Skip to main content
OpenTrials
Recruiting

NCT Number: NCT07822997

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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Pulmonary Hypertension Cardiovascular Diseases Hypertension Hypertension, Pulmonary Lung Diseases Quality of Life Respiratory Tract Diseases Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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;
  • Native speaker of Brazilian Portuguese;
  • Literate patient.

Exclusion criteria

  • 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;
  • Refusal to participate in the study.

Treatment and study plan

Primary outcomes

  1. Internal consistency of the EmPHasis-10-BR questionnaire measured by Cronbach's alpha

    Time frame: 7-28 days

    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. Test-retest reliability measured by Spearman's correlation coefficient

    Time frame: 7-28 days

  3. Concurrent validity assessed by correlation with SF-36 questionnaire

    Time frame: 7-28 days

    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).

Secondary outcomes

  1. Concurrent validity of the EmPHasis-10-BR questionnaire measured by correlation with SF-36 scores

    Time frame: Between enrollment to 7-28 days

    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).

  2. Correlation between age and EmPHasis-10-BR score

    Time frame: Between enrollment to 7-28 days

    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

  3. Test-retest reliability of the EmPHasis-10-BR questionnaire when administered remotely, measured by correlation coefficients

    Time frame: Between enrollment to 7-28 days

    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).

  4. Correlation between EmPHasis-10-BR scores and FRAIL-BR scores

    Time frame: Between enrollment to 7-28 days

    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).

  5. Correlation between EmPHasis-10-BR scores and the Fried Frailty Phenotype Criteria scores

    Time frame: Between enrollment to 7-28 days

    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) Fried Frailty Phenotype Criteria: Assesses physical frailty across 5 criteria (unintentional weight loss, weakness/grip strength, exhaustion, slowness/gait speed, and low physical activity). 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).

  6. Comparison of EmPHasis-10-BR scores across age categories

    Time frame: 7-28 days

    EmPHasis-10-BR total scores will be compared across three age categories: <50 years, 50-64 years, and ≥65 years. Age is measured in years. The Kruskal-Wallis test will be used when the distribution is non-normal, and one-way ANOVA will be used when the assumptions of normality and homoscedasticity are reasonably met.

  7. Comparison of EmPHasis-10-BR scores by gender

    Time frame: 7-28 days

    EmPHasis-10-BR total scores will be compared between male and female participants. Gender will be categorized as male or female. The Mann-Whitney U test will be used for non-normally distributed scores, and the independent-samples t-test will be used when the assumptions of normality and homoscedasticity are reasonably met.

  8. Comparison of EmPHasis-10-BR scores by modified NYHA functional class for pulmonary hypertension

    Time frame: 7-28 days

    EmPHasis-10-BR total scores will be compared between participants classified as functional class I-II and III-IV according to the modified New York Heart Association (NYHA) functional classification for pulmonary hypertension. The Mann-Whitney U test will be used for non-normally distributed scores, and the independent-samples t-test will be used when the assumptions of normality and homoscedasticity are reasonably met.

  9. Comparison of EmPHasis-10-BR scores across ESC risk stratification categories

    Time frame: 7-28 days

    EmPHasis-10-BR total scores will be compared across three pulmonary hypertension risk categories according to the European Society of Cardiology (ESC) risk stratification: low risk, intermediate risk, and high risk. The Kruskal-Wallis test will be used for non-normally distributed scores, and one-way ANOVA will be used when the assumptions of normality and homoscedasticity are reasonably met.

  10. Comparison of EmPHasis-10-BR scores across four ESC risk stratification categories

    Time frame: 7-28 days

    EmPHasis-10-BR total scores will be compared across four pulmonary hypertension risk categories according to the European Society of Cardiology (ESC) risk stratification: low risk, intermediate-low risk, intermediate-high risk, and high risk. The Kruskal-Wallis test will be used for non-normally distributed scores, and one-way ANOVA will be used when the assumptions of normality and homoscedasticity are reasonably met.

  11. orrelation between six-minute walk test distance and EmPHasis-10-BR score

    Time frame: 7- 28 days

    Six-minute walk test distance, measured in meters, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis. A multivariable linear regression model will also be performed with the EmPHasis-10-BR total score as the dependent variable and six-minute walk test distance as the primary independent variable, adjusting for potential confounders including age, sex, WHO functional class, pulmonary hypertension etiology, BNP/NT-proBNP level, pulmonary artery pressure, and cardiac output.

  12. Correlation between echocardiographic pulmonary artery systolic pressure and EmPHasis-10-BR score

    Time frame: 7-28 days

    Echocardiographic pulmonary artery systolic pressure (PASP), measured in mmHg, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  13. Correlation between echocardiographic TAPSE/PASP ratio and EmPHasis-10-BR score

    Time frame: 7-28 days

    The echocardiographic tricuspid annular plane systolic excursion to pulmonary artery systolic pressure ratio (TAPSE/PASP), measured in mm/mmHg, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  14. Correlation between echocardiographic right atrial area and EmPHasis-10-BR score

    Time frame: 7-28days

    Echocardiographic right atrial area, measured in cm², will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis

  15. Correlation between cardiac magnetic resonance right ventricular ejection fraction and EmPHasis-10-BR score

    Time frame: 7-28 days

    Right ventricular ejection fraction (RVEF), assessed by cardiac magnetic resonance imaging and expressed as a percentage, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  16. Correlation between cardiac magnetic resonance right atrial area and EmPHasis-10-BR score

    Time frame: 7- 28 days

    Right atrial area, assessed by cardiac magnetic resonance imaging and measured in cm², will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  17. Correlation between peak oxygen uptake and EmPHasis-10-BR score

    Time frame: 7-28 days

    Peak oxygen uptake (VO₂ peak), assessed by cardiopulmonary exercise testing and expressed in mL/kg/min, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  18. Correlation between VE/VCO₂ slope and EmPHasis-10-BR score

    Time frame: 7-28 days

    The VE/VCO₂ slope, assessed by cardiopulmonary exercise testing and expressed in L/L, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  19. Correlation between peak oxygen pulse and EmPHasis-10-BR score

    Time frame: 7-28 days

    Peak oxygen pulse, assessed by cardiopulmonary exercise testing and expressed in mL/beat, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  20. Correlation between mean pulmonary artery pressure and EmPHasis-10-BR score

    Time frame: 7-28 days

    ean pulmonary artery pressure, measured by right heart catheterization and expressed in mmHg, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  21. Correlation between right atrial pressure and EmPHasis-10-BR score

    Time frame: 7-28 days

    Right atrial pressure, measured by right heart catheterization and expressed in mmHg, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  22. Correlation between cardiac index and EmPHasis-10-BR score

    Time frame: 7-28 days

    Cardiac index, measured by right heart catheterization and expressed in L/min/m², will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

  23. Correlation between pulmonary vascular resistance and EmPHasis-10-BR score

    Time frame: 7-28 days

    Pulmonary vascular resistance, measured by right heart catheterization and expressed in Wood units, will be evaluated in relation to the total score of the EmPHasis-10-BR questionnaire using Spearman rank correlation and linear regression analysis.

Study contacts

Contact information is provided by the study sponsor or research team.

Bruna H Bianchini, MD

CONTACT

[email protected]

55+ 48 991023563

Marcelo B Gazzana, MD, PhD

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Hospital de Clinicas de Porto Alegre

Other

Registry information

Official study title

Cultural Adaptation, Validation, and Reliability of the EMPHASIS-10 Questionnaire for Assessing Quality of Life in Patients With Pulmonary Arterial Hypertension and Chronic Thromboembolic Pulmonary Hypertension: Brazilian Version

Acronym: EMPHASIS-10-BR

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Sep 16, 2026
Registry last updated
Sep 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.