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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Group | Experimental | Participants in this arm will take part in one of three phases of the questionnaire validation study:
In the psychometric phase, participants will complete the final version of the SAVOIR questionnaire. Additional clinical and functional data will be collected from medical records, including: risk stratification using the COMPERA 2.0 method, These data will be used to explore associations between patient knowledge and clinical/functional outcomes. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Application of Patient Knowledge Questionnaire on Pulmonary Hypertension | Other | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Content Validity Index (CVI) of the Questionnaire Items | Proportion of agreement among expert judges regarding the relevance and clarity of each item. Items with CVI ≥ 0.80 will be considered valid. | 4 weeks after expert panel review |
| Semantic Clarity Rating by Patients | Assessment of the clarity and comprehension of each item by patients using a structured form. Items with ≥ 85% of patients rating them as clear or very clear will be considered semantically valid. | 2 weeks after patient interview |
| Internal Consistency of the Questionnaire (Cronbach's Alpha) | Psychometric evaluation of the final version of the SAVOIR questionnaire using Cronbach's alpha. A coefficient ≥ 0.70 will be considered acceptable. The analysis will be conducted in a sample of up to 200 patients. | 4 weeks after questionnaire completion |
| Measure | Description | Time Frame |
|---|---|---|
| Test-Retest Reliability (Intraclass Correlation Coefficient - ICC) | Stability of the questionnaire scores over time will be assessed by reapplying the instrument after 1 to 2 weeks in a subsample of patients. ICC ≥ 0.80 will be considered acceptable. | 2 weeks after first questionnaire administration |
| Completion Rate and Acceptability of the Questionnaire |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Caio Fernandes Principal Investigato, PhD | Contact | +55 11 2661 5034 | caio.cesar@hc.fm.usp.br |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Instituto do Coração (InCor), Hospital das ClÃnicas HCFMUSP, Faculdade de Medicina, Universidade de São Paulo | Recruiting | São Paulo | São Paulo | 05403-900 | Brazil |
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This is a methodological study for the development and validation of a questionnaire. The model includes sequential phases: construction, content validation by expert judges, semantic validation with patients, and psychometric analysis in a cross-sectional design. During the final phase, clinical and functional data will be collected from medical records to assess associations between knowledge scores and patient outcomes such as risk stratification (COMPERA 2.0). The study does not involve any intervention or assignment of participants to treatment arms.
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No masking was applied. This is an open-label, non-randomized methodological study to validate a questionnaire. All participants and investigators were aware of the intervention (questionnaire application).
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|
Percentage of participants who complete the questionnaire without missing data and report that the items are clear and easy to understand. |
| At the time of first questionnaire administration (Day 1) |
| 6. Association Between questionnaire Score and Risk Stratification (COMPERA 2.0) | Association between total DICTA score and patient risk status based on the COMPERA 2.0 stratification tool. | Within 4 weeks of questionnaire completion |
| Association Between questionnaire Score and Number of PAH-related Hospitalizations | Correlation between questionnaire score and number of PAH-related hospitalizations in the 12 months prior to questionnaire administration. | Within 4 weeks of questionnaire completion |
| Association Between the questionnaire Score (being developed) and Quality of Life Measures (EMPHASIS-10) | Association between questionnaire score and scores on quality of life tools. | Within 4 weeks of questionnaire completion |
| Association Between Patient Knowledge and Treatment Adherence using Martin-Bayarre-Grau (MBG Score) | Assess the association between the questionnaire scores and treatment adherence, as measured by the Martin-Bayarre-Grau (MBG) scale. The goal is to explore whether higher knowledge levels are correlated with greater adherence to treatment in patients with pulmonary arterial hypertension. | Within 4 weeks of questionnaire completion |
| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| D006976 | Hypertension, Pulmonary |
| D006266 | Health Education |
| D002908 | Chronic Disease |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D006973 | Hypertension |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000099060 | Adherence Interventions |
| D055118 | Medication Adherence |
| D010349 | Patient Compliance |
| D010342 | Patient Acceptance of Health Care |
| D000074822 | Treatment Adherence and Compliance |
| D015438 | Health Behavior |
| D001519 | Behavior |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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