Can parenteral treprostinil help children with pediatric Pulmonary Arterial Hypertension based on recent studies?

Parenteral treprostinil is a prostacyclin therapy given by injection (under the skin or into a vein) for pulmonary arterial hypertension (PAH). In children, PAH is rare and severe, and treatment guidelines often rely on adult data. Recent studies, including a 2024 systematic review and meta-analysis, suggest that parenteral treprostinil can help improve clinical status and may serve as a bridge to transplant or long-term therapy in pediatric PAH. However, the evidence comes from observational studies, not large randomized trials, so the certainty is moderate.

What the research says

A 2024 systematic review and meta-analysis identified 32 studies involving 766 children with PAH treated with parenteral prostacyclins, of which 649 received treprostinil ⁴⁷. The meta-analysis included 143 treprostinil-naïve patients from five publications and showed improvements in clinical outcomes, though no randomized controlled trials were available ⁴⁷. A separate study from 2018 reported on eight children treated with subcutaneous treprostinil: four improved or stabilized, two were lost early due to insufficient response, and three eventually had successful lung transplantation ⁶. The authors concluded that long-term subcutaneous treprostinil can be safe and well-tolerated in children, even at higher doses, and may avoid complications of central lines ⁶. Another study from 2020 examined oral treprostinil pharmacokinetics in 32 children, finding that oral dosing led to higher peak but lower trough concentrations compared to parenteral, with wide variability ⁵. This suggests that parenteral (injectable) treprostinil may provide more stable drug levels, which could be important for efficacy ⁵. Overall, the evidence supports that parenteral treprostinil is a viable option for pediatric PAH, especially for high-risk patients, but more research is needed ⁴⁷.

What to ask your doctor

• What are the potential benefits of parenteral treprostinil for my child's specific PAH severity and type?
• What are the common side effects of subcutaneous or intravenous treprostinil in children, and how are they managed?
• How does treprostinil compare to other prostacyclin therapies (like epoprostenol) for pediatric PAH?
• Is my child a candidate for transitioning from parenteral to oral treprostinil, and what does that involve?
• What monitoring is needed during treprostinil therapy, and how often will we need follow-up visits?