D28-15 Mind the Gap: A Global Disconnect Between Approved Antifibrotic Use and Off-label PH Therapies in PH-ILD

Rationale

Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD) presents a severe clinical phenotype with historically limited therapeutic options. A recent European survey confirmed a heavy reliance on off-label therapies for pulmonary hypertension associated with interstitial lung disease (PH-ILD), highlighting a critical unmet need. Whether this practice pattern and clinician uncertainty are consistent worldwide is unknown. This study aims to characterize the global scope of this unmet need by mapping prescription patterns across 32 countries.

Methods

Building on a European survey model, we conducted a multinational, cross-sectional survey of Pulmonologists, Cardiologists, and Rheumatologists across 32 countries. The survey quantified site-level prescription rates for antifibrotics and off-label PH-specific therapies (PDE-5 inhibitors, ERAs, Prostacyclin analogs, and sGC stimulators). Data were analyzed using descriptive statistics.

Results

Analysis of 232 responses (138 complete) revealed a more conservative global approach than European data suggests. Notably, 74% of global respondents reported zero off-label usage, contrasting with the European finding that 50% of patients received off-label treatment. Antifibrotics showed consistent global integration (mean prescription rate: 52%, IQR: 41%-67%). Among clinicians utilizing off-label therapy (26%), PDE-5is and ERAs were preferred, mirroring the European pattern where PDE-5i was the dominant first-line choice. Newer agents (sGC stimulators) saw minimal uptake. Regionally, Asia and South America demonstrated higher off-label use than Europe and North America. These findings should be interpreted considering the cross-sectional design, reliance on self-reported data, and uneven geographic representation, with data from Africa limited to South Africa.

Conclusion

This global survey uncovers a stark dichotomy in PH-ILD management and significant regional disparities. It corroborates the European-identified unmet need, yet reveals that global clinician hesitancy to use off-label PH therapies is more profound. The consistent preference for PDE-5is/ERAs when treatment is attempted establishes them as a de facto, yet inconsistently applied, strategy. Despite limitations including potential response bias, these findings underscore an urgent need for globally harmonized, evidence-based guidelines and further clinical research to standardize care and address the persistent gap between real-world practice and patient need.

This abstract is funded by: None