The myth of the stable pulmonary arterial hypertension patient.

Pulmonary arterial hypertension (PAH) is a rare, progressive disease that often leads to right heart failure and premature death. Despite increased awareness and an expanding treatment landscape in recent decades, long-term prognosis is poor for patients with PAH. Recently, emphasis has evolved from goal-oriented therapy to risk-assessment and achieving low-risk status. Findings from recent clinical trials suggest that functional class II patients, long assumed to be stable, are not stable. Therefore, frequent assessment of all patients with PAH is essential toward escalating treatment as indicated to optimize clinical outcomes. Lowering mortality risk, preventing disease progression, and optimizing quality of life of patients with PAH is paramount.

Combination therapy in pulmonary arterial hypertension: is this the new standard of care?

Pulmonary arterial hypertension (PAH) is a rare, progressive, and potentially fatal cardiopulmonary syndrome that imposes a significant burden on patients in terms of morbidity and mortality, and on managed care organizations in terms of resource utilization. The majority of PAH-approved therapies are high-touch, high-management, high-cost treatments dispensed through specialty pharmacies. Current treatment guidelines recommend combination therapy for patients who show inadequate clinical response or who deteriorate on monotherapy. Combination therapies target 2, or sometimes 3, distinct PAH-associated signaling pathways: the endothelin, prostacyclin, and nitric oxide pathways. Registry data suggest that combination therapy is utilized in more than half of patients with PAH in the United States. Evidence supporting the use of combination therapy is provided through clinical trials, retrospective research, registry data, and expert guidelines. Managed care decision makers are charged with making population-based decisions on resource allocation. These decision makers must always consider cost, but must also be aware that clinical evidence suggests that early treatment with combination therapy can significantly reduce disease burden, may reduce hospitalizations, and should be considered when making coverage decisions.