title: Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children.
abstract: 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.
authors.affiliation: Amgen, Thousand Oaks, California, USA.
authors.forename: Paul
authors.initials: P
authors.lastname: Woodard
authors.forename: Paul A
authors.initials: PA
authors.lastname: Carpenter
authors.forename: Stella M
authors.initials: SM
authors.lastname: Davies
authors.forename: Thomas G
authors.initials: TG
authors.lastname: Gross
authors.forename: Wensheng
authors.initials: W
authors.lastname: He
authors.forename: Mei-Jie
authors.initials: MJ
authors.lastname: Zhang
authors.forename: Biljana N
authors.initials: BN
authors.lastname: Horn
authors.forename: David A
authors.initials: DA
authors.lastname: Margolis
authors.forename: John P
authors.initials: JP
authors.lastname: Perentesis
authors.forename: Jean E
authors.initials: JE
authors.lastname: Sanders
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
authors.forename: Adriana
authors.initials: A
authors.lastname: Seber
authors.forename: William G
authors.initials: WG
authors.lastname: Woods
authors.forename: Mary
authors.initials: M
authors.lastname: Eapen
mesh_terms: 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
chemical_list: D000970:Antineoplastic Agents; D006680:HLA Antigens
references.citation: Blood. 1999 Apr 1;93(7):2196-201
references.pmid: 10090927
references.citation: Br J Haematol. 1997 Mar;96(4):749-56
references.pmid: 9074418
references.citation: Blood. 2007 Dec 15;110(13):4576-83
references.pmid: 17785583
references.citation: Stat Med. 1997 Apr 30;16(8):901-10
references.pmid: 9160487
references.citation: Leukemia. 2003 Feb;17(2):277-82
references.pmid: 12592323
references.citation: Bone Marrow Transplant. 2001 Feb;27(4):349-54
references.pmid: 11313663
references.citation: Blood. 1990 Jun 15;75(12):2459-64
references.pmid: 2350582
references.citation: Leukemia. 1995 Sep;9(9):1569-72
references.pmid: 7658725
references.citation: J Clin Oncol. 2002 Jan 15;20(2):434-40
references.pmid: 11786571
references.citation: Leukemia. 1994 May;8(5):844-9
references.pmid: 8182940
references.citation: Biol Blood Marrow Transplant. 2009 Aug;15(8):948-55
references.pmid: 19589484
references.citation: Blood. 2002 Aug 15;100(4):1201-7
references.pmid: 12149198
references.citation: Bone Marrow Transplant. 2004 Apr;33(8):805-14
references.pmid: 14755311
references.citation: Bone Marrow Transplant. 2007 Aug;40(4):329-33
references.pmid: 17589538
references.citation: Bone Marrow Transplant. 1995 Jun;15(6):825-8
references.pmid: 7581076