NCT02335268

Validation of a Predictive Model of Response to Romiplostim in Patients With IPSS Low or Intermediate-1 Risk MDS and Thrombocytopenia

Prospective Validation of a Predictive Model of Response to Romiplostim in Patients With IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) and Thrombocytopenia - the EUROPE-trial

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 77 (actual)
• Sponsor: Gesellschaft fur Medizinische Innovation - Hamatologie und Onkologie mbH · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

There are currently no licensed drugs in the EU to treat thrombocytopenia in MDS patients classified as IPSS low/int-1. Prior studies with romiplostim (a TPO receptor agonist) in MDS found that baseline concentration of TPO as well as transfusion history were predictive of subsequent response in a retrospective model. The current prospective study has the aim to explore whether both pretreatment variables (endogenous TPO, TPO-level, platelet transfusion history) can predict the response to subsequent short-term treatment with romiplostim.

Detailed description

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematologic malignancies of the pluripotent hematopoietic stem cells characterized by clonal hematopoiesis, progressive bone marrow failure, and the propensity to transform to acute myeloid leukemia (AML) (Malcovati et al., 2013). There are currently no licensed drugs in the EU to treat thrombocytopenia in MDS patients classified as IPSS low/int-1. Prior studies with romiplostim (a TPO receptor agonist) in MDS found that baseline concentration of TPO as well as transfusion history were predictive of subsequent response in a retrospective model. Classically, MDS is associated with apoptosis and excessive proliferation, resulting in a combination of a hyper-cellular marrow and peripheral cytopenia. The rationale for using romiplostim in MDS is to stimulate normal progenitor cells to increase platelet counts. Upon correction of thrombocytopenia, responding MDS patients should have a decreased risk of bleeding and a reduction in platelet transfusions (Giagounidis et al., 2014). This reduction in platelet transfusions may in turn decrease the risks of alloimmunization and the resultant morbidity and costs associated with that condition.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	N-Plate / romiplostim	medical intervention in 3 patient groups (MDS patients with IPSS Low/Int-1) that are stratified according to their baseline TPO-Level and previous transfusions

Timeline

• Start date: 2015-05-21
• Primary completion: 2021-07-01
• Completion: 2021-07-01
• First posted: 2015-01-09
• Last updated: 2023-08-16
• Results posted: 2023-07-14

Locations

36 sites across 2 countries: France, Germany