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Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome

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Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome. / Bortnick, Rachel; Wlodarski, Marcin; de Haas, Valerie; De Moerloose, Barbara; Dworzak, Michael; Hasle, Henrik; Masetti, Riccardo; Starý, Jan; Turkiewicz, Dominik; Ussowicz, Marek; Kozyra, Emilia; Albert, Michael; Bader, Peter; Bordon, Victoria; Cario, Gunnar; Beier, Rita; Schulte, Johannes; Bresters, Dorine; Müller, Ingo; Pichler, Herbert; Sedlacek, Petr; Sauer, Martin G; Zecca, Marco; Göhring, Gudrun; Yoshimi, Ayami; Noellke, Peter; Erlacher, Miriam; Locatelli, Franco; Niemeyer, Charlotte M; Strahm, Brigitte.

in: BONE MARROW TRANSPL, Jahrgang 56, Nr. 11, 11.2021, S. 2732-2741.

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

Harvard

Bortnick, R, Wlodarski, M, de Haas, V, De Moerloose, B, Dworzak, M, Hasle, H, Masetti, R, Starý, J, Turkiewicz, D, Ussowicz, M, Kozyra, E, Albert, M, Bader, P, Bordon, V, Cario, G, Beier, R, Schulte, J, Bresters, D, Müller, I, Pichler, H, Sedlacek, P, Sauer, MG, Zecca, M, Göhring, G, Yoshimi, A, Noellke, P, Erlacher, M, Locatelli, F, Niemeyer, CM & Strahm, B 2021, 'Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome', BONE MARROW TRANSPL, Jg. 56, Nr. 11, S. 2732-2741. https://doi.org/10.1038/s41409-021-01374-y

APA

Bortnick, R., Wlodarski, M., de Haas, V., De Moerloose, B., Dworzak, M., Hasle, H., Masetti, R., Starý, J., Turkiewicz, D., Ussowicz, M., Kozyra, E., Albert, M., Bader, P., Bordon, V., Cario, G., Beier, R., Schulte, J., Bresters, D., Müller, I., ... Strahm, B. (2021). Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome. BONE MARROW TRANSPL, 56(11), 2732-2741. https://doi.org/10.1038/s41409-021-01374-y

Vancouver

Bortnick R, Wlodarski M, de Haas V, De Moerloose B, Dworzak M, Hasle H et al. Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome. BONE MARROW TRANSPL. 2021 Nov;56(11):2732-2741. https://doi.org/10.1038/s41409-021-01374-y

Bibtex

@article{ce81f72b06104b22b141dda511b4fd16,
title = "Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome",
abstract = "GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germline GATA2 mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the probability of overall survival and disease-free survival (DFS) was 75% and 70%, respectively. Non-relapse mortality and relapse equally contributed to treatment failure. There was no evidence of increased incidence of graft-versus-host-disease or excessive rates of infections or organ toxicities. Advanced disease and monosomy 7 (-7) were associated with worse outcome. Patients with refractory cytopenia of childhood (RCC) and normal karyotype showed an excellent outcome (DFS 90%) compared to RCC and -7 (DFS 67%). Comparing outcome of GATA2mut with GATA2wt patients, there was no difference in DFS in patients with RCC and normal karyotype. The same was true for patients with -7 across morphological subtypes. We demonstrate that HSCT outcome is independent of GATA2 germline mutations in pediatric MDS suggesting the application of standard MDS algorithms and protocols. Our data support considering HSCT early in the course of GATA2 deficiency in young individuals.",
author = "Rachel Bortnick and Marcin Wlodarski and {de Haas}, Valerie and {De Moerloose}, Barbara and Michael Dworzak and Henrik Hasle and Riccardo Masetti and Jan Star{\'y} and Dominik Turkiewicz and Marek Ussowicz and Emilia Kozyra and Michael Albert and Peter Bader and Victoria Bordon and Gunnar Cario and Rita Beier and Johannes Schulte and Dorine Bresters and Ingo M{\"u}ller and Herbert Pichler and Petr Sedlacek and Sauer, {Martin G} and Marco Zecca and Gudrun G{\"o}hring and Ayami Yoshimi and Peter Noellke and Miriam Erlacher and Franco Locatelli and Niemeyer, {Charlotte M} and Brigitte Strahm",
note = "{\textcopyright} 2021. The Author(s).",
year = "2021",
month = nov,
doi = "10.1038/s41409-021-01374-y",
language = "English",
volume = "56",
pages = "2732--2741",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "11",

}

RIS

TY - JOUR

T1 - Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome

AU - Bortnick, Rachel

AU - Wlodarski, Marcin

AU - de Haas, Valerie

AU - De Moerloose, Barbara

AU - Dworzak, Michael

AU - Hasle, Henrik

AU - Masetti, Riccardo

AU - Starý, Jan

AU - Turkiewicz, Dominik

AU - Ussowicz, Marek

AU - Kozyra, Emilia

AU - Albert, Michael

AU - Bader, Peter

AU - Bordon, Victoria

AU - Cario, Gunnar

AU - Beier, Rita

AU - Schulte, Johannes

AU - Bresters, Dorine

AU - Müller, Ingo

AU - Pichler, Herbert

AU - Sedlacek, Petr

AU - Sauer, Martin G

AU - Zecca, Marco

AU - Göhring, Gudrun

AU - Yoshimi, Ayami

AU - Noellke, Peter

AU - Erlacher, Miriam

AU - Locatelli, Franco

AU - Niemeyer, Charlotte M

AU - Strahm, Brigitte

N1 - © 2021. The Author(s).

PY - 2021/11

Y1 - 2021/11

N2 - GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germline GATA2 mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the probability of overall survival and disease-free survival (DFS) was 75% and 70%, respectively. Non-relapse mortality and relapse equally contributed to treatment failure. There was no evidence of increased incidence of graft-versus-host-disease or excessive rates of infections or organ toxicities. Advanced disease and monosomy 7 (-7) were associated with worse outcome. Patients with refractory cytopenia of childhood (RCC) and normal karyotype showed an excellent outcome (DFS 90%) compared to RCC and -7 (DFS 67%). Comparing outcome of GATA2mut with GATA2wt patients, there was no difference in DFS in patients with RCC and normal karyotype. The same was true for patients with -7 across morphological subtypes. We demonstrate that HSCT outcome is independent of GATA2 germline mutations in pediatric MDS suggesting the application of standard MDS algorithms and protocols. Our data support considering HSCT early in the course of GATA2 deficiency in young individuals.

AB - GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We analyzed the outcome of 65 patients reported to the registry of the European Working Group (EWOG) of MDS in childhood carrying a germline GATA2 mutation (GATA2mut) who had undergone hematopoietic stem cell transplantation (HSCT). At 5 years the probability of overall survival and disease-free survival (DFS) was 75% and 70%, respectively. Non-relapse mortality and relapse equally contributed to treatment failure. There was no evidence of increased incidence of graft-versus-host-disease or excessive rates of infections or organ toxicities. Advanced disease and monosomy 7 (-7) were associated with worse outcome. Patients with refractory cytopenia of childhood (RCC) and normal karyotype showed an excellent outcome (DFS 90%) compared to RCC and -7 (DFS 67%). Comparing outcome of GATA2mut with GATA2wt patients, there was no difference in DFS in patients with RCC and normal karyotype. The same was true for patients with -7 across morphological subtypes. We demonstrate that HSCT outcome is independent of GATA2 germline mutations in pediatric MDS suggesting the application of standard MDS algorithms and protocols. Our data support considering HSCT early in the course of GATA2 deficiency in young individuals.

U2 - 10.1038/s41409-021-01374-y

DO - 10.1038/s41409-021-01374-y

M3 - SCORING: Journal article

C2 - 34244664

VL - 56

SP - 2732

EP - 2741

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 11

ER -