TY - JOUR
T1 - Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO
AU - Kubasch, Anne Sophie
AU - Giagounidis, Aristoteles
AU - Metzgeroth, Georgia
AU - Jonasova, Anna
AU - Herbst, Regina
AU - Diaz, Jose Miguel Torregrosa
AU - De Renzis, Benoit
AU - Götze, Katharina S.
AU - Huetter-Kroenke, Marie Luise
AU - Gourin, Marie Pierre
AU - Slama, Borhane
AU - Dimicoli-Salazar, Sophie
AU - Cony-Makhoul, Pascale
AU - Laribi, Kamel
AU - Park, Sophie
AU - Jersemann, Katja
AU - Schipp, Dorothea
AU - Metzeler, Klaus H.
AU - Tiebel, Oliver
AU - Sockel, Katja
AU - Gloaguen, Silke
AU - Mies, Anna
AU - Chermat, Fatiha
AU - Thiede, Christian
AU - Sapena, Rosa
AU - Schlenk, Richard F.
AU - Fenaux, Pierre
AU - Platzbecker, Uwe
AU - Adès, Lionel
N1 - Publisher Copyright:
© 2022, The Author(s).
PY - 2022/10
Y1 - 2022/10
N2 - 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.
AB - 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.
UR - https://www.scopus.com/pages/publications/85137517585
U2 - 10.1038/s41375-022-01669-z
DO - 10.1038/s41375-022-01669-z
M3 - Article
C2 - 36071100
AN - SCOPUS:85137517585
SN - 0887-6924
VL - 36
SP - 2519
EP - 2527
JO - Leukemia
JF - Leukemia
IS - 10
ER -
