Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis

Study Description

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Brief Summary:

Idiopathic pulmonary fibrosis (IPF) is a progressing scarring disease of the lungs with an average survival of only 30-36 months since the time of diagnosis. The clinical course of IPF is highly variable, with some patients remaining stable for a prolonged period of time, even in the absence of medical treatment, while others experience rapid and relentless progression. In some cases, the clinical course consists of a stepwise rather than steady decline, with periods of stability alternating with acute respiratory worsening. The variability in clinical course makes it challenging to define prognosis in these patients and, importantly, to determine the right time window for lung transplantation (LTx) referral, listing and priority status on the waiting list.

The risk stratification score (RISE) is a new staging system for IPF based on the MRC dyspnea score, on physiology variables (pulmonary function tests) captured in the Composite Physiologic Index (CPI), and on the 6-minute walk distance. RISE showed to predict survival in newly diagnosed patients with IPF better than any other individual clinical, functional or radiographic variable. RISE also predicted survival on the waiting list in patients assessed and listed for LTx.

Condition or disease
Idiopathic Pulmonary Fibrosis

Detailed Description:

The objective of this study is to validate the RISE as a reliable tool to predict survival in patients newly diagnosed with IPF and prospectively followed for a period of 3 years, or until death or lung transplant.

This is an observational, prospective cohort study.

Inclusion criterion will be a new diagnosis of IPF based on the American Thoracic Society/European Respiratory Society criteria (Am J Respir Crit Care Med 2011;183:788-824). A pattern consistent with IPF will be confirmed by expert radiologists, unless a surgical lung biopsy is available.

Patients newly diagnosed with IPF will be included in the study. At each visit, typically every 4 months, the MRC dyspnea score (MRCDS), the pulmonary function tests (FEV1, FVC and DLCO), and the 6-minute walk distance (6MWD) will be recorded. The RISE will be calculated ad described in Eur Respir J 2012;40:101-109.

MRCDS, PFTs and 6MWD are part of the standard of care in IPF and are routinely obtained at each visit of IPF patients in the ILD clinic (typically every 4 months).

Patients will be prospectively followed for a period of 3 years and mortality events will be recorded. Mortality will be the primary endpoint. Acute exacerbations (AEs) of IPF, as defined in AJR Am J Roentgenol 1997;168:79-83, will also be recorded. AEs will be the secondary endpoint. At the end of the study period, both baseline RISE and longitudinal changes of RISE will be tested as predictors of mortality. Other individual variables wil also be tested as predictors of mortality including age at the time of diagnosis, time between onset of symptoms and diagnosis (months), gender, body mass index, smoking history (pack-years), PFTs, 6MWD (meters and % predicted), and radiographic fibrosis score on high resolution chest CT scan (HRCT) at the time of diagnosis.

Values will expressed as mean±SD. Comparisons between survivors and non-survivors will be made with unpaired t-test or with the Mann-Whitney U-test, where appropriate. The optimal cut-off value for different variables to detect mortality or AE will assessed using receiver operating characteristics (ROC) analysis. Survival will evaluated using Kaplan-Meier curves and the log-rank test. Cox proportional hazards regression analysis will used to identify significant variables predicting survival status. Results will be summarized as hazard ratios, representing the relative risk of dying as a result of a specific characteristic during the observation period. Variables selected via univariate analysis (p<0.05) will evaluated in the multivariate Cox regression analysis. p-values <0.05 will regarded as significant.

Study Design

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Study Type :	Observational
Estimated Enrollment :	500 participants
Observational Model:	Cohort
Time Perspective:	Prospective
Official Title:	Validation of the Risk Stratification Score in Idiopathic Pulmonary Fibrosis
Actual Study Start Date :	December 2015
Estimated Primary Completion Date :	December 2019
Estimated Study Completion Date :	December 2022

Groups and Cohorts

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Group/Cohort
Idiopathic pulmonary fibrosis; Patients newly diagnosed with idiopathic pulmonary fibrosis

Outcome Measures

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Primary Outcome Measures :

1. Survival [ Time Frame: 3 years ]
   Primary outcome is 3-year survival since the time of diagnosis

Secondary Outcome Measures :

1. Acute exacerbations of IPF [ Time Frame: 3 years ]
   Secondary outcome is the incidence of acute exacerbations since the time of diagnosis

Eligibility Criteria

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Ages Eligible for Study:	18 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

Patients newly diagnosed with idiopathic pulmonary fibrosis.

Criteria

Inclusion Criteria:

• A new diagnosis of IPF based on the American Thoracic Society/European Respiratory Society criteria (Am J Respir Crit Care Med 2011;183:788-824) and confirmed by a panel of expert chest radiologists.

Exclusion Criteria:

• Interstitial lung disease other than IPF

Contacts and Locations

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Contacts

Contact: Marco Mura, MD, PhD	+1-5196676744	marco.mura@lhsc.on.ca

Locations

Canada, Ontario
London Health Science Centre	Recruiting
London, Ontario, Canada, N6A 5W9
Contact: Marco Mura, MD, PhD +15196676744 marco.mura@lhsc.on.ca
Contact: Karishma Hosein +15196858500 ext 55749 karishma.hosein@lhsc.on.ca
Principal Investigator: Marco Mura, MD, PhD

Sponsors and Collaborators

Lawson Health Research Institute

More Information

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Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Marco Mura, Associated Scientist, Lawson Health Research Institute
ClinicalTrials.gov Identifier:	NCT02632123 History of Changes
Other Study ID Numbers:	10010803
First Posted:	December 16, 2015
Last Update Posted:	March 6, 2018
Last Verified:	March 2018

Keywords provided by Marco Mura, Lawson Health Research Institute:

idiopathic pulmonary fibrosis; outcome; prognosis; survival

Additional relevant MeSH terms:

Fibrosis; Pulmonary Fibrosis; Idiopathic Pulmonary Fibrosis; Idiopathic Interstitial Pneumonias	Pathologic Processes; Lung Diseases; Respiratory Tract Diseases; Lung Diseases, Interstitial