Parenteral Treprostinil Shows Efficacy in Pediatric PAH, Meta-Analysis Confirms

BACKGROUND: Paediatric pulmonary arterial hypertension (PAH) shares commonalities with adult disease but is essentially different regarding complexity and is usually more challenging to treat. Current treatment recommendations are based on expert opinion, small-scale paediatric studies and knowledge and consolidated guidelines for adults. Parenteral prostacyclins are recommended for high-risk patients but evidence is limited to cohort studies and retrospective data evaluations. The aim of this article was to summarise the available evidence on the efficacy and safety of parenteral treprostinil for paediatric PAH through a systematic review and to evaluate selected efficacy end-points through meta-analysis. METHOD: A systematic literature search (January 2000-April 2024) was conducted in PubMed, Google Scholar and clinical trial registries. Eligible studies included those reporting long-term outcomes of parenteral treprostinil in children with PAH. Moreover, a meta-analysis of selected efficacy end-points was performed based on published results from studies meeting predefined criteria. RESULTS: 32 studies encompassing 766 paediatric PAH patients treated with parenteral prostacyclins were identified; 649 patients received treprostinil. The meta-analysis was based on five publications including a total of 143 treprostinil-naïve patients. Despite the lack of randomised controlled trials, available data clearly indicate a treatment benefit of parenteral treprostinil in paediatric PAH. Literature data are supported by statistically significant results in the meta-analysis for PAH-relevant efficacy end-points. CONCLUSION: Based on currently available published data, parenteral treprostinil is effective and safe in the treatment of paediatric PAH.