title: Allogeneic bone marrow transplantation for juvenile myelomonocytic leukemia: a single center experience of 23 patients.
abstract: Juvenile myelomonocytic leukemia (JMML) is a childhood leukemia for which allogeneic BMT is the only curative therapy. At our pediatric stem cell transplantation unit, we performed 26 BMTs in 23 children (age 0.5-12.7 years). Conditioning was CY/TBI based (1980-1996, n=14) or BU/CY/melphalan based (1996-2001, n=9). Donors were HLA-identical siblings (n=11), unrelated volunteers (n=9) or mismatched family members (n=3). A total of 10 patients survive in CR (median follow-up 6.8 years, range 3.1-22.2 years). Relapse or persistent disease was observed in eight and two patients, respectively. Nine of these patients died, one achieved a second remission following acute nonlymphatic leukemia chemotherapy (duration to date 5.3 years). Transplant-related mortality occurred in four patients. Overall survival at 5 and 10 years was 43.5%. Using T-cell-depleted, one-antigen mismatched unrelated donors was the only significant adverse factor associated with relapse in multivariate analysis (P=0.039, hazard ratio 4.9). Together with a trend towards less relapse in patients with graft-versus-host-disease and in patients transplanted with matched unrelated donors, this suggests a graft-versus-leukemia effect of allogeneic BMT in JMML.
authors.affiliation: Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. e.t.korthof@lumc.nl
authors.forename: E T
authors.initials: ET
authors.lastname: Korthof
authors.forename: P P
authors.initials: PP
authors.lastname: Snijder
authors.forename: A A
authors.initials: AA
authors.lastname: de Graaff
authors.forename: A C
authors.initials: AC
authors.lastname: Lankester
authors.forename: R G M
authors.initials: RG
authors.lastname: Bredius
authors.forename: L M
authors.initials: LM
authors.lastname: Ball
authors.forename: J L W T
authors.initials: JL
authors.lastname: Lie
authors.forename: J M
authors.initials: JM
authors.lastname: Vossen
authors.forename: R M
authors.initials: RM
authors.lastname: Egeler
mesh_terms: D016026:Bone Marrow Transplantation / Q000379:methods* / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease; D020544:Graft vs Leukemia Effect; D006648:Histocompatibility; D006801:Humans; D007223:Infant; D015477:Leukemia, Myelomonocytic, Chronic / Q000401:mortality / Q000628:therapy*; D008212:Lymphocyte Depletion; D008297:Male; D015999:Multivariate Analysis; D012008:Recurrence; D012189:Retrospective Studies; D012307:Risk Factors; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods / Q000401:mortality; D014184:Transplantation, Homologous; D016896:Treatment Outcome