Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®​): 6-months, open label, observational, non-interventional study

Background: Veletri®, as a new epoprostenol formulation, provides better drug stability and improved clinical use. This study aims to expand clinical experience with regard to tolerability, safety and survival. Method: Pulmonary arterial hypertension (PAH) patients at high risk despite double oral combination therapy were included in this prospective, open label, observational, non-interventional study. Clinical data were assessed at baseline, after 3 and 6 months and survival at the end of study. Adverse events (AEs) and serious AEs (SAEs) were documented. Results: Overall, 15 patients (60±13.7 years, WHO functional class III-IV, severely impaired right ventricular pump function, mean pulmonary arterial pressure 54.8±8.9mmHg, mean pulmonary vascular resistance 4.4±0.7 (median 3.8) WU) were enrolled and treated with a mean dosage of 7.9±3.9 (median 7.5) ng/kg/min. Ten patients completed the study (treatment withdrawal n=2, death n=3). After a mean follow-up of 19.1±13.5 (median 18.0) months, 7 patients died and 3 were listed for lung transplantation. Seven AEs (nausea n=3, diarrhea n=1, flushing n=2, headaches n=1) and three SAEs (catheter infection n=2, catheter occlusion n=1) were related to Veletriâ. The 1- and 2-year survival rate was 73.3% and 52.4%, respectively. Conclusion: The study showed that safety and tolerability of epoprostenol (Veletriâ) was comparable to previous prostacyclin formulations and was feasible in most patients. The clinically determined maximum tolerable dosage was lower than reported dosages. Survival was as expected in these severely impaired patients.