Pichler, HerbertSedlacek, PetrMeisel, RolandBeier, RitaFaraci, MauraKalwak, KrzysztofIfversen, MarianneMüller, IngoStein, JerryVettenranta, KimKropshofer, GabrieleKolenova, AlexandraKarlhuber, SusanneGlogova, EvgeniaPoetschger, UlrikePeters, ChristinaSuttorp, MeinolfMatthes-Leodolter, SusanneBalduzzi, Adriana2025-01-132025-01-132024Pichler, H, Sedlacek, P, Meisel, R, Beier, R, Faraci, M, Kalwak, K, Ifversen, M, Müller, I, Stein, J, Vettenranta, K, Kropshofer, G, Kolenova, A, Karlhuber, S, Glogova, E, Poetschger, U, Peters, C, Suttorp, M, Matthes-Leodolter, S & Balduzzi, A 2024, 'Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia : A prospective multicentre trial of the I-BFM Study Group', British Journal of Haematology, vol. 205, no. 1, pp. 268-279. https://doi.org/10.1111/bjh.19535http://hdl.handle.net/10138/590319This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan-based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic myeloid leukaemia (CML) in chronic phase (CP). Thirty-two patients were transplanted from matched siblings or matched unrelated donors. In 22 patients, HSCT was performed due to insufficient molecular response or loss of response to first- or second-generation tyrosine kinase inhibitor (TKI), with pretransplant BCR::ABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCR::ABL1-guided intervention with TKI retreatment in the first year and donor lymphocyte infusions (DLI) in the second-year post-transplant. All patients engrafted. The 1-year transplant-related mortality was 3% (confidence interval [CI]: 0%–6%). After a median follow-up of 6.3 years, 5-year overall survival and event-free survival are 97% (CI: 93%–100%) and 91% (CI: 79%–100%) respectively. The current 5-year leukaemia-free survival with BCR::ABL1 <0.01% is 97% (CI: 88%–100%) and the current TKI- and DLI-free survival is 95% (CI: 85%–100%). The incidence of chronic graft-versus-host disease (GvHD) was 32%, being severe in four patients (13%). At last follow-up, 31 patients are GvHD-free and have stopped immunosuppression. RIC HSCT following pretreatment with TKI is feasible and effective in children and adolescents with CP-CML with an excellent disease-free and TKI-free survival.12engcc_by_nc_ndinfo:eu-repo/semantics/openAccessallogeneic stem cell transplantationchildren and adolescentschronic myeloid leukaemiareduced intensity conditioningGeneral medicine, internal medicine and other clinical medicineArticleopenAccesse4f07be2-cbac-4a36-b453-fdc2c4b3866685194875268001231997300001