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Autor
Albert, Michael 1 Bader, Peter 1 Beier, Rita 1 Bordon, Victoria 1 Bortnick, Rachel 1 Bresters, Dorine 1 Cario, Gunnar 1 De Moerloose, Barbara 1 Dworzak, Michael 1 Erlacher, Miriam 1 Göhring, Gudrun 1 Hasle, Henrik 1 Kozyra, Emilia 1 Locatelli, Franco 1 Masetti, Riccardo 1 Müller, Ingo 1 Niemeyer, Charlotte M 1 Noellke, Peter 1 Pichler, Herbert 1 Sauer, Martin G 1
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Pracoviště
Department for Children and Adolescen... 1 Department of Bone Marrow Transplanta... 1 Department of Hematology St Jude Chil... 1 Department of Human Genetics Hannover... 1 Department of Pediatric Hematology On... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Oncology Hema... 1 Department of Pediatric Oncology Hema... 1 Department of Pediatric Oncology and ... 1 Department of Pediatrics Aarhus Unive... 1 Department of Pediatrics Christian Al... 1 Department of Pediatrics Dr von Haune... 1 Department of Pediatrics St Anna Chil... 1 Department of Pediatrics University H... 1 Department of Pediatrics and Adolesce... 1 Department of Pediatrics and Adolesce... 1 German Cancer Consortium Heidelberg a... 1 Pediatric Hematology Oncology Fondazi... 1
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Autor
Albert, Michael 1 Bader, Peter 1 Beier, Rita 1 Bordon, Victoria 1 Bortnick, Rachel 1 Bresters, Dorine 1 Cario, Gunnar 1 De Moerloose, Barbara 1 Dworzak, Michael 1 Erlacher, Miriam 1 Göhring, Gudrun 1 Hasle, Henrik 1 Kozyra, Emilia 1 Locatelli, Franco 1 Masetti, Riccardo 1 Müller, Ingo 1 Niemeyer, Charlotte M 1 Noellke, Peter 1 Pichler, Herbert 1 Sauer, Martin G 1
- Organizace
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Pracoviště
Department for Children and Adolescen... 1 Department of Bone Marrow Transplanta... 1 Department of Hematology St Jude Chil... 1 Department of Human Genetics Hannover... 1 Department of Pediatric Hematology On... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Hematology an... 1 Department of Pediatric Oncology Hema... 1 Department of Pediatric Oncology Hema... 1 Department of Pediatric Oncology and ... 1 Department of Pediatrics Aarhus Unive... 1 Department of Pediatrics Christian Al... 1 Department of Pediatrics Dr von Haune... 1 Department of Pediatrics St Anna Chil... 1 Department of Pediatrics University H... 1 Department of Pediatrics and Adolesce... 1 Department of Pediatrics and Adolesce... 1 German Cancer Consortium Heidelberg a... 1 Pediatric Hematology Oncology Fondazi... 1
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Bortnick, Rachel
Autor Bortnick, Rachel Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg, Germany Department of Pediatrics, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria
- Wlodarski, Marcin
- de Haas, Valerie
- De Moerloose, Barbara
- Dworzak, Michael
- Hasle, Henrik
- Masetti, Riccardo
- Starý, Jan
- Turkiewicz, Dominik
- Ussowicz, Marek
NLK
Free Medical Journals
od 1997 do Před 1 rokem
Freely Accessible Science Journals
od 1997 do Před 1 rokem
ProQuest Central
od 2000-01-01 do Před 1 rokem
Open Access Digital Library
od 1997-01-01
Health & Medicine (ProQuest)
od 2000-01-01 do Před 1 rokem
PubMed
34244664
DOI
10.1038/s41409-021-01374-y
Knihovny.cz E-zdroje
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.
- MeSH
- chromozomální delece MeSH
- dítě MeSH
- lidé MeSH
- mladiství MeSH
- myelodysplastické syndromy * genetika terapie MeSH
- nemoc štěpu proti hostiteli * etiologie MeSH
- transkripční faktor GATA2 genetika MeSH
- transplantace hematopoetických kmenových buněk * metody MeSH
- zárodečné mutace MeSH
- Check Tag
- dítě MeSH
- lidé MeSH
- mladiství MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
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