Quality of Life Questionnaire in Pulmonary Hypertension EMPHASIS-10: Brazilian Adaptation and Validation
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorHospital de Clinicas de Porto Alegre
About this trial
Pulmonary hypertension (PH) is a severe and heterogeneous condition associated with poor prognosis and significant impairment in quality of life. Patient-reported outcome questionnaires are useful, low-cost tools for clinical assessment and risk stratification. The EmPHasis-10 questionnaire is a brief and easy-to-administer instrument that has demonstrated good correlation with clinical outcomes across different PH populations; however, a validated Brazilian Portuguese version is not yet available.
This study aims to translate, culturally adapt, and validate the EmPHasis-10 questionnaire for Brazilian patients with pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). The investigators designed a cross-sectional observational study including 50 adult patients with a confirmed diagnosis of PAH (group 1) or CTEPH (group 4), followed at the Pulmonary Circulation Outpatient Clinic of Hospital de Clínicas de Porto Alegre. The EmPHasis-10-BR will be administered alongside the SF-36 questionnaire for concurrent validity analysis, as well as frailty assessment instruments. Reliability will be evaluated through test-retest analysis in both in-person and remote settings.
Clinical, functional, and hemodynamic variables will also be collected to investigate associations between EmPHasis-10-BR scores and risk stratification parameters recommended by international guidelines. Statistical analyses will include Cronbach's alpha to assess internal consistency, Spearman's correlation coefficients for test-retest reliability, and comparisons with previously validated instruments.
The investigators expect that the validation of the EmPHasis-10-BR will provide a practical, reliable, and culturally appropriate tool for quality-of-life assessment in Brazilian patients with PH, supporting standardized clinical follow-up, risk stratification, and potentially contributing to therapeutic decision-making.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Willingness to participate in the study after being informed and having provided written informed consent;
Age ≥ 18 years;
Diagnosis of precapillary pulmonary hypertension from groups 1 and 4, confirmed by right heart catheterization;
Clinically stable patients with no changes in therapy for at least 3 months;
Disqualifiers
Diagnosis of pulmonary hypertension from groups 2, 3, and/or 5;
Spirometry showing forced vital capacity (FVC) ≤ 70% predicted and/or forced expiratory volume in the first second (FEV₁) ≤ 60% predicted;
Severe communication impairment, including major cognitive deficit or severe hearing or visual impairment, resulting in inability to respond to the questionnaires or to understand the study procedures;
Patients who experience clinical exacerbation or worsening between the days on which the questionnaires are administered;
Trial population
The study population will consist of patients diagnosed with pulmonary hypertension from clinical groups 1 and 4, according to the classification of the most recent World Symposium on Pulmonary Hypertension, who are followed on an outpatient basis at the Pulmonary Circulation Clinic of the Pulmonology Department of Hospital de Clínicas de Porto Alegre (HCPA). Patients will be consecutively recruited during the study period. A minimum disease duration or follow-up period of 6 months will be required.
Trial design
Design model
Case-only
Time perspective
Cross-sectional
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Internal consistency of the EmPHasis-10-BR questionnaire measured by Cronbach's alpha
EmPHasis-10 Brazilian Portuguese version (EmPHasis-10-BR): Evaluates health-related quality of life in pulmonary hypertension. Scores range from 0 to 50, where higher scores indicate a worse outcome (greater disease impact/lower quality of life).
Time frame
7-28 days2
Test-retest reliability measured by Spearman's correlation coefficient
Time frame
7-28 days3
Concurrent validity assessed by correlation with SF-36 questionnaire
Concurrent validity considering the SF-36 (Brazilian Portuguese version), a generic questionnaire of 36 items evaluating health-related quality of life across 8 domains (Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health) and 2 summary component scores (PCS and MCS). Transformed scores for each domain/component range from 0 to 100, where higher scores indicate a better outcome (better overall health status and higher quality of life).
Time frame
7-28 daysSecondary outcomes
1
Concurrent validity of the EmPHasis-10-BR questionnaire measured by correlation with SF-36 scores
Concurrent validity of the EmPHasis-10 Brazilian Portuguese version (EmPHasis-10-BR) will be evaluated by assessing its correlation with the 36-Item Short Form Health Survey (SF-36) using the Spearman rank correlation coefficient. 1) EmPHasis-10-BR: A disease-specific questionnaire evaluating health-related quality of life in pulmonary hypertension. Total scores range from 0 to 50, where higher scores indicate a worse outcome (greater disease impact and lower quality of life). 2) SF-36 (Brazilian Portuguese version): A generic questionnaire of 36 items evaluating health-related quality of life across 8 domains (Physical Functioning, Role-Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role-Emotional, and Mental Health) and 2 summary component scores (PCS and MCS). Correlation values range from -1.0 (perfect negative correlation) to +1.0 (perfect positive correlation).
Time frame
Between enrollment to 7-28 days2
Correlation between age and EmPHasis-10-BR score
Participant age, measured in years, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis
Time frame
Between enrollment to 7-28 days3
Test-retest reliability of the EmPHasis-10-BR questionnaire when administered remotely, measured by correlation coefficients
EmPHasis-10 Brazilian Portuguese version (EmPHasis-10-BR): Evaluates health-related quality of life in pulmonary hypertension. Scores range from 0 to 50, where higher scores indicate a worse outcome (greater disease impact/lower quality of life).
Time frame
Between enrollment to 7-28 days4
Correlation between EmPHasis-10-BR scores and FRAIL-BR scores
The correlation between health-related quality of life and frailty will be assessed using the Spearman's correlation coefficient. 1) EmPHasis-10 Brazilian Portuguese version (EmPHasis-10-BR): Evaluates health-related quality of life in pulmonary hypertension. Scores range from 0 to 50, where higher scores indicate a worse outcome (greater disease impact/lower quality of life). 2) FRAIL Scale - Brazilian Portuguese version (FRAIL-BR): Assesses physical frailty based on 5 components (Fatigue, Resistance, Ambulation, Illnesses, and Loss of Weight). Total scores range from 0 to 5, where 0 = robust, 1-2 = pre-frail, and 3-5 = frail. Higher scores indicate a worse outcome (greater degree of frailty). Correlation values range from -1.0 (perfect negative correlation) to +1.0 (perfect positive correlation).
Time frame
Between enrollment to 7-28 daysSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov