Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

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.

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Publication Details

Journal
Blood Advances
Published
2024-11-27
DOI
https://doi.org/10.1182/bloodadvances.2024014548
Citations
2
Primary Topic
Acute Lymphoblastic Leukemia research
Type
article
Field-Weighted Citation Impact
1.09

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article

Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

Krzysztof Kałwak, Herbert Pichler, Jean‐Hugues Dalle, Christina Peters et al.
2 citations
Blood Advances
Acute Lymphoblastic Leukemia research
1.09
article

Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

Krzysztof Kałwak, Herbert Pichler, Jean‐Hugues Dalle, Christina Peters, Jerry Stein, Ulrike Pötschger, Franco Locatelli, Peter Bader, Gergely Kriván, Ernest Bilić, Katharina Kleinschmidt, Jochen Buechner, Melissa Gabriel, M. Akif Yeşilipek, Roland Meisel, Cristina Díaz de Heredia, Jacek Toporski, Evgenios Goussetis, Marianne Ifversen, Raquel Staciuk, Adriana Balduzzi, Marc Ansari, Kim Vettenranta, Marc Bierings, Laura M. Moser, Peter Švec, Petr Sedláček, Marleen Renard, Tayfun Güngör
article en
2 citations

Abstract

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.

Blood AdvancesVol. 9(4)
Goethe University Frankfurt (DE), University of Geneva (CH), Oslo University Hospital (NO), Karolinska University Hospital (SE), University of Helsinki (FI), University Hospital Centre Zagreb (HR), Université Paris Cité (FR), Garrahan Hospital (AR), Helsinki University Hospital (FI), Children's Hospital at Westmead (AU), Copenhagen University Hospital (DK), Rigshospitalet (DK), University Hospital Regensburg (DE), Wroclaw Medical University (PL), Helsinki Children's Hospital (FI), National Institute of Cardiovascular Diseases (SK), Hôpital Beau-Séjour (CH), Assistance Publique – Hôpitaux de Paris (FR), University Hospital in Motol (CZ), University Hospital of Geneva (CH), Schneider Children's Medical Center (IL), Antalya Eğitim ve Araştırma Hastanesi (TR), Azienda Ospedaliera San Gerardo (IT), Children's Hospital Agia Sophia (GR), Bambino Gesù Children's Hospital (IT), St Anna Children's Hospital (AT), Hôpital Robert-Debré (FR), Princess Máxima Center (NL), Vall d'Hebron Hospital Universitari (ES), University Children's Hospital Zurich (CH), University Hospital Frankfurt (DE), National Center for Epidemiology (HU), University of the Sacred Heart (JP), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Iaso Children’s Hospital (GR), Antalya IVF (TR), St. Anna Children's Cancer Research Institute, Heinrich Heine University Düsseldorf (DE), Geneva College (US), University of Milano-Bicocca (IT), Comenius University Bratislava (SK), Medical University of Vienna (AT), KU Leuven (BE)
Cancer Research Institute
Good health and well-being
Openalex Percentile: Top 19%
Acute Lymphoblastic Leukemia research
1.09
DOIOpen Access PDFOpenAlex
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