Real-World Outcomes of Treprostinil Treatment in Very-Low-Birth-Weight Infants with Pulmonary Hypertension: A Nationwide Cohort Study

다국어 초록 (Multilingual Abstract)

Purpose: Treprostinil is used off-label for refractory pulmonary hypertension (PH) in very-low-birth-weight infants (VLBWI); however, real-world outcome data remain scarce. This study aimed to describe the clinical and long-term neurodevelopmental outcomes of treprostinil-treated VLBWI with PH.
Methods: Data from 21,645 VLBWI in a nationwide, multicenter registry (Korean National Network) admitted between 2013 and 2023 were retrospectively analyzed to identify those receiving treprostinil for PH. Baseline characteristics and in-hospital morbidities were compared between survivors and non-survivors. Long-term neurodevelopmental outcomes at 18–24 months and 3 years of corrected age were evaluated in survivors.
Results: Treprostinil was administered to 2.0% (39/1,962) of VLBWI with PH, typically as combination therapy. Among the 32 infants (14 survivors, 18 non-survivors), in-hospital mortality was 56.3%, predominantly due to cardiopulmonary causes (83.3%). Lower cord blood pH was a potential predictor of mortality (odds ratio, 0.924 per 0.01 increase in pH; 95% confidence interval, 0.812 to 0.989; P=0.016). Survivors had higher rates of moderate-to-severe bronchopulmonary dysplasia, treated patent ductus arteriosus, and retinopathy of prematurity requiring intervention. This likely reflects a time-at-risk bias, as non-survivors died early (median, 9.5 days). By 3 years corrected age, all survivors were weaned off oxygen and PH medications. However, growth failure (87.5%) and developmental delay (50.0%) persisted despite continuous rehabilitation (85.7%) and speech therapy (50.0%).
Conclusion: Treprostinil is reserved for refractory PH, which involves high mortality. Despite discontinuation of cardiopulmonary support, survivors experienced substantial long-term growth failure and neurodevelopmental impairment