Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome

What Was Tested

This registered phase 2 trial tested Romiplostim.

It had two arms (groups): I (Romiplostim) (experimental) and II (Eltrombopag) (experimental).

The registry classes the interventions as drug.

More on the Plan

The patients will be randomized in a 1:1 fashion to receive either romiplostim or eltrombopag.

Bleeding severity will be evaluated with a modified World Health Organization (WHO) Bleeding Scale, lab tests (blood counts, biochemical analyses), ECG will be assessed after 30 days of treatment (30 +/- 7 days).

No response will be defined as not achieving a platelet count of \> 30 x 109/L from the baseline count.

Refractory patients will be switched to alternate TPO-RA agonist.

Who Took Part

The registry lists 30 planned participants were the target.

It was open to people of any sex aged up to 18 years.

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

Examples of who could join: Genetically verified Wiskott-Aldrich syndrome. Thrombocytopenia (platelet count \< 70 x 109/L). Age: under 18 years. Subject/legal representative has signed written informed consent.

The full record lists 4 inclusion and 5 exclusion criteria; among the exclusions: patients, who do not meet the inclusion criteria; any prior history of arterial or venous thrombosis within the past year; arm ii (eltrombopag):.

How the Study Was Run

Design: randomized, crossover and open-label.

Because people in this Romiplostim study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Each person switched between Romiplostim and the comparison at some point, usually with a gap in between.

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

It started in 2019-11-05 with completion listed for 2022-12.

It ran at 1 site in Russia.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Percentage of patients with overall platelet response (complete response + partial response) for romiplostim and eltrombopag group (time frame: 1 month (30 day +/- 7 days)).

It also tracked 3 secondary outcomes, for example Number of participants with bleeding events and severity of bleeding in romiplostim and eltrombopag group and Number of participants with drug related adverse events in each treatment group.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Romiplostim in 30 people.

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

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

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