Current epoprostenol use in patients with severe idiopathic, heritable or anorexigen-associated pulmonary arterial hypertension: Data from the French pulmonary hypertension registry

Abstract Objectives The current use of intravenous epoprostenol in patients with severe idiopathic, heritable or anorexigen-use associated pulmonary arterial hypertension (IHA-PAH) was investigated. Methods This observational study evaluated newly diagnosed (≤1year) patients with IHA-PAH, enrolled in the French pulmonary hypertension (PH) registry between 2006 and 2010 and treated with epoprostenol. Among 209 consecutive patients receiving epoprostenol for the treatment of severe PH, 78 had IHA-PAH, including 43 patients naive of previous PAH-specific treatment. Results After 4months of epoprostenol therapy, improvement was observed for treatment naive patients (n=43) and for patients who had received previous PAH-specific therapy (n=35): NYHA functional class improved in 79% and 44% of these patients, respectively, 6-minute walk distance increased by 146 (p −1 ·m −2 (p=0.006), and pulmonary vascular resistance decreased by 700 (p −5 (p=0.009). In the treatment-naive patient group, upfront combination of epoprostenol and oral PAH therapy tended to be more beneficial compared with epoprostenol monotherapy and was associated with improvement in cardiac index(p=0.03). The observed 1- and 3-year survival estimates from epoprostenol initiation were 84% and 69%, respectively. The highest survival rates were observed for treatment-naive patients receiving upfront combination of epoprostenol and oral PAH therapy (92% and 88% at 1 and 3years, respectively). Conclusions First-line therapy with epoprostenol, especially when combined with oral PAH treatment, was associated with a substantial improvement in clinical and hemodynamic status and favorable survival estimates in patients with severe IHA-PAH.