Long-Term Mortality and Morbidity Impact on Patients with Pulmonary Arterial Hypertension (PAH) If Access to...

Pulmonary arterial hypertension (PAH) is a rare, progressive disease associated with significant morbidity and mortality. The phase 3 STELLAR trial tested sotatercept plus background therapy (BGT) versus placebo plus BGT, where BGT included mono-, double-, or triple-PAH targeted therapy. Building on the trial's findings, a population health model was recently published assessing the long-term clinical impact of sotatercept. This analysis expands on this model and compares the clinical outcomes of immediate treatment initiation with sotatercept plus BGT against delayed treatment initiation with sotatercept plus BGT using a six-state Markov-type model and over a lifetime horizon.

State-transition probabilities were obtained from STELLAR, while mortality rates adjusted for risk strata and probabilities of lung/heart-lung transplants were derived from COMPERA PAH registry data and literature.

In the base case, a 2-year delay in treatment with sotatercept plus BGT resulted in an average of 12.4 years life expectancy, whereas immediate initiation of sotatercept led to an average of 16.5 years, a difference of 4.1 years. Immediate treatment with sotatercept plus BGT was also associated with a gain in infused prostacyclin-free life-years and resulted in 210 PAH hospitalizations avoided and 5 lung/heart-lung transplant avoided per 1000 patients.

This research suggests that early addition of sotatercept to BGT has the potential to increase life expectancy among patients with PAH and to reduce PAH hospitalizations, prostacyclin-use, and lung/heart-lung transplants needs. Real-world data are needed to confirm these findings, guiding clinicians and healthcare decision-makers in optimizing PAH treatment strategies.

ClinicalTrials.gov identifier, NCT04576988 (STELLAR).