Efficacy and safety of treprostinil in pediatric pulmonary hypertension following fontan surgery: a real-world analysis.

Introduction: This study aimed to evaluate the efficacy and safety of treprostinil in managing pulmonary hypertension (PH) following Fontan surgery in children. The hypothesis was that early postoperative administration of treprostinil could reduce pulmonary arterial pressure and improve oxygenation, utilizing real-life data to better reflect clinical practice.

Methods: A single-center, retrospective study was conducted at the Shanghai Children's Medical Center. The study included patients who underwent Fontan surgery between January 2015 and December 2023, comparing those treated with treprostinil (T Group) and those not treated (NT Group). Propensity-score matching was applied to adjust for baseline differences. Extracted demographic and clinical data included age, weight, height, type of cardiac defect, and surgical details. Postoperative variables included hemodynamic data, inotropic scores(IS), mean pulmonary arterial pressure(mPAP), pulmonary to systemic pressure ratio (Pp/Ps), PaO₂/FiO₂(P/F), volume of drainage and fluid balance. Statistical analysis was performed using SPSS software, with significance set at p<0.05.

Results: A total of 264 patients were identified, with 126 in the T Group and 138 in the NT Group. After matching, 154 patients were enrolled in (77 patients in each group). Mortality rates were similar (T: 0% vs NT: 1.3%, p=0.375). The T group showed significantly higher effective rates at both POD1 (90.9% vs.75.3%, p=0.010) and POD2 (93.5% vs. 79.2%, p=0.005). In patients receiving treprostinil ≥20 ng/kg/min, both mPAP and Pp/Ps showed significant reductions (p<0.001). Compared with baseline, the T group had significantly lower mPAP (P=0.045) and Pp/Ps (P=0.009), and a higher P/F ratio (P=0.02) on POD1. Significant main effects of treprostinil were observed for Pp/Ps (p<0.001), IS (p = 0.012), and P/F (p<0.001).T group had longer mechanical ventilation (p = 0.001) and ICU stay (p = 0.031). No major adverse events were observed.

Conclusion: Treprostinil treatment significantly reduced mPAP and Pp/Ps in the early postoperative period following Fontan surgery, with higher doses of treprostinil yielding more substantial hemodynamic improvements. But it did not significantly alter short-term clinical recovery, with no major adverse reactions observed.