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

Abstract

BackgroundChronic immune thrombocytopenia (ITP) in children can negatively impact their health‐related quality of life (HRQoL) and impose a burden on their parents. This study sought to examine the effect of romiplostim on HRQoL and parental burden in children with primary ITP.ProcedureThis was a phase 3, randomized, double‐blind, placebo‐controlled study. Children aged <18 years with ITP ≥6 months were randomly assigned to receive romiplostim or placebo for 24 weeks. 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. Psychometric properties of the KIT were evaluated and the minimally important difference (MID) was estimated for different KIT versions.ResultsSixty‐two patients (42 romiplostim and 20 placebo) were enrolled. Changes in KIT scores by treatment group showed numerically greater and more often statistically significant improvements from baseline to each assessment for children receiving romiplostim versus placebo. Mixed‐effects analysis demonstrated statistically significantly greater reduction in parental burden from baseline in the romiplostim group versus placebo. Ranges for the MID were estimated as 9–13 points for the Child Self‐Report version and 11–13 points for the Parent Impact version.ConclusionsThe treatment with romiplostim may be associated with improved HRQoL in children with primary ITP and reduced burden to their parents.

Patterns and influences in health‐related quality of life in children with immune thrombocytopenia: A study from the Dallas ITP Cohort

“…studied HRQoL in 22 children with chronic ITP participating in a pilot study of romiplostim and observed that parents of children receiving romiplostim displayed significant improvement in parent burden compared with those whose children were receiving placebo. Recently, a randomized, double‐blind clinical trial on the effects of romiplostim on HRQoL in children whose ITP course was at least 6 months duration, revealed that disease burden improves in parents of children on romiplostim compared with placebo . Though prior studies have not found a correlation between platelet count and KIT scores, our findings support that higher platelet counts at 6 and 12 months from diagnosis correlate with higher parent KIT scores.…”

“…Recently, a randomized, double-blind clinical trial on the effects of romiplostim on HRQoL in children whose ITP course was at least 6 months duration, revealed that disease burden improves in parents of children on romiplostim compared with placebo. 18 Though prior studies have not found a correlation between platelet count and KIT scores, 4,7,10 our findings support that higher platelet counts at 6 and 12 months from diagnosis correlate with higher parent KIT scores. Mokhtar et al 9 recently published a cross-sectional study of HRQoL using the KIT in 80 Egyptian children with ITP and noted that platelet count had a weak positive correlation with parent reports.…”

Health‐related quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study

“…The KIT assesses worry and restrictions in activity in children with ITP, and has shown good reliability and validity (Klaassen et al , ). The KIT was used in a 24‐week, randomised, double‐blind study to evaluate HRQoL in children treated with romiplostim versus placebo (Mathias et al , ). Similar to our results during the randomised phase, changes in the Child Self‐Report and Parent Proxy KIT scores in the romiplostim trial did not significantly differ by treatment group.…”

“…Similar to our results during the randomised phase, changes in the Child Self‐Report and Parent Proxy KIT scores in the romiplostim trial did not significantly differ by treatment group. Additionally, in analyses of responders versus non‐responders, the KIT change scores showed more improved (but small) changes in scores in responders to romiplostim compared with non‐responders at most time points (Mathias et al , ).…”

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

“…Although parent proxy-reports suggested an improvement in HRQOL among those treated with romiplostim, no statistically significant difference was noted in child self-report scores or parent proxy-report scores by time point or by the treatment group. Mathias et al46 completed a similar assessment of HRQOL among children with chronic ITP by also embedding KIT assessments as part of an aforementioned randomized placebo-controlled study. In this study, a total of 62 children were assessed including 42 in the romiplostim group and 20 in the placebo group.…”