Background Veletri®, a new epoprostenol formulation, provides better drug stability and improved
clinical use compared to previous epoprostenol formulations. This study aims to expand
clinical experience in the use of Veletri®, especially with regard to tolerability,
safety and survival.
Method Pulmonary arterial hypertension (PAH) patients at high risk despite double oral combination
therapy were consecutively included in this prospective, open label, observational,
non-interventional study. Clinical data were assessed at baseline, after 3 and 6 months.
Adverse events (AE´s) and serious adverse events (SAE´s) were documented. Survival
of all patients was assessed at the end of study.
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±8.9 mmHg, mean
pulmonary vascular resistance 4.4±0.7 (median 3.8) Wood Units) 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, seven patients died and three 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 Veletri®. The 1- and 2-year
survival rate was 73.3% and 52.4%, respectively.
Conclusion The study showed that safety and tolerability of epoprostenol (Veletri®) was comparable
to previous prostacyclin formulations and was feasible in most patients. The clinically
determined maximum tolerable dosage was lower than dosages reported in the literature.
Survival was as expected in these severely impaired patients.
