Long-term outcome in pulmonary arterial hypertension patients treated with treprostinil.

Pulmonary arterial hypertension (PAH) is fatal if untreated. Intravenous epoprostenol improves exercise capacity and hemodynamics in PAH, and increases survival in idiopathic PAH (IPAH). To evaluate the effects of subcutaneous (SC) treprostinil, a longer acting prostacyclin analogue, followed by the addition of other PAH therapies if needed, we followed 860 PAH patients treated with SC treprostinil for up to 4 years.Survival is reported as Kaplan-Meier estimates; for 332 IPAH patients with baseline hemodynamics, observed survival is also compared with predicted survival, using the NIH formula.Of the total 860 patients, 199 (23%) discontinued due to AEs, 136 (16%) died, 117 (14%) discontinued due to deterioration, 29 (3%) withdrew consent and 11 (1%) underwent transplantation. 97 patients (11%) switched from SC treprostinil to an alternative prostacyclin analogue; bosentan was added in 105 patients (12%) and sildenafil in 25 patients (3%).Survival was 87%-68% over 1-4 years for all 860 patients and 88%-70% over 1-4 years with SC treprostinil monotherapy. For the IPAH subset with baseline hemodynamics (n=332), 91%-72% over 1-4 years; in contrast, predicted survival was 69%-38% over 1-4 years. The safety profile for long-term SC treprostinil was consistent with previous short-term trials with no unexpected AEs.