title: Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative therapy for juvenile myelomonocytic leukemia (JMML). We, the European Working Group on Childhood MDS (EWOG-MDS) and the European Blood and Marrow Transplantation (EBMT) Group, report the outcome of 100 children (67 boys and 33 girls) with JMML given unmanipulated HSCT after a preparative regimen including busulfan, cyclophosphamide, and melphalan. Forty-eight and 52 children received transplants from an HLA-identical relative or an unrelated donor (UD), respectively. The source of hematopoietic stem cells was bone marrow, peripheral blood, and cord blood in 79, 14, and 7 children, respectively. Splenectomy had been performed before HSCT in 24 children. The 5-year cumulative incidence of transplantation-related mortality and leukemia recurrence was 13% and 35%, respectively. Age older than 4 years predicted an increased risk of disease recurrence. The 5-year probability of event-free survival for children given HSCT from either a relative or a UD was 55% and 49%, respectively (P = NS), with median observation time of patients alive being 40 months (range, 6 to 144). In multivariate analysis, age older than 4 years and female sex predicted poorer outcome. Results of this study compare favorably with previously published reports. Disease recurrence remains the major cause of treatment failure. Outcome of UD-HSCT recipients is comparable to that of children receiving transplants from an HLA-identical sibling.
authors.affiliation: Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy. f.locatelli@smatteo.pv.it
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Peter
authors.initials: P
authors.lastname: Nöllke
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Korthof
authors.forename: Edoardo
authors.initials: E
authors.lastname: Lanino
authors.forename: Christina
authors.initials: C
authors.lastname: Peters
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Hartmut
authors.initials: H
authors.lastname: Kabisch
authors.forename: Cornelio
authors.initials: C
authors.lastname: Uderzo
authors.forename: Carmen S
authors.initials: CS
authors.lastname: Bonfim
authors.forename: Peter
authors.initials: P
authors.lastname: Bader
authors.forename: Dagmar
authors.initials: D
authors.lastname: Dilloo
authors.forename: Jan
authors.initials: J
authors.lastname: Stary
authors.forename: Alexandra
authors.initials: A
authors.lastname: Fischer
authors.forename: Tom
authors.initials: T
authors.lastname: Révész
authors.forename: Monika
authors.initials: M
authors.lastname: Führer
authors.forename: Henrik
authors.initials: H
authors.lastname: Hasle
authors.forename: Monika
authors.initials: M
authors.lastname: Trebo
authors.forename: Marry M
authors.initials: MM
authors.lastname: van den Heuvel-Eibrink
authors.forename: Susanna
authors.initials: S
authors.lastname: Fenu
authors.forename: Brigitte
authors.initials: B
authors.lastname: Strahm
authors.forename: Giovanna
authors.initials: G
authors.lastname: Giorgiani
authors.forename: Mario Regazzi
authors.initials: MR
authors.lastname: Bonora
authors.forename: Ulrich
authors.initials: U
authors.lastname: Duffner
authors.forename: Charlotte M
authors.initials: CM
authors.lastname: Niemeyer
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D007951:Leukemia, Myeloid / Q000276:immunology / Q000473:pathology* / Q000601:surgery*; D008297:Male; D012008:Recurrence; D015996:Survival Rate; D016896:Treatment Outcome