Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP)

Background: Childhood ITP can have a negative impact on the child and his/her family even though it is typically a benign disorder with low risk of serious bleeding. In adults and now children, romiplostim increases the platelet count without significant adverse effects. In this study, the impact of romiplostim treatment on the HRQoL of children with chronic ITP was assessed using the Kid's ITP Tools (KIT). Procedure: Subjects 1-18 years old, with chronic ITP (>6 months), were enrolled in a multi-center, randomized, double-blind, placebo-controlled phase 1/2 treatment study with romiplostim (reported elsewhere). Subjects and/or proxies completed the KIT at baseline, week 5, and week 13. Scores were computed for child self-report (children >7 years), proxy-report, and parent impact. Changes in mean scores from baseline to week 13 were computed. Results: Twenty-two children (17 receiving romiplostim, 5 placebo) and/or their parents provided data. Change in mean scores demonstrated significant improvement in HRQoL for romiplostim versus placebo for parent impact (24±17 vs. -6±8; P=0.008). Change scores for child self-report trended toward improvement with romiplostim and decreased with placebo (5±10 vs. -7±17; P=0.29). Romiplostim proxy-report mean change scores were 6 points higher than placebo (8±16 vs. 2±12; P=0.50). Conclusions: Romiplostim significantly reduced parental burden in this study. Whether the same and/or additional improvements in HRQoL would be demonstrated by a larger, longer study of romiplostim-treated children with ITP remains to be determined.