CHEST Guidelines-Psychometric-Validation-of-the-Pulmonary-Arterial-

Psychometric-Validation-of-the-Pulmonary-Arterial-

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The article details the psychometric validation process of the Pulmonary Arterial Hypertension-Symptoms and Impact Questionnaire (PAH-SYMPACT), which was developed following the 2009 FDA PRO guidance. This questionnaire aims to capture patient-reported outcomes specific to pulmonary arterial hypertension (PAH), a chronic and serious condition marked by symptoms such as fatigue, chest pain, and shortness of breath, impacting patients' overall quality of life.<br /><br />The validation utilized data from the SYMPHONY Trial, where macitentan was administered to 278 participants, predominantly female, with a mean age of 60, over 16 weeks. The study's objective was to confirm the questionnaire's content validity and test its psychometric properties including reliability, sensitivity, and construct validity. Exploratory and confirmatory factor analyses resulted in the selection of 11 symptoms and 11 impact items, grouped into four domains—cardiopulmonary symptoms, cardiovascular symptoms, physical impacts, and cognitive/emotional impacts. These domains exhibited high internal consistency (Cronbach’s alpha ≥ 0.80), strong test-retest reliability, and good known-groups and construct validity, indicating effectiveness in differentiating between various levels of disease severity and showing correlations with other recognized quality of life and symptom indices.<br /><br />The study highlighted that PAH-SYMPACT is unaffected by oxygen use, enhancing its versatility for clinical trials and practice. However, despite its strengths, the study's limitations included the lack of a control group and a predominance of less severe PAH cases, which may have resulted in floor effects for certain items. Nevertheless, the PAH-SYMPACT stands out as a robust instrument conforming to FDA standards, providing a comprehensive measure for monitoring PAH impacts on patients’ lives.

Keywords

Pulmonary Arterial Hypertension

PAH-SYMPACT

patient-reported outcomes

psychometric validation

SYMPHONY Trial

macitentan

content validity

reliability

construct validity

quality of life