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
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Internal consistency of the EmPHasis-10-BR questionnaire measured by Cronbach's alpha
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
结局指标
主要结局
Internal consistency of the EmPHasis-10-BR questionnaire measured by Cronbach's alpha
时间窗: 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).
Test-retest reliability measured by Spearman's correlation coefficient
时间窗: 7-28 days
Concurrent validity assessed by correlation with SF-36 questionnaire
时间窗: 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).
次要结局
- Concurrent validity of the EmPHasis-10-BR questionnaire measured by correlation with SF-36 scores(Between enrollment to 7-28 days)
- Correlation between age and EmPHasis-10-BR score(Between enrollment to 7-28 days)
- Test-retest reliability of the EmPHasis-10-BR questionnaire when administered remotely, measured by correlation coefficients(Between enrollment to 7-28 days)
- Correlation between EmPHasis-10-BR scores and FRAIL-BR scores(Between enrollment to 7-28 days)
- Correlation between EmPHasis-10-BR scores and the Fried Frailty Phenotype Criteria scores(Between enrollment to 7-28 days)
- Comparison of EmPHasis-10-BR scores across age categories(7-28 days)
- Comparison of EmPHasis-10-BR scores by gender(7-28 days)
- Comparison of EmPHasis-10-BR scores by modified NYHA functional class for pulmonary hypertension(7-28 days)
- Comparison of EmPHasis-10-BR scores across ESC risk stratification categories(7-28 days)
- Comparison of EmPHasis-10-BR scores across four ESC risk stratification categories(7-28 days)
- orrelation between six-minute walk test distance and EmPHasis-10-BR score(7- 28 days)
- Correlation between echocardiographic pulmonary artery systolic pressure and EmPHasis-10-BR score(7-28 days)
- Correlation between echocardiographic TAPSE/PASP ratio and EmPHasis-10-BR score(7-28 days)
- Correlation between echocardiographic right atrial area and EmPHasis-10-BR score(7-28days)
- Correlation between cardiac magnetic resonance right ventricular ejection fraction and EmPHasis-10-BR score(7-28 days)
- Correlation between cardiac magnetic resonance right atrial area and EmPHasis-10-BR score(7- 28 days)
- Correlation between peak oxygen uptake and EmPHasis-10-BR score(7-28 days)
- Correlation between VE/VCO₂ slope and EmPHasis-10-BR score(7-28 days)
- Correlation between peak oxygen pulse and EmPHasis-10-BR score(7-28 days)
- Correlation between mean pulmonary artery pressure and EmPHasis-10-BR score(7-28 days)
- Correlation between right atrial pressure and EmPHasis-10-BR score(7-28 days)
- Correlation between cardiac index and EmPHasis-10-BR score(7-28 days)
- Correlation between pulmonary vascular resistance and EmPHasis-10-BR score(7-28 days)
研究者
Marcelo Basso Gazzana
Physician assistant pulmonologist; Professor of Graduate Program in Pneumological Sciences,
Hospital de Clinicas de Porto Alegre