Using ROMIPLOSTINE for Persistent Thrombocytopenia With Transfusion-dependent Patients Who Received Allogeneic Hematopoietic Stem Cell

What Was Tested

In this phase 1, phase 2 trial, researchers set out to test Romiplostim.

This is a Phase I/II multicenter study which aims to assess the toxicity profile of Romiplostim in patients with transfusion-dependent thrombocytopenia after allogeneic HSCT.

A total of 24 patients with transfusion-dependent thrombocytopenia after allogeneic HSCT will be included.

The main endpoint is the incidence and severity of adverse events.

It had 1 arm (groups): Romiplostim (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 24 planned participants were the target.

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 be ≥ 18 years, willing and able to sign informed consent. Prolonged (\> 2 months) transfusion-dependent thrombocytopenia. Screenings mean platelet count≤ 20 x giga/L or screenings mean platelet count ≤ 50 x giga/L with a history of bleeding. (ECOG) performance status of 0-2. Adequate liver function.

The full record lists 9 inclusion and 13 exclusion criteria; among the exclusions: relapse/progression of hematological malignancy (marrow examination required); non-controlled acute and/or chronic graft versus host disease (gvhd); active or uncontrolled infections.

How the Study Was Run

It was run as a single-group and open-label study.

Nothing was hidden in this open-label Romiplostim study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2013-11 with completion listed for 2015-12.

It ran at 1 site in France.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Adverse events (time frame: 12 months).

It also tracked 8 secondary outcomes, for example Durable platelet response after transplant:, Relapse rate, Graft versus host disease (GVHD), Non relapse mortality rate, number of platelet transfusions and Overall number of bleeding events.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2 trial of Romiplostim in 24 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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