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Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms: results of the EUROPE trial by EMSCO : myelodysplastic neoplasms

The EUROPE phase 2 trial investigated the predictive value of biomarkers on the clinical efficacy of single agent romiplostim (ROM) treatment in patients with lower-risk myelodysplastic neoplasms (LR-MDS) and thrombocytopenia within the ‘European Myelodysplastic Neoplasms Cooperative Group‘ (EMSCO)...

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Main Authors: Kubasch, Anne Sophie (Author) , Giagounidis, Aristoteles (Author) , Metzgeroth, Georgia (Author) , Jonasova, Anna (Author) , Herbst, Regina (Author) , Diaz, Jose Miguel Torregrosa (Author) , De Renzis, Benoit (Author) , Götze, Katharina (Author) , Huetter-Kroenke, Marie-Luise (Author) , Gourin, Marie-Pierre (Author) , Slama, Borhane (Author) , Dimicoli-Salazar, Sophie (Author) , Cony-Makhoul, Pascale (Author) , Laribi, Kamel (Author) , Park, Sophie (Author) , Jersemann, Katja (Author) , Schipp, Dorothea (Author) , Metzeler, Klaus H. (Author) , Tiebel, Oliver (Author) , Sockel, Katja (Author) , Gloaguen, Silke (Author) , Mies, Anna (Author) , Chermat, Fatiha (Author) , Thiede, Christian (Author) , Sapena, Rosa (Author) , Schlenk, Richard Friedrich (Author) , Fenaux, Pierre (Author) , Platzbecker, Uwe (Author) , Adès, Lionel (Author)
Format: Article (Journal)
Language:English
Published: 07 September 2022
In: Leukemia
Year: 2022, Volume: 36, Issue: 10, Pages: 2519-2527
ISSN:1476-5551
DOI:10.1038/s41375-022-01669-z
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1038/s41375-022-01669-z
Verlag, kostenfrei, Volltext: http://www.nature.com/articles/s41375-022-01669-z
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Author Notes:Anne Sophie Kubasch, Aristoteles Giagounidis, Georgia Metzgeroth, Anna Jonasova, Regina Herbst, Jose Miguel Torregrosa Diaz, Benoit De Renzis, Katharina S. Götze, Marie-Luise Huetter-Kroenke, Marie-Pierre Gourin, Borhane Slama, Sophie Dimicoli-Salazar, Pascale Cony-Makhoul, Kamel Laribi, Sophie Park, Katja Jersemann, Dorothea Schipp, Klaus H. Metzeler, Oliver Tiebel, Katja Sockel, Silke Gloaguen, Anna Mies, Fatiha Chermat, Christian Thiede, Rosa Sapena, Richard F. Schlenk, Pierre Fenaux, Uwe Platzbecker and Lionel Adès
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Summary:The EUROPE phase 2 trial investigated the predictive value of biomarkers on the clinical efficacy of single agent romiplostim (ROM) treatment in patients with lower-risk myelodysplastic neoplasms (LR-MDS) and thrombocytopenia within the ‘European Myelodysplastic Neoplasms Cooperative Group‘ (EMSCO) network. A total of 77 patients with LR-MDS and a median platelet count of 25/nl were included, all patients received ROM at a starting dose of 750 μg by SC injection weekly. Thirty-two patients (42%) achieved a hematologic improvement of platelets (HI-P) with a median duration of 340 days. Neutrophil (HI-N) and erythroid (HI-E) responses were observed in three (4%) and seven (9%) patients, respectively. We could not confirm previous reports that HI-P correlated with baseline endogenous thrombopoietin levels and platelet transfusion history, but SRSF2 mutation status and hemoglobin levels at baseline were significantly linked to HI-P. Sequential analysis of variant allelic frequency of mutations like SRSF2 did not reveal an impact of ROM on clonal evolution in both responders and non-responders. In summary, our study confirms the safety and efficacy of ROM in LR-MDS patients and may allow to better define subgroups of patients with a high likelihood of response.
Item Description:Gesehen am 14.08.2023
Physical Description:Online Resource
ISSN:1476-5551
DOI:10.1038/s41375-022-01669-z

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