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The SAVOIR study: protocol for the development and validation of a PAH knowledge questionnaire
Ilma Alves de Oliveira Nascimento1, Gabriella Gomes Carvalho1, Nathalia Zorze Rossetto1
1Pulmonary Hypertension Unit, Heart Institute (InCor), Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.
Background:
Pulmonary arterial hypertension (PAH) is a chronic, progressive, and life-threatening disease that requires complex and sustained management. In addition to pharmacological treatment, effective disease control depends on patient engagement, treatment adherence, and adequate understanding of the disease. Despite the recognized importance of patient education, no validated instrument is currently available to assess disease knowledge among patients with PAH, particularly in Portuguese-speaking populations.
Objectives:
The SAVOIR study aims to develop and validate a structured questionnaire designed to assess disease knowledge in patients with pulmonary arterial hypertension.
Methods:
This methodological study will be conducted in accordance with COSMIN recommendations for studies on measurement properties and follows relevant SPIRIT guidance for protocol reporting. The study is registered at ClinicalTrials.gov (NCT07120789). The development and validation process will be carried out in four sequential phases: (1) questionnaire development based on international guidelines (ESC/ERS 2022) and literature review; (2) content validation by expert judges; (3) semantic validation with patients diagnosed with PAH; and (4) psychometric evaluation in a pilot study including approximately 200 patients. The instrument will assess knowledge across domains including disease concepts, symptoms, diagnostic evaluation, treatment, self-management, and communication with healthcare providers. Measurement properties will include content validity, internal consistency, test-retest reliability, structural validity, and construct validity.
Expected Results And Conclusion:
The SAVOIR questionnaire is expected to provide a standardized tool to assess disease knowledge in PAH. By identifying knowledge gaps and enabling targeted educational strategies, this instrument may contribute to improved patient engagement, treatment adherence, and clinical outcomes. In addition, the study will explore the relationship between disease knowledge and established clinical and prognostic parameters, supporting its potential relevance in routine clinical practice.
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