Why the study?
The value placed by patients and their caregivers on the components of composite outcomes in pulmonary arterial hypertension remains unknown.
How do patients and caregivers perceive the importance of components of composite outcomes in pulmonary arterial hypertension trials?
How do patients and caregivers perceive the importance of components of composite outcomes in pulmonary arterial hypertension trials?
Patients and caregivers value death as the only critical outcome in PAH trials, highlighting the need to integrate patient perspectives in clinical trial design.
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PAH trial composites warrant cautious interpretation now; leaves open patient-valued weighting of future endpoints.
The value placed by patients and their caregivers on the components of composite outcomes in pulmonary arterial hypertension (PAH) remains unknown. We surveyed the importance of these outcomes from a patients' and caregivers' perspective, with participants (n=335, including 257 patients with PAH) rating individual components defining clinical worsening in PAH trials as of critical, major, mild-to-moderate or minor importance. Most outcomes were considered of major or mild-to-moderate importance to patients. Death was the only outcome considered of critical importance. Perceptions of clinical outcomes varied between patients and caregivers. Integrating patients' perception in the elaboration of clinical trials is essential.
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Mai et al. (2023) studied this question.
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