Romiplostim Gets a Thumbs Up for Children with Thrombocytopenia

Romiplostim has been indicated for adults with chronic immune thrombocytopenia (ITP), but what about for kids? A phase three study examined the notion during a presentation at the 57^th American Society of Hematology Annual Meeting (ASH 2015) in Orlando, Florida.

Previous studies have determined that symptomatic ITP negatively impacts the health-related quality of life in children. Not only that, but it also tends to increase parental burden. Susan Mathias, MPH, from Health Outcomes Solutions, and colleagues evaluated if romiplostim could positively change both of those points. In addition, they aimed to analyze the drug’s influence on platelet response.

To uncover the answers, the team used the Kids’ ITP Tool (KIT) which was designed to measure health-related quality of life in children with ITP and assess the burden of the condition on parents. There are three versions of the tool which each have 26 items with a single final score of zero to 100, Mathias explained during the ASH 2015 presentation. The first is the Child Self-Report version for children who are at least 7-years-old. The Parent Proxy version is for parents to fill out if their child is younger than 7. The third kind is the Parent Impact version which is to be completed by all parents so that they can describe their child’s activity and disease outcomes, and how that burdens their lives.

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Using a cohort of 62 patients younger than 18, the researchers randomized them to receive either weekly romipolostim or placebo for 24 weeks. All of the children, 44% of which were male and an average age of 9.6, had been living with diagnosed ITP for at least six months. Forty-two patients received the romipolostim treatment while the other 20 had the placebo. The KIT was administered at baseline, as well as at week 8, 16, and 24.

“It appears that all scores were uniformly higher,” Mathias explained about the romipolostim group based on the Child Self-Report results. The medication showed improvement from baseline to all three points of measure. Scores for parental burden were also much better at each of the three points when compared to baseline. The placebo only showed significant improvement from baseline to eight weeks.

“So in general, based on unadjusted scores, we saw numerically greater improvements from baseline to each assessment for children receiving romiplostim versus the placebo,” Mathias summed up. However, a notable amount of parents did not complete the Parent Impact version of the KIT.

Out of all the entire population, 55% (33 patients) achieved overall platelet response and 39% (24 patients) achieved durable platelet response.

“To conclude, we found that treatment with romiplostim may be associated with reduced parents’ burden,” Mathias said.

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