Approved ITP treatment found effective, works well for both adults and children
Review of 9 trials shows injection therapy cuts need for rescue meds
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The approved injection therapy Nplate (romiplostim) is well tolerated and effective for both adults and children with immune thrombocytopenia (ITP), according to a new review of more than half a dozen clinical trials.
The data showed that treatment with Nplate significantly increased platelet counts — levels of the tiny cell fragments in blood that help form clots to stop bleeding — among the trials’ participants, particularly adults. The therapy’s use also reduced the need for emergency medications among the ITP patients, whose rare autoimmune disorder can lead to both internal and external bleeding.
“This meta-analysis of nine randomized controlled studies offers compelling evidence for the efficacy and safety of [Nplate] in” people with ITP, the researchers wrote. “Our results show that platelet counts improved significantly at several periods in time, … and that the need for rescue therapy was lower, all without a substantial increase in overall adverse events.”
The safety profile of Nplate was generally similar to that of placebos used in testing, the team noted.
According to the researchers, this study “provides directly applicable information for current clinical practice and may help physicians balance efficacy, safety, patient preference, and therapy accessibility when making individualized [treatment] decisions.”
The study, “Efficacy and Safety of Romiplostim Versus Placebo in Immune Thrombocytopenia: A Systematic Review and Meta-Analysis,” was published in the journal Clinical and Applied Thrombosis/Hemostasis.
ITP occurs when the immune system attacks and destroys platelets, leading to symptoms such as easy bruising and excessive bleeding.
First-line therapies for ITP, such as corticosteroids and intravenous immunoglobulin (IVIG), can provide initial benefits. However, many patients relapse or develop treatment-resistant disease.
Investigating Nplate use in children vs. adults
Nplate is used as a second-line therapy to stimulate platelet production. In clinical trials, it has been shown to increase platelet levels and reduce bleeding compared with a placebo or standard treatment. It’s also been found to have long-lasting effects.
However, there is limited information on whether the treatment has similar effects in children and adults with ITP, and also on comparisons between Nplate and a placebo.
To learn more, researchers from Pakistan and the Maldives conducted a systematic review and meta-analysis of clinical trials that enrolled both adults and children and compared Nplate with placebo or standard care. The review covered nine studies involving 748 patients. The Nplate and control groups had generally similar characteristics, the researchers noted.
Nplate was associated with significantly higher platelet counts than a placebo at all assessed time points, up to week 8 of treatment. The effects were generally larger in adults than children, although significant improvements were observed in both groups.
According to the researchers, these results “show that platelet recovery starts early and is maintained over time,” and provide “clinically significant information about the anticipated onset and durability of response following [Nplate] initiation.”
In addition, use of Nplate improved the likelihood of achieving a platelet response — an increase in platelet count by more than a set threshold — by about six times, a benefit seen in adults and children. However, some variability was seen between studies, the researchers noted.
“The substantial rise in platelet response rates reinforces the therapeutic efficacy of [Nplate],” the investigators wrote.
Nplate also increased the likelihood of achieving an increase in platelet counts of at least 20 × 10⁹/L above baseline, or the study’s start, compared with the placebo. Overall, patients receiving the treatment were more than three times as likely to reach this platelet increase, although the results varied considerably between studies, the data showed.
The benefit was significant in adults, while the increase in children did not initially reach statistical significance. However, after the researchers removed the study responsible for most of the variability, the treatment effect became stronger and statistically significant in both age groups.
Need for rescue meds cut by half compared with a placebo
Nplate also reduced the need for emergency (rescue) medications by about half compared with the placebo, the researchers found. This reduction was significant in adults but not in children, although the difference between the two age groups was not statistically significant.
“The overall drop in rescue therapy implies that [Nplate] may lower the burden of treatment and the use of healthcare,” the researchers wrote.
The team noted that, overall, Nplate had a safety profile similar to the placebo, with no significant differences in the rate of side effects, serious adverse events, or bleeding-related events. Infections — both of the upper respiratory tract infections and the common cold — neurological effects, and most gastrointestinal issues, such as diarrhea, nausea, and vomiting, were also similar between the groups.
Three side effects were significantly more common with Nplate: muscle pain, mouth and throat pain, and rash. However, those findings were based on a small number of cases and should therefore be interpreted with caution, according to the researchers.
“Long-term follow-up studies are necessary to assess enduring efficacy and postponed adverse effects,” the researchers wrote, adding that, “to improve treatment options and understand how different ages respond, more studies on children are needed.”
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