EUROPE Study Investigates Biomarker-Driven Response Prediction Model to Romiplostim in Lower-Risk MDS

A prospective study failed to confirm the general predictive value of a biomarker-driven prediction model of the response to single-agent romiplostim in patients with lower-risk myelodysplastic neoplasms and thrombocytopenia, although it concluded that romiplostim is safe and highly efficacious in a large subset of patients.

The results were presented in an article published in Leukemia by Anne Sophie Kubasch, MD, of the Department of Hematology, Cellular Therapy, and Hemostaseology at Leipzig University in Germany, and colleagues.

The phase II EUROPE trial assessed a cohort of patients from the European Myelodysplastic Neoplasms Cooperative Group network.

A total of 77 patients with lower-risk myelodysplastic neoplasms and a median platelet count of 25/nL were included at 29 trial sites in Germany, France, and the Czech Republic. All patients received romiplostim at a starting dose of 750 μg by subcutaneous injection weekly. Thirty-two patients (42%) achieved a hematologic improvement of platelets (HI-P), with a median duration of 340 days. Neutrophil and erythroid responses were observed in three (4%) and seven (9%) patients, respectively.

The baseline variables used to predict HI-P versus non-HI-P by logistic regression included age, disease duration, hemoglobin, neutrophil count, platelet count, thrombopoietin level, sex, cytogenetics, World Health Organization classification, number of prior platelet transfusion units, and cytopenia.

Prior predictors of romiplostim response, namely lower baseline thrombopoietin levels and limited platelet transfusion events, were not validated in the EUROPE study. However, the study found that HI-P responses did correlate with baseline hemoglobin levels and the presence of SRSF2 mutations.

“Further larger, prospective, and controlled studies are warranted to specify the definitive role of thrombopoietin receptor agonists in the treatment landscape of [lower-risk myelodysplastic neoplasms],” the investigators wrote.

Reference

Kubasch AS, Giagounidis A, Metzgeroth G, et al. Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO. Leukemia. 2022;36(10):2519-2527.