Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial
| Autor: | LOCATELLI F, NOLLKE P, ZECCA M, KORTHOF E, LANINO E, PETERS C, KABISCH H, UDERZO C, BONFIM CS, BADER P, DILLOO D, STARY J, FISCHER A, REVESZ T, FUHRER M, HASLE H, TREBO M, VAN DEN HEUVEL EIBRINK MM, FENU S, STRAHM B, GIORGIANI G, BONORA MR, DUFFNER U, NIEMEYER CM, EUROPEAN WORKING GROUP ON CHILDHOOD MDS, EUROPEAN BLOOD AND MARROW TRANSPLANTATION GROUP, PESSION, ANDREA |
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| Přispěvatelé: | Pediatrics, LOCATELLI F, NOLLKE P, ZECCA M, KORTHOF E, LANINO E, PETERS C, PESSION A., KABISCH H, UDERZO C, BONFIM CS, BADER P, DILLOO D, STARY J, FISCHER A, REVESZ T, FUHRER M, HASLE H, TREBO M, VAN DEN HEUVEL-EIBRINK MM, FENU S, STRAHM B, GIORGIANI G, BONORA MR, DUFFNER U, NIEMEYER CM, EUROPEAN WORKING GROUP ON CHILDHOOD MDS, EUROPEAN BLOOD AND MARROW TRANSPLANTATION GROUP. |
| Rok vydání: | 2005 |
| Předmět: |
Male
medicine.medical_specialty Pediatrics Adolescent medicine.medical_treatment Immunology Graft vs Host Disease Hematopoietic stem cell transplantation Biochemistry Disease-Free Survival Recurrence hemic and lymphatic diseases medicine Humans Cumulative incidence Child Survival rate Juvenile myelomonocytic leukemia business.industry Hematopoietic Stem Cell Transplantation Infant Cell Biology Hematology medicine.disease Surgery Europe Survival Rate Leukemia Treatment Outcome surgical procedures operative medicine.anatomical_structure Leukemia Myeloid Child Preschool Cord blood Female Bone marrow business Busulfan Follow-Up Studies medicine.drug |
| Zdroj: | Locatelli, F, Nöllke, P, Zecca, M, Korthof, E, Lanino, E, Peters, C, Pession, A, Kabisch, H, Uderzo, C, Bonfim, CS, Bader, P, Dilloo, D, Stary, J, Fischer, A, Revesz, T, Führer, M, Hasle, H, Trebo, M, Heuvel-Eibrink, MM, Fenu, S, Strahm, B, Giorgiani, G, Bonora, MR, Duffner, U & Niemeyer, CM 2005, ' Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial ', Blood, vol. 105, pp. 410-419 . Blood, 105(1), 410-419. American Society of Hematology |
| ISSN: | 1528-0020 0006-4971 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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