Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension

This is a methodological study with a quantitative approach, developed in four main phases, aimed at constructing and validating a questionnaire to assess the knowledge of patients with pulmonary arterial hypertension (PAH). The study will be conducted at a reference outpatient center in Brazil and will follow established methodological guidelines for the development and validation of health-related instruments.

Phase 1 - Questionnaire Development:

The initial version of the questionnaire, will be created based on the 2022 ESC/ERS Guidelines for PAH, a review of the scientific literature on patient knowledge in chronic diseases, and validated instruments in other conditions (e.g., chronic kidney disease, inflammatory bowel disease, rheumatoid arthritis). Items will cover the following domains: basic concepts, signs and symptoms, diagnosis and exams, available treatments and their mechanisms, adherence and self-care, and side effect management. Items will be multiple-choice with the option "I don't know".

Phase 2 - Content Validation:

At least five expert judges with experience in PAH, health education, and instrument development will evaluate each item for clarity, relevance, representativeness, and appropriateness using a 4-point Likert scale. The Content Validity Index (CVI) will be calculated for each item (I-CVI) and for the total instrument (S-CVI). Items with I-CVI ≥ 0.78 will be retained.

Phase 3 - Semantic Validation:

A sample of at least 10 patients diagnosed with PAH will be asked to complete the questionnaire while verbalizing their understanding ("think-aloud" technique). Revisions will be made based on participant feedback to ensure clarity and comprehension.

Phase 4 - Pilot Study and Psychometric Analysis:

The refined questionnaire will be applied to a sample of up to 200 patients with confirmed PAH. Psychometric analysis will include internal consistency (Cronbach's alpha) and, if applicable, test-retest reliability (intraclass correlation coefficient - ICC). Exploratory factor analysis may be used to assess internal structure.

In addition to validation, the study will collect clinical and functional data from medical records, including:

Risk stratification using COMPERA 2.0 methodology

The relationship between knowledge scores (SAVOIR) and these outcomes will be analyzed, aiming to explore whether higher patient knowledge is associated with better risk status, lower hospitalization rates, and higher quality of life.

This instrument is expected to help identify educational gaps, support targeted health education strategies, and contribute to improved clinical outcomes and shared decision-making in the care of PAH patients.

Inclusion criteria

• Adults aged 18 years or older
• Confirmed diagnosis of Group 1 Pulmonary Arterial Hypertension (PAH)
• Ability to read and understand the questionnaire language
• Provided informed consent to participate in the study
• For expert judges (content validation phase): professionals with recognized expertise in pulmonary hypertension, with academic or clinical experience in the field

Exclusion criteria

• Cognitive, visual, or auditory impairments that hinder questionnaire comprehension
• Refusal to participate or withdrawal of informed consent
• Inability to complete the questionnaire due to clinical instability