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
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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. / Pichler, Herbert; Sedlacek, Petr; Meisel, Roland; Beier, Rita; Faraci, Maura; Kalwak, Krzysztof; Ifversen, Marianne; Müller, Ingo; Stein, Jerry; Vettenranta, Kim; Kropshofer, Gabriele; Kolenova, Alexandra; Karlhuber, Susanne; Glogova, Evgenia; Poetschger, Ulrike; Peters, Christina; Suttorp, Meinolf; Matthes-Leodolter, Susanne; Balduzzi, Adriana.
In: BRIT J HAEMATOL, Vol. 205, No. 1, 07.2024, p. 268-279.Research output: SCORING: Contribution to journal › SCORING: Journal article › Transfer › peer-review
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TY - JOUR
T1 - 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
AU - Pichler, Herbert
AU - Sedlacek, Petr
AU - Meisel, Roland
AU - Beier, Rita
AU - Faraci, Maura
AU - Kalwak, Krzysztof
AU - Ifversen, Marianne
AU - Müller, Ingo
AU - Stein, Jerry
AU - Vettenranta, Kim
AU - Kropshofer, Gabriele
AU - Kolenova, Alexandra
AU - Karlhuber, Susanne
AU - Glogova, Evgenia
AU - Poetschger, Ulrike
AU - Peters, Christina
AU - Suttorp, Meinolf
AU - Matthes-Leodolter, Susanne
AU - Balduzzi, Adriana
N1 - © 2024 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.
PY - 2024/7
Y1 - 2024/7
N2 - 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.
AB - 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.
U2 - 10.1111/bjh.19535
DO - 10.1111/bjh.19535
M3 - SCORING: Journal article
C2 - 38803040
VL - 205
SP - 268
EP - 279
JO - BRIT J HAEMATOL
JF - BRIT J HAEMATOL
SN - 0007-1048
IS - 1
ER -