Quality of Life Questionnaire in Pulmonary Hypertension EMPHASIS-10: Brazilian Adaptation and Validation

Hospital de Clinicas de Porto Alegre50 enrolled

Overview

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.

LOGISTICS AND STUDY PROCEDURES The use of the EmPHasis-10 questionnaire was authorized by the Pulmonary Hypertension Association - United Kingdom (PHA-UK), the organization holding its copyright, and the translation process will be conducted in accordance with the guidelines provided by PHA-UK. The translation process will initially consist of two independent translations into Brazilian Portuguese performed by two independent translators. Subsequently, the questionnaire will be back-translated into English by a third independent translator. All translations will be reviewed by a committee composed of three pulmonologists specialized in pulmonary circulation, members of the research team, and fluent in both languages. A review report of the back-translation will be submitted to PHA-UK, which may provide suggestions that will be forwarded to the investigators for further adjustments, if necessary. After the final version is established, the questionnaire will be pilot-tested in five patients. After completing the questionnaire, these patients will be asked about comprehension of each EmPHasis-10-BR item using the following questions: Was this item understood? What does this item mean? Can this item be explained in different words? Can alternative wording be suggested? A report based on this pilot study will be prepared and reviewed by pulmonary hypertension specialists to identify any additional adjustments that may be required. After finalization, patients who meet the eligibility criteria will undergo collection of demographic data and information related to health status, comorbidities, symptoms, and complementary test results. Eligible patients will be invited to participate during outpatient clinic visits and will be referred to the Pulmonology Department after completion of the routine appointment to initiate study procedures. At the first study visit, after signing the written informed consent form, the EmPHasis-10-BR questionnaire will be administered by a trained interviewer. Test-retest reliability will be assessed through an in-person reassessment conducted 7 to 14 days later at the Pulmonology Department. During this second visit, after administration of the EmPHasis-10-BR, participants will also complete the CAMPHOR questionnaire, the SF-36 questionnaire and the Fried Frailty Phenotype criteria. A third contact will be conducted by telephone 7 to 14 days after Visit 2 to validate the questionnaire for remote administration. In addition, routine clinical data used for pulmonary hypertension risk stratification will be collected through medical record review, in accordance with standard outpatient care at the Pulmonary Circulation Clinic. These data will include laboratory tests, echocardiography, six-minute walk test, cardiopulmonary exercise testing, cardiac magnetic resonance imaging, and right heart catheterization results. All collected data will be entered into a Google Sheets® database by a single investigator. STUDY VARIABLES Independent variables: demographic data; smoking history and smoking index; anthropometric measurements; comorbidities; medications; NYHA functional class; pulmonary hypertension etiology and group; ESC risk stratification; complementary test results. Dependent variable: EmPHasis-10-BR score. STATISTICAL ANALYSIS Quantitative variables will be expressed as mean ± standard deviation or median and interquartile range, according to data distribution. The Shapiro-Wilk test will be used to assess normality. Qualitative variables will be expressed as percentages, and 95% confidence intervals will be calculated when appropriate. Internal consistency of the EmPHasis-10-BR will be evaluated using Cronbach's alpha. Values below 0.7 will be considered indicative of insufficient internal consistency. Item-total correlations are expected to range between 0.2 and 0.8. Test-retest reliability will be assessed by correlating EmPHasis-10-BR scores obtained at different time points (Visit 1 and Visit 2), assuming no clinical change occurred between administrations. A minimum correlation coefficient of 0.85 will be required to indicate adequate reliability, using Spearman's correlation test. A similar analysis will be performed comparing in-person (Visit 2) and remote (Visit 3) administrations. Additionally, EmPHasis-10-BR scores will be compared with general and disease-specific quality-of-life questionnaires validated in Portuguese, namely the 36-item Short Form Health Survey (SF-36) and the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR). All statistical tests will be two-tailed, and p values \< 0.05 will be considered statistically significant. Data will be analyzed using the Statistical Package for the Social Sciences (SPSS), version 18.0 for Windows (SPSS Inc., Chicago, IL, USA). RISKS The potential risks associated with participation in this study include unauthorized disclosure or identification of patient-related data. However, all measures to ensure confidentiality will be adopted. The investigators commit to preserving participants' privacy throughout the study and to using all collected information solely for the execution of this research project. BENEFITS The expected benefits of this study include the validation of the EmPHasis-10 questionnaire for use in Brazilian Portuguese, aiming to standardize clinical assessment and generate reliable data for longitudinal follow-up. Additionally, the instrument may support non-invasive, low-cost risk stratification and prognostic assessment, with potential applicability across different levels of healthcare.

Outcomes

Primary Outcomes

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 days

Test-retest reliability measured by Spearman's correlation coefficient

Time frame: 7-28 days

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 days

Secondary Outcomes

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).