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Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies

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Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. / Kalwak, Krzysztof; Mielcarek, Monika; Patrick, Katharine; Styczynski, Jan; Bader, Peter; Corbacioglu, Selim; Burkhardt, Birgit; Sykora, Karl Walter; Drabko, Katarzyna; Gozdzik, Jolanta; Fagioli, Franca; Greil, Johann; Gruhn, Bernd; Beier, Rita; Locatelli, Franco; Müller, Ingo; Schlegel, Paul Gerhardt; Sedlacek, Petr; Stachel, Klaus Daniel; Hemmelmann, Claudia; Möller, Ann-Kristin; Baumgart, Joachim; Vora, Ajay.

In: BONE MARROW TRANSPL, Vol. 55, No. 10, 10.2020, p. 1996-2007.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Kalwak, K, Mielcarek, M, Patrick, K, Styczynski, J, Bader, P, Corbacioglu, S, Burkhardt, B, Sykora, KW, Drabko, K, Gozdzik, J, Fagioli, F, Greil, J, Gruhn, B, Beier, R, Locatelli, F, Müller, I, Schlegel, PG, Sedlacek, P, Stachel, KD, Hemmelmann, C, Möller, A-K, Baumgart, J & Vora, A 2020, 'Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies', BONE MARROW TRANSPL, vol. 55, no. 10, pp. 1996-2007. https://doi.org/10.1038/s41409-020-0869-6

APA

Kalwak, K., Mielcarek, M., Patrick, K., Styczynski, J., Bader, P., Corbacioglu, S., Burkhardt, B., Sykora, K. W., Drabko, K., Gozdzik, J., Fagioli, F., Greil, J., Gruhn, B., Beier, R., Locatelli, F., Müller, I., Schlegel, P. G., Sedlacek, P., Stachel, K. D., ... Vora, A. (2020). Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. BONE MARROW TRANSPL, 55(10), 1996-2007. https://doi.org/10.1038/s41409-020-0869-6

Vancouver

Kalwak K, Mielcarek M, Patrick K, Styczynski J, Bader P, Corbacioglu S et al. Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies. BONE MARROW TRANSPL. 2020 Oct;55(10):1996-2007. https://doi.org/10.1038/s41409-020-0869-6

Bibtex

@article{c5887c3a6c014bc1b1816d076e9b44d2,
title = "Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies",
abstract = "Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dose of 10 mg/m2/day (7.7%), 12 g/m2/day (35.4%), or 14 g/m2/day (56.9%) according to their individual body surface area in combination with fludarabine and thiotepa. The incidence of complete donor chimerism at day +28 was 98.4% with no primary and only one secondary graft failure. At 36 months, NRM was only 3.1%, while relapse incidence was 21.7%, and overall survival was 83.0%. The cumulative incidence of acute graft-vs.-host disease was 45.3% for grades I-IV and 26.6% for grades II-IV. At 36 months, 25.8% overall and 19.4% moderate/severe chronic graft-vs.-host disease were reported. These data confirm the safe and effective use of treosulfan-based conditioning in pediatric patients with hematological malignancies. Therefore, treosulfan/fludarabine/thiotepa can be recommended for myeloablative conditioning in children with hematological malignancies.",
author = "Krzysztof Kalwak and Monika Mielcarek and Katharine Patrick and Jan Styczynski and Peter Bader and Selim Corbacioglu and Birgit Burkhardt and Sykora, {Karl Walter} and Katarzyna Drabko and Jolanta Gozdzik and Franca Fagioli and Johann Greil and Bernd Gruhn and Rita Beier and Franco Locatelli and Ingo M{\"u}ller and Schlegel, {Paul Gerhardt} and Petr Sedlacek and Stachel, {Klaus Daniel} and Claudia Hemmelmann and Ann-Kristin M{\"o}ller and Joachim Baumgart and Ajay Vora",
year = "2020",
month = oct,
doi = "10.1038/s41409-020-0869-6",
language = "English",
volume = "55",
pages = "1996--2007",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "10",

}

RIS

TY - JOUR

T1 - Treosulfan-fludarabine-thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies

AU - Kalwak, Krzysztof

AU - Mielcarek, Monika

AU - Patrick, Katharine

AU - Styczynski, Jan

AU - Bader, Peter

AU - Corbacioglu, Selim

AU - Burkhardt, Birgit

AU - Sykora, Karl Walter

AU - Drabko, Katarzyna

AU - Gozdzik, Jolanta

AU - Fagioli, Franca

AU - Greil, Johann

AU - Gruhn, Bernd

AU - Beier, Rita

AU - Locatelli, Franco

AU - Müller, Ingo

AU - Schlegel, Paul Gerhardt

AU - Sedlacek, Petr

AU - Stachel, Klaus Daniel

AU - Hemmelmann, Claudia

AU - Möller, Ann-Kristin

AU - Baumgart, Joachim

AU - Vora, Ajay

PY - 2020/10

Y1 - 2020/10

N2 - Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dose of 10 mg/m2/day (7.7%), 12 g/m2/day (35.4%), or 14 g/m2/day (56.9%) according to their individual body surface area in combination with fludarabine and thiotepa. The incidence of complete donor chimerism at day +28 was 98.4% with no primary and only one secondary graft failure. At 36 months, NRM was only 3.1%, while relapse incidence was 21.7%, and overall survival was 83.0%. The cumulative incidence of acute graft-vs.-host disease was 45.3% for grades I-IV and 26.6% for grades II-IV. At 36 months, 25.8% overall and 19.4% moderate/severe chronic graft-vs.-host disease were reported. These data confirm the safe and effective use of treosulfan-based conditioning in pediatric patients with hematological malignancies. Therefore, treosulfan/fludarabine/thiotepa can be recommended for myeloablative conditioning in children with hematological malignancies.

AB - Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adults. Therefore, we prospectively evaluated the safety and efficacy of treosulfan-based conditioning in children with hematological malignancies in this phase II trial. Overall, 65 children with acute lymphoblastic leukemia (35.4%), acute myeloid leukemia (44.6%), myelodysplastic syndrome (15.4%), or juvenile myelomonocytic leukemia (4.6%) received treosulfan intravenously at a dose of 10 mg/m2/day (7.7%), 12 g/m2/day (35.4%), or 14 g/m2/day (56.9%) according to their individual body surface area in combination with fludarabine and thiotepa. The incidence of complete donor chimerism at day +28 was 98.4% with no primary and only one secondary graft failure. At 36 months, NRM was only 3.1%, while relapse incidence was 21.7%, and overall survival was 83.0%. The cumulative incidence of acute graft-vs.-host disease was 45.3% for grades I-IV and 26.6% for grades II-IV. At 36 months, 25.8% overall and 19.4% moderate/severe chronic graft-vs.-host disease were reported. These data confirm the safe and effective use of treosulfan-based conditioning in pediatric patients with hematological malignancies. Therefore, treosulfan/fludarabine/thiotepa can be recommended for myeloablative conditioning in children with hematological malignancies.

U2 - 10.1038/s41409-020-0869-6

DO - 10.1038/s41409-020-0869-6

M3 - SCORING: Journal article

C2 - 32203268

VL - 55

SP - 1996

EP - 2007

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 10

ER -