Efficacy of oral treprostinil for treating pulmonary arterial hypertension: a systematic review and meta-analysis.

Introduction: Pulmonary arterial hypertension (PAH) is a progressive condition characterized by elevated blood pressure in the pulmonary arteries, leading to significant morbidity and mortality. Treprostinil, a synthetic analogue of prostacyclin, has emerged as a potential treatment option.

Aim: The efficacy and safety of oral treprostinil in PAH patients remain subjects of ongoing research.

Methods: Comprehensive literature searches were performed across multiple databases for studies assessing the efficacy and/or safety of treprostinil in PAH patients. Quality assessment and statistical analysis were performed using the Cochrane Collaboration's tool and Comprehensive Meta-Analysis software, respectively.

Results: From 680 records identified, four studies met the inclusion criteria. These studies demonstrated that oral treprostinil significantly improved exercise capacity as measured by the 6-minute walk distance (6MWD), with a mean difference of 13.13 m in favor of treprostinil, despite high heterogeneity. The analysis also showed an increased risk of adverse events leading to discontinuation in the treprostinil group (OR = 4.39) but a protective effect against clinical worsening (OR = 0.554). No significant impact on mortality was observed.

Conclusions: Oral treprostinil offers a significant benefit in improving exercise capacity in PAH patients, with a potential role in delaying clinical worsening. However, the increased risk of adverse events necessitates careful patient management. These findings support the inclusion of oral treprostinil in the treatment strategy for PAH, underscoring the need for further research to optimize its use and understand its long-term impact on patient outcomes.Level of evidence: I.