Minireviews
Copyright ©The Author(s) 2025. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Clin Cases. Aug 16, 2025; 13(23): 107279
Published online Aug 16, 2025. doi: 10.12998/wjcc.v13.i23.107279
Off-label use of treprostinil in adult patients in clinical cases
Christina Mouratidou, Efstathios T Pavlidis, Georgios Katsanos, Serafeim-Chrysovalantis Kotoulas, Georgios Tsoulfas, Eleni Mouloudi, Ioannis A Taitzoglou, Ioannis N Galanis, Theodoros E Pavlidis
Christina Mouratidou, Serafeim-Chrysovalantis Kotoulas, Eleni Mouloudi, Intensive Care Unit, Hippokration General Hospital, Thessaloniki 54642, Greece
Efstathios T Pavlidis, Ioannis N Galanis, Theodoros E Pavlidis, The 2nd Department of Propaedeutic Surgery, Hippokration General Hospital, School of Medicine, Aristotle University of Thessaloniki, Thessaloniki 54642, Greece
Georgios Katsanos, Georgios Tsoulfas, Department of Transplantation Surgery, Center for Research and Innovation in Solid Organ Transplantation, Aristotle University of Thessaloniki, School of Medicine, Thessaloniki 54642, Greece
Ioannis A Taitzoglou, Laboratory of Physiology, Faculty of Veterinary Medicine, Aristotle University of Thessaloniki, Thessaloniki 54124, Greece
Co-corresponding authors: Efstathios T Pavlidis and Theodoros E Pavlidis.
Author contributions: Pavlidis ET and Pavlidis TE contribute equally to this study as co-corresponding authors; Mouratidou C, Pavlidis ET, Mouloudi E, Taitzoglou IA, designed research and analysed data; Kotoulas SC, Tsoulfas G performed research contributed new analytic tools, evaluated data and review the paper; Katsanos G, Galanis IN contributed to data collection and analysed data; Pavlidis TE analysed data review and approved the paper.
Conflict-of-interest statement: There is no conflict of interest associated with any of the senior author or other coauthors contributed their efforts in this manuscript.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Theodoros E Pavlidis, MD, PhD, Professor, The 2nd Department of Propaedeutic Surgery, Hippokration General Hospital, School of Medicine, Aristotle University of Thessaloniki, Konstantinoupoleos 49, Thessaloniki 54642, Greece. pavlidth@auth.gr
Received: March 20, 2025
Revised: April 3, 2025
Accepted: May 7, 2025
Published online: August 16, 2025
Processing time: 77 Days and 2 Hours
Abstract

Treprostinil is a relatively new tricyclic prostacyclin analog with a stable structure, extended half-life and improved potency. Currently, treprostinil is indicated by the Food and Drug Administration in the United States for the treatment of pulmonary arterial hypertension (group 1 in the pulmonary hypertension classification of the World Health Organization). It has a potent vasodilating effect along with the inhibition of platelet aggregation and the attenuation of the inflammatory response in pulmonary and systemic circulation. It is available in the following formulations: Subcutaneous, intravenous, inhaled and oral. Although unknown to many clinicians, several encouraging reports of off-label treprostinil use in the adult population suggest its potential effectiveness in other clinical conditions. Currently under investigation are digital ischemia secondary to systemic sclerosis, chronic limb ischemia, hepatic ischemia-reperfusion injury and group 3 and 4 pulmonary hypertension. Based on review and analysis of the available literature, this article provides a thorough update on the off-label use of treprostinil in adult patients.

Keywords: Treprostinil; Off-label use; Pulmonary hypertension; Digital ischemia; Chronic limb ischemia; Hepatic ischemia–reperfusion injury

Core Tip: Treprostinil is a synthetic prostacyclin analog with a stable structure that is currently used for the treatment of pulmonary arterial hypertension. By way of vasodilation and the inhibition of platelet aggregation, treprostinil increases the blood supply in pulmonary circulation and reduces the cardiac workload. In addition to its main indication, several positive reports of off-label treprostinil use in the adult population indicate its benefit in other clinical conditions. Hepatic ischemia–reperfusion injury, chronic limb ischemia, digital ischemia and group 3 and 4 pulmonary hypertension are currently under study as possible indications for treprostinil use outside its approved labeling.