Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial |
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Authors: | Locatelli Franco,Nöllke Peter,Zecca Marco,Korthof Elisabeth,Lanino Edoardo,Peters Christina,Pession Andrea,Kabisch Hartmut,Uderzo Cornelio,Bonfim Carmen S,Bader Peter,Dilloo Dagmar,Stary Jan,Fischer Alexandra,Révész Tom,Führer Monika,Hasle Henrik,Trebo Monika,van den Heuvel-Eibrink Marry M,Fenu Susanna,Strahm Brigitte,Giorgiani Giovanna,Bonora Mario Regazzi,Duffner Ulrich,Niemeyer Charlotte M European Working Group on Childhood MDS European Blood Marrow Transplantation Group |
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Affiliation: | Oncoematologia Pediatrica, Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy. f.locatelli@smatteo.pv.it |
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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. (Blood. 2005;105:410-419) |
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