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Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM
K. Kalwak, LM. Moser, U. Pötschger, P. Bader, K. Kleinschmidt, R. Meisel, JH. Dalle, A. Yesilipek, A. Balduzzi, G. Krivan, E. Goussetis, R. Staciuk, P. Sedlacek, H. Pichler, P. Svec, M. Gabriel, T. Güngör, E. Bilic, J. Buechner, M. Renard, K....
Language English Country United States
Document type Journal Article, Comparative Study, Multicenter Study, Randomized Controlled Trial
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- MeSH
- Precursor Cell Lymphoblastic Leukemia-Lymphoma * therapy mortality MeSH
- Busulfan * analogs & derivatives therapeutic use MeSH
- Child MeSH
- Transplantation, Homologous MeSH
- Humans MeSH
- Adolescent MeSH
- Graft vs Host Disease * etiology MeSH
- Child, Preschool MeSH
- Transplantation Conditioning * methods MeSH
- Hematopoietic Stem Cell Transplantation * adverse effects MeSH
- Vidarabine analogs & derivatives therapeutic use administration & dosage MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Multicenter Study MeSH
- Randomized Controlled Trial MeSH
- Comparative Study MeSH
The superiority of total body irradiation (TBI)-based vs chemotherapy conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with acute lymphoblastic leukemia (ALL) has been established in the international, prospective phase-3 FORUM study, randomizing 417 patients aged 4-18 years in complete remission (CR), who received allo-HSCT from HLA-matched sibling or unrelated donors. Because of the unavailability of TBI in some regions and to accommodate individual contraindications, this study reports the prespecified comparison of outcomes of patients receiving busulfan (BU)- or treosulfan (TREO)-based regimens from 2013 to 2018. Overall, 180 and 128 patients received BU/thiotepa (THIO)/fludarabine (FLU) or TREO/THIO/FLU, respectively. Data were analyzed as of February 2023, with a median follow-up of 4.2 years (range, 0.3-9.1). 3-year overall survival was 0.71 (BU, 95% confidence interval [0.64-0.77]) and 0.72 (TREO, [0.63-0.79]) and 3-year event-free survival was 0.60 (BU, [0.53-0.67]) and 0.55 (TREO, [0.46-0.63]). The 3-year cumulative incidence of relapse (BU, 0.31 [0.25-0.38]; TREO, 0.36 [0.27-0.44]); and nonrelapse mortality (BU, 0.08 [0.05-0.13]; TREO, 0.09 [0.05-0.15]) were comparable. One case of fatal veno-occlusive disease occurred in each group. No significant differences in acute and chronic graft-versus-host disease (GVHD) or 3-year GVHD-free and relapse-free survival (BU, 0.48 [0.41-0.55]; TREO, 0.45 [0.37-0.54]) were recorded. Outcomes for patients in first and second CR were similar irrespective of the regimen. In conclusion, BU/THIO/FLU or TREO/THIO/FLU regimens can be an alternative to TBI for patients with ALL aged >4 years with contraindications or lack of access to TBI. This trial was registered at www.ClinicalTrials.gov as #NCT01949129.
Department of Paediatric Oncology University Hospital Leuven Leuven Belgium
Department of Pediatric Hematology and Oncology Oslo University Hospital Oslo Norway
Department of Pediatric Hematology and Oncology University Hospital Motol Prague Czech Republic
Division of Pediatric Hematology and Oncology Hospital Universitari Vall d'Hebron Barcelona Spain
Hospital de Pediatría Prof Dr Juan P Garrahan Buenos Aires Argentina
Medical Park Antalya Hospital Antalya Turkey
Pediatric Hematopoietic Stem Cell Unit Fondazione IRCCS San Gerardo dei Tintori Monza Italy
Princess Máxima Center for Pediatric Oncology Utrecht The Netherlands
School of Medicine and Surgery University of Milano Bicocca Monza Italy
St Anna Children's Cancer Research Institute Vienna Austria
Stem Cell Transplant Unit Agia Sofia Children's Hospital Athens Greece
References provided by Crossref.org
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