Abstract
Use of subcutaneous treprostinil (TRE) in children with pulmonary hypertension (PH) secondary to lung disease (group 3 PH) is not well described. We hypothesized that initiation of TRE is associated with improvement in PH severity and right ventricular (RV) function with minimal early side effects. We conducted a single-center retrospective analysis of children with group 3 PH treated with TRE between 2006 and 2022. We compared echocardiographic changes in PH severity and RV systolic function over the first 3-6 months of TRE and described adverse effects over the first 48 h of therapy. Forty-one patients (56% male), aged 6 days to 15 years old at the time of TRE initiation, were included. The most common associated diagnoses were bronchopulmonary dysplasia (63%), congenital diaphragmatic hernia (10%), and pulmonary hypoplasia (10%). TRE was associated with improvement in PH severity (p < 0.001), estimated by interventricular septal position at end-systole (p = 0.001), RV systolic pressure to systemic blood pressure ratio (p < 0.001), and flow direction across a simple shunt (p = 0.153). There were improvements in individual markers of RV function including tricuspid annular plane systolic excursion (p = 0.005), tricuspid annulus dilation (p = 0.004), RV global longitudinal strain (p < 0.001), RV free wall longitudinal strain (p < 0.001), and qualitative RV systolic function (p = 0.002). Over the first 48 h of TRE, the dose was reduced in 3 patients (7%) due to hypotension, hypoxemia, or emesis, all of whom tolerated re-escalation within 48 h. TRE is well tolerated and is associated with early improvement in PH severity and RV function in children with group 3 PH.