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Parenteral treprostinil in paediatric pulmonary arterial hypertension: a systematic review and meta-analysis

Julie Wacker1,2, Raphael Joye3,2, Maurice Beghetti3,2

  • 1Paediatric Cardiology Unit, Service of Pediatric Specialties, Department of Woman, Child and Adolescent Medicine, Geneva University Hospitals and Faculty of Medicine, University of Geneva, Geneva, Switzerland julie.wacker@hug.ch.

European Respiratory Review : an Official Journal of the European Respiratory Society
|March 25, 2026
PubMed

Insights

Parenteral treprostinil demonstrates efficacy and safety in treating pediatric pulmonary arterial hypertension (PAH). This systematic review and meta-analysis supports its use for this complex pediatric condition.

Area of Science:

  • Pediatric Cardiology
  • Pulmonary Hypertension Research
  • Pharmacological Treatments

Background:

  • Pediatric pulmonary arterial hypertension (PAH) presents unique complexities compared to adult forms, often requiring specialized treatment approaches.
  • Current pediatric PAH treatment relies on expert consensus and adult guidelines, with limited specific evidence for high-risk interventions like parenteral prostacyclins.
  • Parenteral prostacyclins are recommended for high-risk pediatric PAH patients, but evidence primarily stems from cohort and retrospective studies.

Purpose of the Study:

  • To systematically review and synthesize evidence on the efficacy and safety of parenteral treprostinil in pediatric PAH.
  • To conduct a meta-analysis evaluating key efficacy endpoints for parenteral treprostinil in pediatric PAH patients.

Main Methods:

  • A comprehensive literature search was performed across PubMed, Google Scholar, and clinical trial registries from January 2000 to April 2024.
  • Eligible studies included those reporting long-term outcomes of parenteral treprostinil in pediatric PAH patients.
  • A meta-analysis focused on specific efficacy endpoints using data from five relevant publications.

Main Results:

  • The review identified 32 studies involving 766 pediatric PAH patients treated with parenteral prostacyclins, with 649 receiving treprostinil.
  • The meta-analysis included 143 treprostinil-naïve patients, revealing statistically significant improvements in PAH-relevant efficacy endpoints.
  • Despite the absence of randomized controlled trials, the data collectively indicate a clear treatment benefit for parenteral treprostinil in pediatric PAH.

Conclusions:

  • Parenteral treprostinil is an effective treatment option for pediatric pulmonary arterial hypertension based on current published data.
  • The safety profile of parenteral treprostinil in pediatric PAH patients is supported by the reviewed literature.
  • Further research, potentially including randomized controlled trials, could further solidify these findings.
Abstract

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