Background: Treatment of pulmonary arterial hypertension (PAH) has advanced considerably decades with the advent of newer therapeutic medications and the associated improvement in 5-year survival. The annual pharmaceutical cost of medical therapy may range from $25000 to $250000 and is driven by newer agents and the proven efficacy of combination therapy. The disease and treatment burden for PAH patients are likely intertwined; therefore, we aimed to assess the psychosocial and healthcare implications of PAH therapy on patients.
Methods: The study was approved by the Mayo Clinic Institutional Review Board. Data was collected prospectively by questionnaire in patients with diagnostic group 1 PAH from 2 separate cohorts. Cohort 1 was comprised of consecutive patients from Pulmonary Hypertension Clinic at Mayo Clinic Florida (MCF). Cohort 2 was comprised of PAH patients who volunteered for research at 2018 Pulmonary Hypertension Association (PHA) International PH conference (Orlando, Florida). A 25-point questionnaire included demographic data, comorbidities, therapy regimens, quality of life, compliance, economic and social challenges of therapy including insurance and out-of-pocket expenses. Patients with group 1 PAH completed the questionnaire after consent. Exclusion criteria included age < 18 years, prior lung transplantation, absence of PAH therapy and clinical trial participants.
Results: Overall 112 patients, 60 in cohort 1 (MCF) and 52 in cohort 2 (PHA), completed the questionnaire. The majority were middle-aged, Caucasian women with either idiopathic (52/112, 46%) or connective tissue disease (28, 25%) associated PAH. Drug therapy spanned all classes with 74% (83/112) on combination therapy. Nonetheless, most patients (91/112, 81%) endorsed active cardiopulmonary symptoms including dissatisfaction with their level of activity despite their current PAH regimen. A minority (12/112, 11%) endorsed the ability to perform two hours of physical work without significant limitations. Participants reported a steady decline in employment rates at the time of diagnosis and with progression of disease with about ½ applying for disability. While most (108/112, 96%) had insurance coverage, out-of-pocket medication expenses were common and most reported that the drug costs were unaffordable without insurance or assistance programs. Statistically, cohort 1 was slightly older (62 ± 11 vs 51 ± 14, p<0.01), had more Caucasians (100% vs 86%, p=0.0007), and had different diagnostic classification (e.g. less idiopathic and heritable PAH, p<0.001, Table 1) but otherwise there was little difference in treatment, activity level, or psychosocial and financial burden.
Conclusion: Despite combination PAH therapy in most patients, a significant number of the study population reported that they had persistent cardiopulmonary symptoms, decreased activity levels, steady decline in employment rates and associated application for disability benefits. Most had insurance coverage but ongoing out-of-pocket medication expenses were common. Comparison of cohorts from a single center (MCF Cohort 1) to a more demographically heterogeneous group that attended the PHA conference (Cohort 2) did not reveal significant differences in disease or psychosocial and financial burden.