> Details
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];
[...]
Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms
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- Media type: E-Article
- Title: 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
- Contributor: Kubasch, Anne Sophie [VerfasserIn]; Giagounidis, Aristoteles [VerfasserIn]; Metzgeroth, Georgia [VerfasserIn]; Jonasova, Anna [VerfasserIn]; Herbst, Regina [VerfasserIn]; Diaz, Jose Miguel Torregrosa [VerfasserIn]; De Renzis, Benoit [VerfasserIn]; Götze, Katharina [VerfasserIn]; Huetter-Kroenke, Marie-Luise [VerfasserIn]; Gourin, Marie-Pierre [VerfasserIn]; Slama, Borhane [VerfasserIn]; Dimicoli-Salazar, Sophie [VerfasserIn]; Cony-Makhoul, Pascale [VerfasserIn]; Laribi, Kamel [VerfasserIn]; Park, Sophie [VerfasserIn]; Jersemann, Katja [VerfasserIn]; Schipp, Dorothea [VerfasserIn]; Metzeler, Klaus H. [VerfasserIn]; Tiebel, Oliver [VerfasserIn]; Sockel, Katja [VerfasserIn]; Gloaguen, Silke [VerfasserIn]; Mies, Anna [VerfasserIn]; Chermat, Fatiha [VerfasserIn]; Thiede, Christian [VerfasserIn]; Sapena, Rosa [VerfasserIn]; Schlenk, Richard Friedrich [VerfasserIn]; Fenaux, Pierre [VerfasserIn]; Platzbecker, Uwe [VerfasserIn]; Adès, Lionel [VerfasserIn]
-
imprint:
07 September 2022
- Published in: Leukemia ; 36(2022), 10, Seite 2519-2527
- Language: English
- DOI: 10.1038/s41375-022-01669-z
- ISSN: 1476-5551
- Identifier:
- Keywords: Haematological diseases ; Translational research
- Origination:
- Footnote:
- Description: 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.
- Access State: Open Access