open access publication

Article, 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, ISSN 1365-2141, 0007-1048, Volume 205, 1, Pages 268-279, 10.1111/bjh.19535

Contributors

Pichler, Herbert 0000-0001-5107-5123 (Corresponding author) [1] [2] Sedlacek, Petr 0000-0001-7895-2886 [3] [4] Meisel, Roland 0000-0001-5230-0628 [5] Beier, Rita 0000-0002-7568-8749 [6] Faraci, Maura 0000-0001-5715-501X [7] Kałwak, Krzysztof 0000-0003-1174-5799 [8] Ifversen, Marianne R S 0000-0002-2558-1935 [9] Müller, Ingo 0000-0002-7477-6632 [10] Stein, Jerry [11] Vettenranta, Kim K 0000-0002-8535-0135 [12] Kropshofer, Gabriele [13] Kolenova, Alexandra 0000-0003-4300-5533 [14] Karlhuber, Susanne [1] Glogova, Evgenia [1] Poetschger, Ulrike [1] [2] Peters, Christina 0000-0003-0369-8515 [1] [2] Suttorp, Meinolf 0000-0002-9522-8838 [15] Matthes-Leodolter, Susanne [1] [2] Balduzzi, Adriana Cristina 0000-0002-5879-0610 [16] [17]

Affiliations

  1. [1] Medical University of Vienna
    2. [NORA names: Austria; Europe, EU; OECD];
  2. [2] St Anna Children's Hospital
    3. [NORA names: Austria; Europe, EU; OECD];
  3. [3] Charles University
    4. [NORA names: Czechia; Europe, EU; OECD];
  4. [4] University Hospital in Motol
    5. [NORA names: Czechia; Europe, EU; OECD];
  5. [5] Heinrich Heine University Düsseldorf
    6. [NORA names: Germany; Europe, EU; OECD];

Abstract

This 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.

Keywords

CP-CML, First, GVHD, GVHD-free, I-BFM, RIC, TKI, adolescents, cell transplantation, children, chronic graft-versus-host disease, chronic myeloid leukemia, chronic phase, conditions, disease, disease-free, donor, donor lymphocyte infusion, efficacy, event-free survival, follow-up, graft-versus-host disease, group, haematopoietic stem cell transplantation, immunosuppression, incidence, incidence of chronic graft-versus-host disease, infusion, inhibitors, intensity conditioning, intervention, kinase inhibitors, leukemia, leukemia-free survival, loss, lymphocyte infusion, median follow-up, molecular response, mortality, multicentre trial, myeloid leukemia, overall survival, patients, phase, post-transplant, pretreatment, prospective multicentre trial, protocol, reduced intensity conditioning, response, response to first-, retreat, safety, second-generation tyrosine kinase inhibitors, siblings, stem cell transplantation, stopping immunosuppression, study group, survival, transcription, transplant-related mortality, transplantation, trials, tyrosine kinase inhibitors, years

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