| Title: |
COMPARABLE OUTCOMES AFTER BUSULFAN- OR TREOSULFAN-BASED CONDITIONING FOR ALLO-HSCT IN CHILDREN WITH ALL-RESULTS OF FORUM |
| Authors: |
Kałwak, Krzysztof; Moser, Laura M; Pötschger, Ulrike; Bader, Peter; Kleinschmidt, Katharina; Meisel, Roland; Dalle, Jean-Hugues; Yeşilipek, M Akif; Balduzzi, Adriana Cristina; Kriván, Gergely; Goussetis, Evgenios; Staciuk, Raquel; Sedlacek, Petr; Pichler, Herbert; Svec, Peter; Gabriel, Melissa; Güngör, Tayfun; Bilic, Ernest; Buechner, Jochen; Renard, Marleen; Vettenranta, Kim; Ifversen, Marianne; Diaz-de-Heredia, Cristina; Stein, Jerry; Toporski, Jacek; Bierings, Marc B; Peters, Christina; Ansari, Marc; Locatelli, Franco |
| Source: |
Kałwak, Krzysztof; Moser, Laura M; Pötschger, Ulrike; Bader, Peter; Kleinschmidt, Katharina; Meisel, Roland; Dalle, Jean-Hugues; Yeşilipek, M Akif; Balduzzi, Adriana Cristina; Kriván, Gergely; Goussetis, Evgenios; Staciuk, Raquel; Sedlacek, Petr; Pichler, Herbert; Svec, Peter; Gabriel, Melissa; Güngör, Tayfun; Bilic, Ernest; Buechner, Jochen; Renard, Marleen; Vettenranta, Kim; Ifversen, Marianne; Diaz-de-Heredia, Cristina; Stein, Jerry; Toporski, Jacek; Bierings, Marc B; Peters, Christina; Ansari, Marc; Locatelli, Franco (2025). COMPARABLE OUTCOMES AFTER BUSULFAN- OR TREOSULFAN-BASED CONDITIONING FOR ALLO-HSCT IN CHILDREN WITH ALL-RESULTS OF FORUM. Blood Advances, 9(4):741-751. |
| Publisher Information: |
American Society of Hematology |
| Publication Year: |
2025 |
| Collection: |
University of Zurich (UZH): ZORA (Zurich Open Repository and Archive |
| Subject Terms: |
Medical Clinic; 610 Medicine & health |
| Description: |
The superiority of TBI-based versus chemotherapy-conditioning for allo-HSCT in children with ALL has been established in the international, prospective phase-III FORUM study (#NCT01949129), randomizing 417 patients ≤ 18 years at diagnosis (4-21 years at HSCT) in CR, transplanted from HLA-matched sibling or unrelated donors. Due to the unavailability of TBI in some regions and to accommodate individual contraindications, this study reports the pre-specified comparison of outcomes of patients receiving busulfan-based (BU) or treosulfan-based (TREO) regimens from 2013 to 2018. 180 and 128 patients (median age 9.9 years) received BU/THIO/FLU or TREO/THIO/FLU, respectively. Data were analysed as of 02/2023, with a median follow-up of 4.2 years (range 0.3-9.1). Three-year overall survival was 0.71 (0.64-0.77) (BU/THIO/FLU) and 0.72 (0.63-0.79) (TREO/THIO/FLU), event-free survival was 0.60 (0.53-0.67) (BU/THIO/FLU) and 0.55 (0.46-0.63) (TREO/THIO/FLU), with both p = NS. The 3-year cumulative incidence of relapse (0.31 (0.25-0.38) BU, 0.36 (0.27-0.44) TREO, p = 0.779) and non-relapse mortality (0.08 (0.05-0.13) BU, 0.09 (0.05-0.15) TREO, p = 0.831) were comparable (p = NS). One case of fatal veno-occlusive disease occurred in each group. No significant differences in acute and chronic GvHD or GvHD-free and relapse-free survival (0.48 (0.41-0.55) BU, 0.45 (0.37-0.54) TREO, p = 0.89) were recorded. Outcomes for CR1 or CR2 patients were similar irrespective of the regimen used. In conclusion, BU/THIO/FLU or TREO/THIO/FLU regimens can be an alternative to TBI for ALL patients > 4 years with contraindications or lack of access to TBI. |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/pdf |
| Language: |
English |
| ISSN: |
2473-9529 |
| Relation: |
https://www.zora.uzh.ch/id/eprint/269917/1/bloodadvances_Treo_vs_Bu.2024014548.pdf; info:pmid/39602342; urn:issn:2473-9529 |
| DOI: |
10.1182/bloodadvances.2024014548 |
| Availability: |
https://www.zora.uzh.ch/id/eprint/269917/; https://www.zora.uzh.ch/id/eprint/269917/1/bloodadvances_Treo_vs_Bu.2024014548.pdf; https://doi.org/10.1182/bloodadvances.2024014548 |
| Rights: |
info:eu-repo/semantics/openAccess ; Creative Commons: Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) ; http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| Accession Number: |
edsbas.6ABE3FD |
| Database: |
BASE |