Uptravi improves physical capacity in children with PAH: Study
Add-on treatment may reduce measure of blood pressure in pulmonary arteries
Uptravi (selexipag) may reduce blood pressure in the lungs of children with pulmonary arterial hypertension (PAH) and help them perform physical tasks, according to a meta-analysis. However, more long-term studies are still needed to confirm these benefits.
The study, “Efficacy and Tolerability of Selexipag in Pediatric Patients with Pulmonary Arterial Hypertension: An Updated Systematic Review and Meta-analysis,” was led by researchers in Greece and published in Pediatric Cardiology.
Uptravi mimics molecule that regulates blood pressure
PAH occurs when the blood vessels in the lungs become narrowed, making it difficult for blood to flow through them and causing high blood pressure in the pulmonary arteries, which carry blood from the heart to the lungs. Over time, high blood pressure puts a strain on the right side of the heart, leading to complications.
Marketed by Johnson & Johnson, Uptravi mimics prostacyclin, a naturally occurring molecule that regulates blood pressure by widening the blood vessels. Uptravi also widens the blood vessels, relieving the abnormally high blood pressure and easing symptoms.
Its use is approved to delay how fast the disease progresses and reduce the risk of hospitalization, but evidence for using Uptravi in children with PAH is still limited. Most existing studies on children are small and vary significantly.
“Its oral administration, longer half-life, and selective action profile make it particularly attractive for chronic management. However, its use in children is off-label, and evidence remains limited,” the researchers wrote, “leaving critical gaps regarding its tolerability and therapeutic benefit in pediatric patients.” Half-life refers to the time needed for a therapy’s initial levels to drop by half.
Treatment helped children walk significantly farther
To learn more, the researchers combined the available evidence in a meta-analysis, a method that pools data from multiple studies. They included 12 studies involving 344 children and adolescents ranging in age from 7.4 to 18.4 years. Most (90.6%) had a confirmed diagnosis of PAH.
One of the main goals was to monitor changes in the six-minute walk distance, which measures how far a patient can walk in six minutes, reflecting physical capacity. While the results varied across studies, Uptravi overall helped children walk significantly farther after treatment.
Combining data from four studies involving 106 children with PAH, the researchers showed that add-on treatment with Uptravi significantly reduced mean pulmonary artery pressure, a measure of blood pressure in the pulmonary arteries. However, it did not alter pulmonary vascular resistance index or NT-proBNP, a marker of heart stress, among the overall study population.
Nearly two-thirds of patients (62.8%) had side effects, but most were mild. Side effects included vomiting, digestive upset, jaw pain, diarrhea, and nausea. Children receiving Uptravi as an add-on treatment to their standard medications exhibited more physical improvement but also more side effects.
Overall, Uptravi appears to help improve physical capacity and may reduce pulmonary artery pressure in children with PAH. It was generally well tolerated, with mostly mild side effects. Still, the researchers emphasize that larger, long-term, well-designed studies are needed to confirm these findings.
Uptravi “represents a valuable option for managing pediatric PAH,” they concluded, adding that “further prospective studies are needed to confirm long-term benefits.”
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