Background: Recent and ongoing developments in the diagnosis, treatment, and management of pulmonary arterial hypertension (PAH) provide deeper insights into pathogenic mechanisms. Approvals of new pharmacotherapies that improve function and reduce morbidity and mortality risks; advances in clinical trial methods, including long-term, event-driven studies with clinically relevant and patient-centered endpoints; and trial results support a new therapeutic management strategy. This new paradigm involves initial treatment with combined therapies that act through different disease pathways. In addition, 2 new sets of clinical practice guidelines for PAH have been published since June 2014. Despite these advances, major gaps have been documented in the diagnosis, treatment, and management of patients with PAH.
Objective: To present current knowledge and evidence on PAH to support managed care professionals and providers in achieving accurate differential diagnosis, promptly referring patients to specialists as necessary, and ensuring that patients receive appropriate, guideline-directed therapies.
Summary: Major gaps in the quality of care provided to patients with PAH include oversights in clinicians' recognition of symptoms, delays in diagnosis, and misdiagnosis ensuing from incomplete evaluations, delays in referral of patients to centers of expertise and initiation of therapy, and inappropriate treatment regimens. To address deficiencies in PAH diagnosis, new practice guidelines emphasize the essential role of right heart catheterization in characterizing and confirming the disease, as well as referral to expert pulmonary hypertension centers to ensure appropriate evaluation and treatment. Updated disease and functional classifications of PAH, along with new research findings on prognostic factors and effects of comorbid conditions, offer key support for making effective therapy and management decisions for patients with PAH at different risk levels and stages of the disease. Since 2013, the U.S. Food and Drug Administration has approved new PAH therapies in the classes of endothelin receptor antagonists, guanylate cyclase stimulators, prostacyclin analogues, and prostacyclin receptor agonists. As demonstrated through phase 3 clinical trials, these generally well-tolerated therapies delay disease progression, improve hemodynamic and functional status, and decrease numbers of hospitalizations. Moreover, 2 sets of recently published guidelines-developed by the American College of Chest Physicians and the European Society of Cardiology/European Respiratory Society-provide evidence-based and expert consensus recommendations for achieving PAH treatment goals. The most recent guidelines include a recommendation for upfront combination therapy for patients with moderate disease, which is supported by new comparative clinical trial evidence. As addressed in this article, these advances in the field of PAH have important implications for managed care and clinical practice, including considerations of cost-benefit outcomes associated with different management strategies.