Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Application of Patient Knowledge Questionnaire on Pulmonary Hypertension.
- Who it may be relevant to
- Registry conditions: Pulmonary Arterial Hypertension (PAH), Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH), Pulmonary Hypertension. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Brazil
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Content, Semantic, and Psychometric Validation of a Patient Knowledge Questionnaire for Pulmonary Hypertension
Overview
This methodological study aims to develop and validate a questionnaire named for the Assessment of Patient Knowledge in Pulmonary Arterial Hypertension. The study will be conducted in four phases: (1) questionnaire development based on guidelines and literature; (2) content validation by expert judges; (3) semantic validation with patients; and (4) psychometric testing in a sample of up to 200 patients with confirmed pulmonary arterial hypertension (PAH). In addition to validation, the study will collect clinical and functional data from medical records, including risk stratification using the COMPERA 2.0 method The final instrument is expected to support patient education strategies and contribute to improved clinical management of PAH.
Detailed description
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.
Interventions
- Other Application of Patient Knowledge Questionnaire on Pulmonary Hypertension
Participants will complete one version of the SAVOIR questionnaire, developed to assess patient knowledge about pulmonary arterial hypertension. Depending on the study phase, participants will contribute to content validation (expert judges), semantic validation (patients), or psychometric validation (patients). In the final phase, additional data including risk stratification (COMPERA 2.0) will be collected for correlational analysis.
Primary outcome measures
- Content Validity Index (CVI) of the Questionnaire Items [Time frame: 4 weeks after expert panel review]
- Semantic Clarity Rating by Patients [Time frame: 2 weeks after patient interview]
- Internal Consistency of the Questionnaire (Cronbach's Alpha) [Time frame: 4 weeks after questionnaire completion]
Secondary outcome measures (6)
- Test-Retest Reliability (Intraclass Correlation Coefficient - ICC) [Time frame: 2 weeks after first questionnaire administration]
- Completion Rate and Acceptability of the Questionnaire [Time frame: At the time of first questionnaire administration (Day 1)]
- 6. Association Between questionnaire Score and Risk Stratification (COMPERA 2.0) [Time frame: Within 4 weeks of questionnaire completion]
- Association Between questionnaire Score and Number of PAH-related Hospitalizations [Time frame: Within 4 weeks of questionnaire completion]
- Association Between the questionnaire Score (being developed) and Quality of Life Measures (EMPHASIS-10) [Time frame: Within 4 weeks of questionnaire completion]
- Association Between Patient Knowledge and Treatment Adherence using Martin-Bayarre-Grau (MBG Score) [Time frame: Within 4 weeks of questionnaire completion]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Health services research
Study locations
Brazil · 1 center
- Instituto do Coração (InCor), Hospital das Clínicas HCFMUSP, Faculdade de Medicina, Univer — São Paulo
Publications
- de Oliveira Nascimento IA, Carvalho GG, Zorze Rossetto N, Cauzzo IS, Milan MM, Jardim CVP, Dos Santos Fernandes CJC. The SAVOIR study: protocol for the development and validation of a PAH knowledge questionnaire. Respir Med. 2026 Jul 9:109028. doi: 10.1016/j.rmed.2026.109028. Online ahead of print. PMID 42425312
Identifiers
NCT: NCT07120789 · SGP 27073