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Real-world Sotatercept Eligibility: Analysis from the FOCUS-PAH Registry
Respiration; International Review of Thoracic Diseases
|June 12, 2026
Summary
Pulmonary arterial hypertension (PAH) trial criteria exclude many real-world patients. Even with expanded criteria, eligibility remains limited, necessitating tailored approaches for sotatercept use in PAH.
Area of Science:
- Cardiology
- Pulmonary Medicine
- Clinical Trials
Background:
- Pulmonary arterial hypertension (PAH) has poor outcomes despite current treatments.
- Sotatercept shows promise but trial criteria may restrict its real-world use.
Purpose of the Study:
- To assess real-world eligibility for sotatercept trials (STELLAR/ZENITH) at baseline and follow-up.
- To identify predictors of non-eligibility in PAH patients.
Main Methods:
- Retrospective analysis of 541 PAH patients meeting inclusion criteria (WHO FC II/III, PVR ≥5 WU, stable therapy).
- Eligibility assessed against STELLAR criteria and extended using ZENITH criteria.
- Multivariable models identified predictors of non-eligibility.
Main Results:
- 19% of patients were ineligible at baseline, primarily due to PAH subtype or vasoreactivity.
- Cumulative eligibility during follow-up was 50%, with PVR <5 WU or therapy changes being common exclusion reasons.
- Older age and triple therapy predicted non-eligibility; idiopathic PAH was protective. ZENITH criteria increased eligibility by 6%.
Conclusions:
- STELLAR trial criteria exclude a significant number of real-world PAH patients, especially during follow-up.
- ZENITH criteria offer only a modest improvement in eligibility.
- Optimized patient selection and timing are crucial for sotatercept's real-world application in PAH.
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