Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2025 Aug 16;13(23):107279.
doi: 10.12998/wjcc.v13.i23.107279.

Off-label use of treprostinil in adult patients in clinical cases

Christina Mouratidou  1 , Efstathios T Pavlidis  2 , Georgios Katsanos  3 , Serafeim-Chrysovalantis Kotoulas  1 , Georgios Tsoulfas  3 , Eleni Mouloudi  1 , Ioannis A Taitzoglou  4 , Ioannis N Galanis  2 , Theodoros E Pavlidis  5
Affiliations
Review

Off-label use of treprostinil in adult patients in clinical cases

Christina Mouratidou et al. World J Clin Cases. .

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

PubMed Disclaimer

Conflict of interest statement

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.

Figures

Figure 1
Figure 1
Simplified mechanisms of actions of treprostinil. IP: Prostacyclin.
Figure 2
Figure 2
Schematic representation of iontophoretic treprostinil delivery.
See this image and copyright information in PMC

References

    1. Feldman J, Habib N, Fann J, Radosevich JJ. Treprostinil in the treatment of pulmonary arterial hypertension. Future Cardiol. 2020;16:547–558. - PubMed
    1. Maron BA, Abman SH, Elliott CG, Frantz RP, Hopper RK, Horn EM, Nicolls MR, Shlobin OA, Shah SJ, Kovacs G, Olschewski H, Rosenzweig EB. Pulmonary Arterial Hypertension: Diagnosis, Treatment, and Novel Advances. Am J Respir Crit Care Med. 2021;203:1472–1487. - PMC - PubMed
    1. Lindegaard Pedersen M, Krüger M, Grimm D, Infanger M, Wehland M. The prostacyclin analogue treprostinil in the treatment of pulmonary arterial hypertension. Basic Clin Pharmacol Toxicol. 2020;126:32–42. - PubMed
    1. Kumar P, Thudium E, Laliberte K, Zaccardelli D, Nelsen A. A Comprehensive Review of Treprostinil Pharmacokinetics via Four Routes of Administration. Clin Pharmacokinet. 2016;55:1495–1505. - PMC - PubMed
    1. Whittle BJ, Silverstein AM, Mottola DM, Clapp LH. Binding and activity of the prostacyclin receptor (IP) agonists, treprostinil and iloprost, at human prostanoid receptors: treprostinil is a potent DP1 and EP2 agonist. Biochem Pharmacol. 2012;84:68–75. - PubMed

LinkOut - more resources