Improvement in non-invasive measurements seen after tadalafil in children with PAH
A positive trend in improvement was seen in children with pulmonary arterial hypertension (PAH) treated with tadalafil, according to results of a phase 3 randomized, double-blind placebo-controlled study.
In this study, pediatric patients with PAH received tadalafil 20 mg or 40 mg based on their weight (heavy-weight: ≥40 kg; middle-weight: ≥25 to <40 kg) or placebo orally once daily for 24 weeks. Because of challenges related to recruitment, the number of planned participants was changed from 134 to ≥34 patients. As a result, statistical significance testing was not performed between treatment groups.
A total of 35 patients received tadalafil and 18 received placebo. Demographics and baseline characteristics were similar between groups. The median age of participants was 14.2 years and 71.4% of patients were in the heavy-weight cohort.
Least square mean six-minute walk distance from baseline to week 24 increased in patients treated with tadalafil versus placebo (60.48 vs 36.60 meters). The placebo-adjusted mean difference was 23.88.
No new safety concerns with tadalafil treatment were found.
One patient from the treatment group and 1 patient from the placebo group discontinued participation in the study due to investigator’s reported clinical worsening.
Ivy D, Bonnet D, Berger R, et al; LVHV Study Group. Efficacy and safety of tadalafil in a pediatric population with pulmonary arterial hypertension: phase 3 randomized, double-blind placebo-controlled study. Pulm Circ. 2021;11(3):20458940211024955. doi: 10.1177/20458940211024955. PMID: 34234945; PMCID: PMC8226239.