scholarly journals Validation of the risk stratification score in idiopathic pulmonary fibrosis: study protocol of a prospective, multi-centre, observational, 3-year clinical trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Gian Marco Manzetti ◽  
Karishma Hosein ◽  
Matthew J. Cecchini ◽  
Keith Kwan ◽  
Mohamed Abdelrazek ◽  
...  
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Risk Stratification ◽  
Clinical Decision Making ◽  
Pulmonary Function Tests ◽  
Prognostic Score ◽  
Walking Distance ◽  
Wide Range ◽  
Patient Reported ◽  
Risk Stratification Score

Abstract Background Idiopathic pulmonary fibrosis (IPF) is characterized by a poor prognosis, with a progressive decline in lung function and considerable variability in the disease’s natural history. Besides lung transplantation (LTx), the only available treatments are anti-fibrosing drugs, which have shown to slow down 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 in the development of 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. Methods This is an observational, multi-centre, prospective cohort study, designed to reflect common clinical practice in IPF. 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, MRC dyspnea score, pulmonary function tests (FEV1, FVC and DLCO), and 6-min walking distance will be recorded. 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 or emphysema. Discussion The objective of this study is to validate 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. Trial registration: U.S National Library of Medicine Clinicaltrials.gov, trial n. NCT02632123 “Validation of the risk stratification score in idiopathic pulmonary fibrosis”. Date of registration: December 16th, 2015.

scholarly journals Patient reported distress can aid clinical decision making in idiopathic pulmonary fibrosis: analysis of the PROFILE cohort

2018 ◽  
Author(s):  
Iain Stewart ◽  
Tricia McKeever ◽  
Rebecca Braybrooke ◽  
Eunice Oballa ◽  
Juliet K Simpson ◽  
...  
Keyword(s):  
Decision Making ◽  
Lung Function ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Disease Progression ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Risk Of Death ◽  
Patient Reported ◽  
Incident Cases

AbstractIdiopathic pulmonary fibrosis is a progressive and fatal interstitial lung disease. We aimed to determine if patient response to a palliative assessment survey could predict disease progression or death.We undertook a cross-sectional study in a UK clinical cohort of incident cases. Rasch-based methodology provided a disease distress value from an abridged 11 item model of the original 45 item survey. Distress values were compared with measures of lung function. Disease progression or mortality alone was predicted at twelve months from survey completion, with risk of death assessed at three, six and twelve months.Disease distress values were negatively correlated with lung function (r=-0.275 percent predicted DLCO). Expected survey scores computed from distress values could distinguish disease progression, 8.8 (p=0.004), and people who died, 10.2 (p=0.002), from those who did not progress, 6.9. Actual survey scores predicted disease progression and mortality with an area under the curve of 0.60 and 0.64, respectively. Each point increment in actual score increased risk of twelve-month mortality by 10%, almost 43% of people scoring above 18 did not survive beyond 105 days.We define a short questionnaire that can score disease distress and predict prognosis, assisting clinical decision making in progressive fibrosis.

scholarly journals Minimal clinically important difference in idiopathic pulmonary fibrosis

Breathe ◽  
2021 ◽  
Vol 17 (2) ◽  
pp. 200345
Author(s):  
Mohleen Kang ◽  
Lucian Marts ◽  
Jordan A. Kempker ◽  
Srihari Veeraraghavan
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Outcome Measures ◽  
Minimal Clinically Important Difference ◽  
Pulmonary Function Tests ◽  
Threshold Value ◽  
Patient Reported Outcome Measures ◽  
List Type ◽  
Clinically Important Difference ◽  
Patient Reported

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive fibrosing lung disease with an estimated median survival of 2–5 years and a significant impact on quality of life (QoL). Current approved medications, pirfenidone and nintedanib, have shown a reduction in annual decline of forced vital capacity but no impact on QoL. The minimal clinically important difference (MCID) is a threshold value for a change in a parameter that is considered meaningful by the patient rather than solely relying on statistically significant change in the parameter. This review provides a brief overview of the MCID methodology along with detailed discussion of reported MCID values for commonly used physiological measures and patient-reported outcome measures in IPF. While there is no gold standard methodology for determining MCID, there are certain limitations in the MCID literature in IPF, mainly the choice of death, hospitalisation and pulmonary function tests as sole anchors, and pervasive use of distribution-based methods which do not take into account the patient's input. There is a critical need to identify accurate thresholds of outcome measures that reflect patient's QoL over time in order to more precisely design and evaluate future clinical trials and to develop algorithms for patient-oriented management of IPF in outpatient clinics.Educational aimsTo understand the concept of MCID and the methods used to determine these values.To understand the indications and limitations of MCID values in IPF.

scholarly journals Assessing the Effectiveness of Pirfenidone in Idiopathic Pulmonary Fibrosis: Long-Term, Real-World Data from European IPF Registry (eurIPFreg)

2020 ◽  
Vol 9 (11) ◽  
pp. 3763
Author(s):  
Ekaterina Krauss ◽  
Silke Tello ◽  
Jochen Wilhelm ◽  
Johanna Schmidt ◽  
Mark Stoehr ◽  
...  
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Real World ◽  
Global Analysis ◽  
Pulmonary Function Tests ◽  
Outcome Data ◽  
Longitudinal Change ◽  
Walking Distance ◽  
Rapid Progression ◽  
Real World Data

Background: Idiopathic pulmonary fibrosis (IPF) is a chronic progressive fibrotic pulmonary disease with rising incidence. In this study the effectiveness of pirfenidone, as measured by longitudinal change in individual slope of forced vital capacity (FVC) prior to and after initiating pirfenidone treatment, was evaluated in IPF patients recruited into the European registry for idiopathic pulmonary fibrosis (eurIPFreg). Secondary variables were the evaluation of the change in individual slope of diffusion capacity of the lungs for carbon monoxide (DLco), the Borg dyspnea scale, and six-minute walking distance (6MWD), as well as survival analyses. Results: Data of 122 eurIPFreg patients, who had at least two pulmonary function tests (PFTs) prior to or under treatment with pirfenidone, were analyzed by calculating slope-changes. The global analysis revealed an average slope change of +1.48 ± 0.28 (% per annum (p.a)) after start of treatment (p < 0.001), reflecting a reduction in annual FVC decline of approx. 50% under pirfenidone; it also showed a reduction in DLco, and increase in 6MWD (both p < 0.0001), as well as a flattening of the Borg dyspnea scale (p = 0.02). The median survival under treatment was 4.82 years. Patients with a more restrictive disease (FVC < 80% pred.), with a rapid progression (FVC decline >10% pred. p.a.), previous smokers and patients > 60 years of age seemed to profit more from pirfenidone treatment. Conclusions: We report the effectiveness of pirfenidone in a European “real world” IPF cohort with outcome data extending up to 9 years. Global analyses demonstrated a positive effect of pirfenidone on the decline of the lung function over time. Survival was dependent on Gender–Age–Physiology (GAP) score and age prior to therapy.

Pulmonary Function Tests and Idiopathic Pulmonary Fibrosis

CHEST Journal ◽  
1997 ◽  
Vol 111 (1) ◽  
pp. 7-8 ◽  
Author(s):  
Steven H. Kirtland ◽  
Richard H. Winterbauer
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Pulmonary Function ◽  
Pulmonary Function Tests ◽  
Function Tests

scholarly journals Baseline characteristics of idiopathic pulmonary fibrosis: analysis from the Australian Idiopathic Pulmonary Fibrosis Registry

2017 ◽  
Vol 49 (2) ◽  
pp. 1601592 ◽  
Author(s):  
Helen E. Jo ◽  
Ian Glaspole ◽  
Christopher Grainge ◽  
Nicole Goh ◽  
Peter M.A. Hopkins ◽  
...  
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Disease Severity ◽  
Large Population ◽  
Underlying Disease ◽  
National Registry ◽  
Hazard Ratio ◽  
Wide Range ◽  
Baseline Characteristics

The prevalence of idiopathic pulmonary fibrosis (IPF), a fatal and progressive lung disease, is estimated at 1.25–63 out of 100 000, making large population studies difficult. Recently, the need for large longitudinal registries to study IPF has been recognised.The Australian IPF Registry (AIPFR) is a national registry collating comprehensive longitudinal data of IPF patients across Australia. We explored the characteristics of this IPF cohort and the effect of demographic and physiological parameters and specific management on mortality.Participants in the AIPFR (n=647, mean age 70.9±8.5 years, 67.7% male, median follow up 2 years, range 6 months–4.5 years) displayed a wide range of age, disease severity and co-morbidities that is not present in clinical trial cohorts. The cumulative mortality rate in year one, two, three and four was 5%, 24%, 37% and 44% respectively. Baseline lung function (forced vital capacity, diffusing capacity of the lung for carbon monoxide, composite physiological index) and GAP (gender, age, physiology) stage (hazard ratio 4.64, 95% CI 3.33–6.47, p<0.001) were strong predictors of mortality. Patients receiving anti-fibrotic medications had better survival (hazard ratio 0.56, 95% CI 0.34–0.92, p=0.022) than those not on anti-fibrotic medications, independent of underlying disease severity.The AIPFR provides important insights into the understanding of the natural history and clinical management of IPF.

scholarly journals Quantification of pulmonary perfusion in idiopathic pulmonary fibrosis with first pass dynamic contrast-enhanced perfusion MRI

Thorax ◽  
2020 ◽  
pp. thoraxjnl-2019-214375
Author(s):  
Nicholas D Weatherley ◽  
James A Eaden ◽  
Paul J C Hughes ◽  
Matthew Austin ◽  
Laurie Smith ◽  
...  
Keyword(s):  
Gas Exchange ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Pulmonary Function Tests ◽  
Pulmonary Vascular Disease ◽  
Bolus Transit ◽  
Dce Mri ◽  
Dynamic Contrast Enhanced ◽  
Contrast Enhanced ◽  
First Pass

IntroductionIdiopathic pulmonary fibrosis (IPF) is a fatal disease of lung scarring. Many patients later develop raised pulmonary vascular pressures, sometimes disproportionate to the interstitial disease. Previous therapeutic approaches that have targeted pulmonary vascular changes have not demonstrated clinical efficacy, and quantitative assessment of regional pulmonary vascular involvement using perfusion imaging may provide a biomarker for further therapeutic insights.MethodsWe studied 23 participants with IPF, using dynamic contrast-enhanced MRI (DCE-MRI) and pulmonary function tests, including forced vital capacity (FVC), transfer factor (TLCO) and coefficient (KCO) of the lungs for carbon monoxide. DCE-MRI parametric maps were generated including the full width at half maximum (FWHM) of the bolus transit time through the lungs. Key metrics used were mean (FWHMmean) and heterogeneity (FWHMIQR). Nineteen participants returned at 6 months for repeat assessment.ResultsSpearman correlation coefficients were identified between TLCO and FWHMIQR (r=−0.46; p=0.026), KCO and FWHMmean (r=−0.42; p=0.047) and KCO and FWHMIQR (r=−0.51; p=0.013) at baseline. No statistically significant correlations were seen between FVC and DCE-MRI metrics. Follow-up at 6 months demonstrated statistically significant decline in FVC (p=0.040) and KCO (p=0.014), with an increase in FWHMmean (p=0.040), but no significant changes in TLCO (p=0.090) nor FWHMIQR (p=0.821).ConclusionsDCE-MRI first pass perfusion demonstrates correlations with existing physiological gas exchange metrics, suggesting that capillary perfusion deficit (as well as impaired interstitial diffusion) may contribute to gas exchange limitation in IPF. FWHMmean showed a significant increase over a 6-month period and has potential as a quantitative biomarker of pulmonary vascular disease progression in IPF.

scholarly journals Serum biomarker CA 15-3 as predictor of response to antifibrotic treatment and survival in idiopathic pulmonary fibrosis

2020 ◽  
Vol 14 (11) ◽  
pp. 997-1007
Author(s):  
Sofia A Moll ◽  
Ivo A Wiertz ◽  
Adriane DM Vorselaars ◽  
Pieter Zanen ◽  
Henk JT Ruven ◽  
...  
Keyword(s):  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Pulmonary Function ◽  
Vital Capacity ◽  
Mixed Model ◽  
Pulmonary Function Tests ◽  
Hazard Ratio ◽  
Poor Survival ◽  
Function Test

Aim: Cancer antigen 15-3 (CA 15-3) is a baseline biomarker in idiopathic pulmonary fibrosis (IPF), but its value during follow-up is unknown. Materials and methods: Associations between serum CA 15-3 and pulmonary function tests during 1-year follow-up were evaluated by a mixed model in 132 IPF treated with pirfenidone or nintedanib. Results: Increased baseline (median: 56 kU/l) and follow-up CA 15-3 levels were inversely associated with forced vital capacity and diffusing capacity of the lung for carbon monoxide (estimates respectively: -5.21 and -4.69; p < 0.001). Baseline and 6-month CA 15-3 above 58.5 (hazard ratio: 1.67; p = 0.031) and 50.5 kU/l (hazard ratio: 2.99; p < 0.001), respectively, showed impaired survival compared with lower levels. Conclusion: CA 15-3 is associated with pulmonary function test during follow-up in IPF on antifibrotic treatment. Higher (follow-up) values are related with poor survival. Therefore, CA 15-3 is a promising follow-up biomarker in IPF.

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