Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study
Abstract Background Epoprostenol AS (Veletri®), a thermostable 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 regarding tolerability, s...
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| Format: | Article |
| Language: | English |
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BMC
2023-01-01
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| Series: | Respiratory Research |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12931-022-02296-z |
| _version_ | 1797945747261358080 |
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| author | Julia Degering Benjamin Egenlauf Satenik Harutyunova Nicola Benjamin Amina Salkić Panagiota Xanthouli Christina A. Eichstaedt Rebekka Seeger Olivier Sitbon Ekkehard Grünig |
| author_facet | Julia Degering Benjamin Egenlauf Satenik Harutyunova Nicola Benjamin Amina Salkić Panagiota Xanthouli Christina A. Eichstaedt Rebekka Seeger Olivier Sitbon Ekkehard Grünig |
| author_sort | Julia Degering |
| collection | DOAJ |
| description | Abstract Background Epoprostenol AS (Veletri®), a thermostable 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 regarding tolerability, safety and survival. Methods Pulmonary arterial hypertension (PAH) patients at high risk despite pretreatment with at least double oral combination therapy and with clinical indication for epoprostenol (Veletri®) treatment 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 (AEs) and serious adverse events (SAEs) were documented. Survival from initiation of Veletri® was assessed at last patient out. Results Fifteen patients (60 ± 13.7 years, WHO functional class III (n = 10) or IV (n = 5), severely impaired right ventricular 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. Eleven patients completed the study (treatment withdrawal n = 1, 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 rates were 73.3% and 52.4%, respectively. Conclusions The study showed that safety and tolerability of epoprostenol AS (Veletri®) was comparable to previous prostacyclin formulations and was feasible for most patients. The maximum tolerable dosage was lower than dosages reported in the literature. In future applications/trials the up-titration process should be pushing for higher dosages of epoprostenol in the occurrence of side effects, as the achievement of a high and effective dosage is crucial for the clinical benefit of the patients. Survival was as expected in these prevalent severely impaired patients. Trial registration The study was registered in the EUPAS registry (EUPAS32492). |
| first_indexed | 2024-04-10T21:00:01Z |
| format | Article |
| id | doaj.art-856e097475d048c79383c6f372a7dd44 |
| institution | Directory Open Access Journal |
| issn | 1465-993X |
| language | English |
| last_indexed | 2024-04-10T21:00:01Z |
| publishDate | 2023-01-01 |
| publisher | BMC |
| record_format | Article |
| series | Respiratory Research |
| spelling | doaj.art-856e097475d048c79383c6f372a7dd442023-01-22T12:22:41ZengBMCRespiratory Research1465-993X2023-01-0124111010.1186/s12931-022-02296-zTolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional studyJulia Degering0Benjamin Egenlauf1Satenik Harutyunova2Nicola Benjamin3Amina Salkić4Panagiota Xanthouli5Christina A. Eichstaedt6Rebekka Seeger7Olivier Sitbon8Ekkehard Grünig9Centre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalDepartment of Respiratory Diseases, Bicêtre Hospital, Paris-Saclay UniversityCentre for Pulmonary Hypertension, Thoraxklinik Heidelberg gGmbH at Heidelberg University HospitalAbstract Background Epoprostenol AS (Veletri®), a thermostable 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 regarding tolerability, safety and survival. Methods Pulmonary arterial hypertension (PAH) patients at high risk despite pretreatment with at least double oral combination therapy and with clinical indication for epoprostenol (Veletri®) treatment 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 (AEs) and serious adverse events (SAEs) were documented. Survival from initiation of Veletri® was assessed at last patient out. Results Fifteen patients (60 ± 13.7 years, WHO functional class III (n = 10) or IV (n = 5), severely impaired right ventricular 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. Eleven patients completed the study (treatment withdrawal n = 1, 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 rates were 73.3% and 52.4%, respectively. Conclusions The study showed that safety and tolerability of epoprostenol AS (Veletri®) was comparable to previous prostacyclin formulations and was feasible for most patients. The maximum tolerable dosage was lower than dosages reported in the literature. In future applications/trials the up-titration process should be pushing for higher dosages of epoprostenol in the occurrence of side effects, as the achievement of a high and effective dosage is crucial for the clinical benefit of the patients. Survival was as expected in these prevalent severely impaired patients. Trial registration The study was registered in the EUPAS registry (EUPAS32492).https://doi.org/10.1186/s12931-022-02296-zEpoprostenolPulmonary hypertensionVeletri |
| spellingShingle | Julia Degering Benjamin Egenlauf Satenik Harutyunova Nicola Benjamin Amina Salkić Panagiota Xanthouli Christina A. Eichstaedt Rebekka Seeger Olivier Sitbon Ekkehard Grünig Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study Respiratory Research Epoprostenol Pulmonary hypertension Veletri |
| title | Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study |
| title_full | Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study |
| title_fullStr | Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study |
| title_full_unstemmed | Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study |
| title_short | Tolerability, safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol (Veletri®): a prospective, 6-months, open label, observational, non-interventional study |
| title_sort | tolerability safety and survival in patients with severe pulmonary arterial hypertension treated with intravenous epoprostenol veletri r a prospective 6 months open label observational non interventional study |
| topic | Epoprostenol Pulmonary hypertension Veletri |
| url | https://doi.org/10.1186/s12931-022-02296-z |
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