Oral treprostinil use in children: a multicenter, observational experience

Area of Science:

• Pediatric pulmonology
• Pharmacology
• Cardiology

Background:

• Pulmonary arterial hypertension (PAH) is a severe, progressive disease affecting both children and adults.
• Limited therapeutic options exist for pediatric PAH, particularly for newer agents like oral treprostinil.
• Treprostinil, a prostanoid, offers a potential treatment avenue, but its pediatric use requires further investigation.

Purpose of the Study:

• To describe the pooled pediatric experience with oral treprostinil initiation over two years.
• To evaluate treatment initiation in both prostanoid-naïve children and those transitioning from other prostanoid formulations.
• To document adverse reactions and their management in pediatric patients receiving oral treprostinil.

Main Methods:

• A descriptive, observational study involving 28 pediatric patients from four centers.
• Analysis of various methods for initiating oral treprostinil therapy.
• Inclusion of patients as young as four years old and weighing as little as 16 kg.
• Documentation of adverse events and their clinical management.

Main Results:

• Most patients (27/28) successfully initiated oral treprostinil therapy.
• Gastrointestinal adverse reactions were common among pediatric patients.
• Approximately half of the patients discontinued therapy within the two-year study period due to adverse events.

Conclusions:

• Oral treprostinil can be initiated in pediatric patients with pulmonary arterial hypertension.
• Gastrointestinal adverse reactions represent a significant challenge, impacting treatment adherence and leading to discontinuation in a substantial proportion of children.
• Further research is needed to optimize the use and tolerability of oral treprostinil in pediatric PAH management.