Abstract Rationale Despite significant therapeutic advancements over the past three decades, pulmonary arterial hypertension (PAH) continues to impose a substantial burden on patients in the United States (US). Real-world data on current morbidity and mortality rates remain limited. Therefore, this study aims to quantify the contemporary burden of PAH—including hospitalization, re-hospitalization, and mortality—using US real-world data. Methods Adult (18+) patients with ≥1 claim for PAH treatment and ≥1 claim for right heart catheterization between 1/1/2017 and 8/31/2024 were selected from the Merative Marketscan Commercial and Medicare databases (earliest claim = index date). Patients were additionally required to have continuous enrollment (CE) with medical and pharmacy benefits for 12 months before (baseline period) and ≥30 days after index (follow-up period), and ≥1 inpatient or ≥ 2 outpatient claims with a pulmonary hypertension diagnosis during the baseline period. Those with evidence of erectile dysfunction, chronic thromboembolic pulmonary hypertension, or pregnancy/delivery were excluded. Sub-cohorts of newly-treated patients (those without PAH treatment at baseline) and PAH-hospitalized patients (those with ≥1 PAH-related hospitalization during the follow-up period and at least 30 days of follow-up post-hospitalization) were also analyzed. Outcomes included: demographics, clinical characteristics, all-cause mortality (for newly-treated sub-cohort), and hospitalization-related variables (e.g., principal diagnosis, length of stay, proportion involving intensive care unit ICU and mechanical ventilation; for PAH-hospitalized sub-cohort). Kaplan-Meier analysis was used to report time to mortality and time to re-hospitalization after accounting for censoring. Results Overall, 2,712 patients met the study criteria, including 2,072 (76.4%) newly-treated and 1,079 (39.8%) PAH-hospitalized. Patients averaged 62 years old and 69% were female. More than half (56.2%) had an all-cause hospitalization during an average 2.2 years of follow-up, with 44.9% having at least one PAH-related hospitalization. Among the PAH-hospitalized subset (average age 63 years; 67% female), patients averaged 1.5 PAH-related hospitalizations per year and the median time to re-hospitalization was 1.2 years. Of the PAH-related hospitalizations, 57.2% required an ICU stay and 9.8% required mechanical ventilation. Among the newly-treated subset (average age 63 years; 73% female), 9.7%, 19.6%, and 25.2% of patients died within 1, 2, and 3 years after treatment initiation. Conclusions Though much improved since no treatments were available 30 years ago, burden of disease for PAH persists, with high rates of hospitalization, re-hospitalization, and mortality. These findings highlight the unmet need for optimal management in PAH and the requirement to continuously monitor disease burden as progress is made with PAH therapies. This abstract is funded by: Johnson & Johnson
Ravichandran et al. (Fri,) studied this question.