Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms - results of the EUROPE trial by EMSCO.
Journal
Leukemia
ISSN: 1476-5551
Titre abrégé: Leukemia
Pays: England
ID NLM: 8704895
Informations de publication
Date de publication:
10 2022
10 2022
Historique:
received:
18
02
2022
accepted:
25
07
2022
revised:
18
06
2022
pubmed:
8
9
2022
medline:
4
10
2022
entrez:
7
9
2022
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 36071100
doi: 10.1038/s41375-022-01669-z
pii: 10.1038/s41375-022-01669-z
pmc: PMC9522582
doi:
Substances chimiques
Biomarkers
0
Hemoglobins
0
Receptors, Fc
0
Recombinant Fusion Proteins
0
Thrombopoietin
9014-42-0
romiplostim
GN5XU2DXKV
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2519-2527Informations de copyright
© 2022. The Author(s).
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