Key result
Macitentan reduces PVRI to ~59% of baseline at 24 weeks in pediatric PAH.
Why the study?
Macitentan is approved for adult patients with PAH, but evidence in pediatric PAH patients is limited.
Does macitentan improve pulmonary vascular resistance index in Japanese pediatric patients with pulmonary arterial hypertension?
Does macitentan improve pulmonary vascular resistance index in Japanese pediatric patients with pulmonary arterial hypertension?
Effect estimate: Geometric mean fold change 59.43% (95% CI 32.02%-110.30%)
Hypothesis-generating for macitentan in Japanese pediatric PAH; leaves open confirmation in randomized trials.
Background: Pulmonary arterial hypertension (PAH) is characterized by a progressive increase in pulmonary arterial pressure (PAP) and pulmonary vascular resistance (PVR), imposing an increased workload on the right ventricle and ultimately leading to right heart failure. Macitentan is a potent dual endothelin receptor antagonist that blocks both endothelin receptor subtypes A and B, and is approved for adult patients with PAH, but evidence in pediatric PAH patients is limited.
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Inuzuka et al. (2026) studied Pulmonary Arterial Hypertension (n=7). Macitentan was evaluated on Geometric mean fold change in pulmonary vascular resistance index (PVRI) at Week 24 (Geometric mean fold change 59.43%, 95% CI 32.02%-110.30%). Macitentan achieved a clinically meaningful reduction in pulmonary vascular resistance index with a geometric mean fold change of 59.43% at 24 weeks in Japanese pediatric patients with pulmonary arterial hypertension.
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