2010

Pubmed ID

20813197

Pubmed Central ID

PMC3033968

DOI

10.1016/j.bbmt.2010.08.016

Scopus ID

2-s2.0-79954608298   18 Citations

Abstract

We describe long-term disease-free survival (DFS) after unrelated donor bone marrow transplantation (BMT) for myelodysplastic syndrome (MDS) in 118 patients aged a??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.

Author List

Woodard P, Carpenter PA, Davies SM, Gross TG, He W, Zhang MJ, Horn BN, Margolis DA, Perentesis JP, Sanders JE, Schultz KR, Seber A, Woods WG, Eapen M

Authors

Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin
David A. Margolis MD Interim Chair, Professor in the Pediatrics department at Medical College of Wisconsin
Mei-Jie Zhang PhD Professor in the Institute for Health and Equity department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adolescent
Antineoplastic Agents
Blood Platelets
Bone Marrow Transplantation
Child
Child, Preschool
Disease-Free Survival
Female
Graft vs Host Disease
HLA Antigens
Humans
Male
Myelodysplastic Syndromes
Neutrophils
Recurrence
Tissue Donors
Transplantation, Homologous
Treatment Failure