Unrelated Donor Bone Marrow Transplantation for Myelodysplastic Syndrome in Children

Unrelated Donor Bone Marrow Transplantation for Myelodysplastic Syndrome in Children

Author Woodard, Paul Google Scholar
Carpenter, Paul A. Google Scholar
Davies, Stella M. Google Scholar
Gross, Thomas G. Google Scholar
He, Wensheng Google Scholar
Zhang, Mei-Jie Google Scholar
Horn, Biljana N. Google Scholar
Margolis, David A. Google Scholar
Perentesis, John P. Google Scholar
Sanders, Jean E. Google Scholar
Schultz, Kirk R. Google Scholar
Seber, Adriana Autor UNIFESP Google Scholar
Woods, William G. Google Scholar
Eapen, Mary Google Scholar
Institution Med Coll Wisconsin
Amgen Inc
Fred Hutchinson Canc Res Ctr
Cincinnati Childrens Hosp
Nationwide Childrens Hosp
Univ Calif San Francisco
Childrens Hosp Wisconsin
Universidade Federal de São Paulo (UNIFESP)
Aflac Canc Ctr & Blood Disorders Serv
Abstract We describe long-term disease-free survival (DFS) after unrelated donor bone marrow transplantation (BMT) for myelodysplastic syndrome (MDS) in <= 8 patients aged 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. Biol Blood Marrow Transplant 17: 723-728 (2011) (C) 2011 American Society for Blood and Marrow Transplantation
Keywords Pediatric myelodysplastic syndrome
Unrelated donor
Bone marrow transplantation
Language English
Sponsor Public Health Service from National Cancer Institute (NCI)
National Heart, Lung and Blood Institute (NHLBI)
National Institute of Allergy and Infectious Diseases (NIAID)
Health Resources and Services Administration (HRSA/DHHS)
Office of Naval Research
American Society for Blood and Marrow Transplantation
Amgen, Inc.
Astellas Pharma US, Inc.
Baxter International, Inc.
Bayer HealthCare Pharmaceuticals
Be the Match Foundation
Biogen IDEC
BioMarin Pharmaceutical, Inc.
Biovitrum AB
BloodCenter of Wisconsin
Blue Cross and Blue Shield Association
Bone Marrow Foundation
Buchanan Family Foundation
Canadian Blood and Marrow Transplant Group
Celgene Corporation
CellGenix, GmbH
Centers for Disease Control and Prevention
Children's Leukemia Research Association
ClinImmune Labs
CTI Clinical Trial and Consulting Services
Cubist Pharmaceuticals
Cylex Inc.
DOR BioPharma, Inc.
Dynal Biotech, an Invitrogen Company
Eisai, Inc.
Enzon Pharmaceuticals, Inc.
European Group for Blood and Marrow Transplantation
Gamida Cell, Ltd.
GE Healthcare
Genentech, Inc.
Genzyme Corporation
Histogenetics, Inc.
HKS Medical Information Systems
Hospira, Inc.
Infectious Diseases Society of America
Kiadis Pharma
Kirin Brewery Co., Ltd.
The Leukemia & Lymphoma Society
Merck Company
Medical College of Wisconsin
MGI Pharma, Inc.
Michigan Community Blood Centers
Millennium Pharmaceuticals, Inc.
Miller Pharmacal Group
Milliman USA, Inc.
Miltenyi Biotec, Inc.
National Marrow Donor Program
Nature Publishing Group
New York Blood Center
Novartis Oncology
Oncology Nursing Society
Osiris Therapeutics, Inc.
Otsuka America Pharmaceutical, Inc.
Pall Life Sciences
Pfizer Inc
Saladax Biomedical, Inc.
Schering Corporation
Society for Healthcare Epidemiology of America
Soligenix, Inc.
StemCyte, Inc.
StemSoft Software, Inc
Sysmex America, Inc.
Thermogenesis Corporation
Vidacare Corporation
Vion Pharmaceuticals, Inc
ViraCor Laboratories
ViroPharma, Inc.
Well-point, Inc.
Grant number Public Health Service from National Cancer Institute (NCI): U24-CA76518
NHLBI: 5U01HL069294
Health Resources and Services Administration (HRSA/DHHS): HHSH234200637015C
Office of Naval Research: N00014-06-1-0704
Office of Naval Research: N00014-08-1-0058
Date 2011-05-01
Published in Biology of Blood and Marrow Transplantation. New York: Elsevier B.V., v. 17, n. 5, p. 723-728, 2011.
ISSN 1083-8791 (Sherpa/Romeo, impact factor)
Publisher Elsevier B.V.
Extent 723-728
Origin http://dx.doi.org/10.1016/j.bbmt.2010.08.016
Access rights Open access Open Access
Type Article
Web of Science ID WOS:000290061500016
URI http://repositorio.unifesp.br/handle/11600/33644

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