Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 260
- 试验地点
- 2
- 主要终点
- Survival
研究概览
简要总结
Idiopathic pulmonary fibrosis (IPF) is characterized by a poor prognosis, with a progressive decline in lung function and a considerable variability in the disease's natural history. Besides lung transplantation (LTx), the only available treatments are anti-fibrosing drugs, which have shown to slower the disease course. Therefore, predicting the prognosis is of pivotal importance to avoid treatment delays, which may be fatal for patients with a high risk of progression. Previous studies showed that a multi-dimensional approach is practical and effective to create a reliable prognostic score for IPF. In the RIsk Stratification scorE (RISE), physiological parameters, an objective measure of patient-reported dyspnea and exercise capacity are combined to capture different domains of the complex pathophysiology of IPF.
This is an observational, multi-centre, prospective cohort study. A development cohort and a validation cohort will be included. Patients newly diagnosed with IPF based on the ATS/ERS criteria and multi-disciplinary discussion will be included in the study. A panel of chest radiologists and lung pathologists will further assess eligibility. At the first visit (time of diagnosis), and every 4-months, MRCDS, pulmonary function tests (FEV1, FVC and DLCO), and 6MWD will be recorded and patients will be prospectively followed for 3 years. Comorbidities will be considered. The radiographic extent of fibrosis on HRCT will be recalculated at a 2-year interval. RISE, Gender-Age-Physiology, CPI and Mortality Risk Scoring System will be calculated at 4-month intervals. Longitudinal changes of each variable considered will be assessed. The primary endpoint is 3-year LTx-free survival from the time of diagnosis. Secondary endpoints include several, clinically-relevant information to ensure reproducibility of results across a wide range of disease severity and in concomitance of associated pulmonary hypertension, emphysema.
The present study aims at validating RISE as a simple, straightforward, inexpensive and reproducible tool to guide clinical decision making in IPF and potentially as an endpoint for future clinical trials.
详细描述
This is an observational, international, prospective cohort study including 2 large tertiary referral centres for interstitial lung disease (ILD)(London, Canada and Rome, Italy). Only patients newly diagnosed with IPF based on the American Thoracic Society/European Respiratory Society criteria and local multi-disciplinary discussion will be included in the study.
As a further inclusion step, for all patients who did not undergo a surgical lung biopsy (SLB), a panel of 3 chest radiologists with ILD expertise will examine each HRCT and patients will be included, if at least 2 out of 3 agree on a pattern of probable/definite UIP. For patients who underwent a SLB, a panel of 3 lung pathologists with ILD expertise will examine each biopsy and patients will be included, if at least 2 out of 3 agree on a pattern of probable/definite UIP.
All known causes of ILD (occupational, environmental, domestic exposures; drug-induced lung toxicity; connective tissue disease) will carefully excluded for the patient to be included in the study. Only patients newly diagnosed with IPF at participating centres will be included in the study. Comorbidities considered will include (associated pulmonary hypertension [APH], only if right heart catheterization[RHC]-proven), chronic obstructive pulmonary disease (COPD), coronary artery disease (CAD), left heart dysfunction (LHD), and sleep apnea (SA).
A development cohort and a validation cohort will be included in the study. In both cohorts. at the first visit (time of diagnosis) and at each subsequent visit at 4-month intervals, for a period of 3 years, Medical Research Council Dyspnea Score (MRCDS), pulmonary function tests (PFTs)(forced expiratory volume during the first second [FEV1], FVC and diffusing lung capacity for carbon monoxide [DLCO]), and 6MWD will be recorded. 6MWD % predicted will be calculated, as previously described.
The following events will be recorded in the study: death; lung transplant (LTx) referral; LTx; acute exacerbation (AEs); hospitalization for respiratory causes; start, stop and switch of anti-fibrotic therapy; start of supplemental home oxygen therapy; start and stop of physiotherapy program enrolments; progression of disease (see secondary endpoints).
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •A new diagnosis of IPF based on the American Thoracic Society/European Respiratory Society criteria (Am J Respir Crit Care Med 2018;198:e44-e68) and confirmed by a panel of expert chest radiologists and lung pathologists in the context of multi-disciplinary discussion.
排除标准
- •Interstitial lung disease other than IPF
- •Not a new diagnosis of IPF
结局指标
主要结局
Survival
时间窗: 3 years
Primary outcome is lung-transplant-free 3-year survival since the time of diagnosis
次要结局
- Results stratification by autoimmune markers(3 years)
- Clinical progression(3 years)
- Incidence of acute exacerbations(3 years)
- Results stratification by anti-fibrotic treatment(3 years)
- Subgroup analysis(3 years)
- Results stratification by anti-fibrotic treatment switch(3 years)
- Results stratification by HRT pattern(3 years)
- 6MWD m vs. % pred as predictors of mortality(3 years)
- Results on patient listed for lung transplant(3 years)
- Incidence of hospitalizations(3 years)
- Results stratification by concomitant emphysema(3 years)
- Time to progression in patients with initially non-clinically significant disease.(3 year)
- Long-term follow-up(5 years)