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

What Was Tested

This registered phase 2 trial tested Romiplostim.

There are currently no licensed drugs in the EU to treat thrombocytopenia in MDS patients classified as IPSS low/int-1.

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.

It had three arms (groups): Group 1 (experimental), Group 2 (experimental) and Group 3 (experimental).

Stratification into group 1 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are +3 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014.

Stratification into group 2 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -1 or -2 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014.

Stratification into group 3 if Score (Baseline-TPO Level and previous thrombocyte transfusions) are -6 TPO based model to predict subsequent response to romiplostim in MDS patients with IPSS Low/Int-1 according to a model developed by Sekeres et. al./ BJH 2014.

The registry classes the interventions as drug.

More on the Plan

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).

Who Took Part

77 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Subjects must meet the following inclusion/.

The full record lists 1 inclusion and 31 exclusion criteria; among the exclusions: must understand and voluntarily sign the informed consent form; age older 18 years at the time of signing the informed consent form; must be able to adhere to the study visit schedule and other protocol requirements.

How the Study Was Run

It was run as a non-randomized, parallel-group and open-label study.

The Romiplostim group and the comparison group ran side by side rather than switching.

Being open-label, the Romiplostim study did not mask who received what.

It started in 2015-05-21 with completion listed for 2021-07-01.

It ran at 36 sites in France (22) and Germany (14).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 6 secondary outcomes, for example The Incidence of Disease Progression to Higher Stage MDS or AML, Association of the Presence of Certain Mutations With Disease Progression in a Retrospective Analysis and Incidence of Bleeding Events.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of Romiplostim in 77 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Romiplostim compound page, all human trials.