Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM
Year: 2024
Type: article
Abstract: 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.
Source: Blood Advances
Authors: Krzysztof Kalwak, Laura M. Moser, Ulrike Pötschger, Peter Bader, Katharina Kleinschmidt, Roland Meisel, Jean-Hugues Dalle, Akif Yesilipek, Adriana Balduzzi, Gergely Krivan, Evgenios Goussetis, Raquel Staciuk, Petr Sedlacek, Herbert Pichler, Peter Svec, Melissa Gabriel, Tayfun Güngör, Ernest Bilic, Jochen Buechner, Marleen Renard, Kim Vettenranta, Marianne Ifversen, Cristina Diaz-de-Heredia, Jerry Stein, Jacek Toporski, Marc Bierings, Christina Peters, Marc Ansari, Franco Locatelli
Institutions: University of Wrocław, Wroclaw Medical University, Goethe University Frankfurt, University Hospital Frankfurt, St. Anna Children's Cancer Research Institute, University Hospital Regensburg, Heinrich Heine University Düsseldorf, Université Paris Cité, Assistance Publique – Hôpitaux de Paris, Hôpital Robert-Debré, Medical Park Tarsus Hastanesi, Acıbadem University Atakent Hospital, Azienda Ospedaliera San Gerardo, University of Milano-Bicocca, Children's Hospital Agia Sophia, Garrahan Hospital, University Hospital in Motol, St Anna Children's Hospital, Medical University of Vienna, Národný ústav detských chorôb, Comenius University Bratislava, Children's Hospital at Westmead, University Children's Hospital Zurich, University Hospital Centre Zagreb, Oslo University Hospital, Universitair Ziekenhuis Leuven, University of Helsinki, Copenhagen University Hospital, Rigshospitalet, Vall d'Hebron Institut de Recerca, Vall d'Hebron Hospital Universitari, Tel Aviv University, Schneider Children's Medical Center, Karolinska University Hospital, Princess Máxima Center, University of Geneva, University Hospital of Geneva, Università Cattolica del Sacro Cuore, Bambino Gesù Children's Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,000
Funder: Cancer Research Institute
OpenAlex ID: W4404793058