A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Determine the Effect of Romiplostim on Health-Related Quality of Life in Children with Primary Immune Thrombocytopenia and Associated Burden in Their Parents

What Was Tested

The paper reports a study of Romiplostim, published as a randomized controlled trial.

This study sought to examine the effect of romiplostim on HRQoL and parental burden in children with primary ITP.

Why It Was Studied

Chronic immune thrombocytopenia (ITP) in children can negatively impact their health-related quality of life (HRQoL) and impose a burden on their parents.

This was a phase 3, randomized, double-blind, placebo-controlled study.

Who Took Part

By PubMed's age tags, the people studied were young children, children and teenagers, both men and women.

How the Study Was Run

The researchers set it up as a randomized, double-blind and placebo-controlled study.

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.

As a double-blind Romiplostim study, the assignments were hidden from both participants and investigators until the end.

Some participants received a placebo instead of Romiplostim, giving a reference point.

Psychometric properties of the KIT were evaluated and the minimally important difference (MID) was estimated for different KIT versions.

What Was Measured

The Kids' ITP Tool (KIT) was used to measure HRQoL and was administered to patients and/or their parents at baseline and weeks 8, 16, and 25.

Mean KIT scores at each assessment and mean changes in KIT scores from baseline were calculated overall by treatment group and platelet response status.

The subject headings attached to it are Cost of Illness, Parents, Purpura, Thrombocytopenic, Idiopathic, Quality of Life, Receptors, Fc, Recombinant Fusion Proteins and Thrombopoietin.

The authors tagged it with the keywords health-related quality of life, kids’ ITP tools, minimally important difference, parental burden, pediatric immune thrombocytopenia and romiplostim.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Romiplostim.

Its findings take up roughly 5 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2016 (Pediatric blood & cancer), a journal based in United States.

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