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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

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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, Jahrgang 205, Nr. 1, 07.2024, S. 268-279.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Transfer › Begutachtung

Harvard

Pichler, 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', BRIT J HAEMATOL, Jg. 205, Nr. 1, S. 268-279. https://doi.org/10.1111/bjh.19535

APA

Pichler, 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. BRIT J HAEMATOL, 205(1), 268-279. https://doi.org/10.1111/bjh.19535

Vancouver

Pichler H, Sedlacek P, Meisel R, Beier R, Faraci M, Kalwak K et al. 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. BRIT J HAEMATOL. 2024 Jul;205(1):268-279. https://doi.org/10.1111/bjh.19535

Bibtex

@article{36199dad9ac04da48f84eea5467a86b5,
title = "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",
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.",
author = "Herbert Pichler and Petr Sedlacek and Roland Meisel and Rita Beier and Maura Faraci and Krzysztof Kalwak and Marianne Ifversen and Ingo M{\"u}ller and Jerry Stein and Kim Vettenranta and Gabriele Kropshofer and Alexandra Kolenova and Susanne Karlhuber and Evgenia Glogova and Ulrike Poetschger and Christina Peters and Meinolf Suttorp and Susanne Matthes-Leodolter and Adriana Balduzzi",
note = "{\textcopyright} 2024 The Author(s). British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.",
year = "2024",
month = jul,
doi = "10.1111/bjh.19535",
language = "English",
volume = "205",
pages = "268--279",
journal = "BRIT J HAEMATOL",
issn = "0007-1048",
publisher = "Wiley-Blackwell",
number = "1",

}

RIS

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 -