Abstract Rationale The 16-week INCREASE trial demonstrated that inhaled Treprostinil improved walk distance in patients with interstitial lung disease-associated pulmonary hypertension (ILD-PH). A secondary post-hoc study suggested inhaled Treprostinil was associated with improvement in FVC in ILD-PH patients. This follow up study has led to the ongoing TETON-IPF and TETON-PPF trials which are investigating inhaled Treprostinil’s effect on FVC in ILD patients without PH. Since inhaled Treprostinil has been in clinical use for ILD-PH since 2021, our study aimed to determine whether real-world data continued to support its potential benefits by retrospectively assessing patients over their first year of treatment. Methods In this retrospective observational cohort study, patients with ILD-PH were identified from a database of individuals receiving treatment for pulmonary hypertension between June 2022 and February 2024. Data were collected at approximately 3, 6-, and 12-month intervals.Inclusion required baseline assessments and at least one follow-up measurement of pulmonary function tests (PFTs), six-minute walk distance (6MWD), right heart catheterization (RHC), N-terminal pro-B-type natriuretic peptide (NT-proBNP), and echocardiography within the study period. Additional data were obtained on concurrent medications (including pulmonary-selective vasodilators, antifibrotic agents, and immunosuppressive therapies), mortality, hospitalizations, and adverse effects attributed to inhaled Treprostinil. Results Of the 120 patients screened from a predetermined PH-Treprostinil database, 59 met inclusion criteria. Approximately 50% were on immunosuppressive therapy, 30% on antifibrotic agents, and 30-40% on additional pulmonary vasodilators during the study period.Compared with baseline, no statistically significant changes were observed in FVC, DLCO, or 6MWD at 3, 6, or 12 months (p 0.05). Among 34 patients with follow-up echocardiography at 6 or 12 months, 21% demonstrated improved RVSP, while a similar proportion remained 35 mmHg. Systolic or diastolic dysfunction was present in 38% of these patients.Repeat RHC showed a nonsignificant trend toward lower mPAP and reduced NT-proBNP, with no change in cardiac index. Over the study period, there were 34 hospitalizations and one death. Conclusions No statistically significant changes were observed in FVC, DLCO, 6MWD, NT-proBNP, mPAP, or cardiac index following inhaled Treprostinil therapy in ILD-PH patients. Study limitations included small sample size, inconsistent data collection, and difficulty tracking dose adjustments. These results emphasize the value of randomized controlled trials such as INCREASE and TETON-IPF while illustrating the challenges of real-world data collection. Comorbidities, concurrent therapies, and overall disease burden may have attenuated the observed treatment effect. This abstract is funded by: None
Tiegs et al. (Fri,) studied this question.