Results of the standard set forpulmonary sarcoidosis: Feasibility and multicentre outcomes

Our study presents findings on a previously developed standard set of clinical outcome data for pulmonary sarcoidosis patients. We aimed to assess whether changes in outcome varied between the different centres and to evaluate the feasibility of collecting the standard set retrospectively. This retrospective observational comparative benchmark study included six interstitial lung disease expert centres based in the Netherlands, Belgium, the UK and the USA. The standard set of outcome measures included 1) mortality, 2) changes in pulmonary function (forced vital capacity (FVC), forced expiratory volume in 1 s, diffusing capacity of the lung for carbon monoxide), 3) soluble interleukin-2 receptor (sIL-2R) change, 4) weight changes, 5) quality-of-life (QoL) measures, 6) osteoporosis and 7) clinical outcome status (COS). Data collection was considered feasible if the data were collected in ⩾80% of all patients. 509 patients were included in the retrospective cohort. In total six patients died, with a mean survival of 38±23.4 months after the diagnosis. Centres varied in mean baseline FVC, ranging from 110 (95% CI 92–124)% predicted to 99 (95% CI 97–123)% pred. Mean baseline body mass index (BMI) of patients in the different centres varied between 27 (95% CI 23.6–29.4) kg·m−2 and 31.8 (95% CI 28.1–35.6) kg·m−2. 310 (60.9%) patients were still on systemic therapy 2 years after the diagnosis. It was feasible to measure mortality, changes in pulmonary function, weight changes and COS. It is not (yet) feasible to retrospectively collect sIL-2R, osteoporosis and QoL data internationally. This study shows that data collection for the standard set of outcome measures for pulmonary sarcoidosis was feasible for four out of seven outcome measures. Trends in pulmonary function and BMI were similar for different hospitals when comparing different practices

First patient-centred set of outcomes for pulmonary sarcoidosis: a multicentre initiative

Introduction Routine and international comparison of clinical outcomes enabling identification of best practices for patients with pulmonary sarcoidosis is lacking. The aim of this study was to develop a standard set of outcome measures for pulmonary sarcoidosis, using the valuebased healthcare principles. Methods Six expert clinics for interstitial lung diseases in four countries participated in a consensus-driven RANDmodified Delphi study. A mixed-method approach was applied for the identification of an outcome measures set and initial conditions for patients with pulmonary sarcoidosis. The expert team consisted of multidisciplinary professionals (n=14) from Cleveland Clinic, Cincinnati MC, Erasmus MC, Leuven UZ, Royal Brompton and St. Antonius Hospital. During a ranking process, participants were instructed to rank variables on a scale from 1 to 10 based on whether it has (1) impact of the outcome on quality of life, (2) impact of quality of care on the outcome and (3) the number of patients negatively affected by the outcome. Results An outcome measures set was defined consisting of seven outcome measures: mortality, pulmonary function, soluble interleukin-2 receptor change as an activity biomarker, weight gain, quality of life, osteoporosis and clinical outcome status. Discussion Collecting outcomes in pulmonary sarcoidosis internationally and the use of a broadly accepted set can enable international comparison. Differences in outcomes can potentially be used as a starting point for quality improvement initiatives

Predicting outcomes in rheumatoid arthritis related interstitial lung disease

Aims: To compare radiology-based prediction models in rheumatoid arthritis-related interstitial lung disease (RA-ILD) to identify patients with a progressive fibrosis phenotype.Methods: RAILD patients had CTs scored visually and by CALIPER and forced vital capacity (FVC) measurements. Outcomes were evaluated using three techniques: 1.Scleroderma system evaluating visual ILD extent and FVC values; 2.Fleischer Society IPF diagnostic guidelines applied to RAILD; 3.CALIPER scores of vessel-related structures (VRS). Outcomes were compared to IPF patients.Results: On univariable Cox analysis, all three staging systems strongly predicted outcome: Scleroderma System:HR=3.78, p=9×10-5; Fleischner System:HR=1.98, p=2×10-3; 4.4% VRS threshold:HR=3.10, p=4×10-4 When the Scleroderma and Fleischner Systems were combined, termed the Progressive Fibrotic System (C-statistic=0.71), they identified a patient subset (n=36) with a progressive fibrotic phenotype and similar 4-year survival to IPF.On multivariable analysis, with adjustment for patient age, gender and smoking status, when analysed alongside the Progressive Fibrotic System, the VRS threshold of 4.4% independently predicted outcome (Model C-statistic=0.77).Conclusions: The combination of two visual CT-based staging systems identified 23% of an RAILD cohort with an IPF-like progressive fibrotic phenotype. The addition of a computer-derived VRS threshold further improved outcome prediction and model fit, beyond that encompassed by RAILD measures of disease severity and extent

Delineating associations of progressive pleuroparenchymal fibroelastosis in patients with pulmonary fibrosis

BACKGROUND: Computer quantification of baseline computed tomography (CT) radiological pleuroparenchymal fibroelastosis (PPFE) associates with mortality in idiopathic pulmonary fibrosis (IPF). We examined mortality associations of longitudinal change in computer-quantified PPFE-like lesions in IPF and fibrotic hypersensitivity pneumonitis (FHP). METHODS: Two CT scans 6-36 months apart were retrospectively examined in one IPF (n=414) and one FHP population (n=98). Annualised change in computerised upper-zone pleural surface area comprising radiological PPFE-like lesions (Δ-PPFE) was calculated. Δ-PPFE >1.25% defined progressive PPFE above scan noise. Mixed-effects models evaluated Δ-PPFE against change in visual CT interstitial lung disease (ILD) extent and annualised forced vital capacity (FVC) decline. Multivariable models were adjusted for age, sex, smoking history, baseline emphysema presence, antifibrotic use and diffusion capacity of the lung for carbon monoxide. Mortality analyses further adjusted for baseline presence of clinically important PPFE-like lesions and ILD change. RESULTS: Δ-PPFE associated weakly with ILD and FVC change. 22-26% of IPF and FHP cohorts demonstrated progressive PPFE-like lesions which independently associated with mortality in the IPF cohort (hazard ratio 1.25, 95% CI 1.16-1.34, p<0.0001) and the FHP cohort (hazard ratio 1.16, 95% CI 1.00-1.35, p=0.045). INTERPRETATION: Progression of PPFE-like lesions independently associates with mortality in IPF and FHP but does not associate strongly with measures of fibrosis progression

Prognostic Imaging Biomarker Discovery in Survival Analysis for Idiopathic Pulmonary Fibrosis

25th International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI) -- SEP 18-22, 2022 -- Singapore, SINGAPOREImaging biomarkers derived from medical images play an important role in diagnosis, prognosis, and therapy response assessment. Developing prognostic imaging biomarkers which can achieve reliable survival prediction is essential for prognostication across various diseases and imaging modalities. In this work, we propose a method for discovering patch-level imaging patterns which we then use to predict mortality risk and identify prognostic biomarkers. Specifically, a contrastive learning model is first trained on patches to learn patch representations, followed by a clustering method to group similar underlying imaging patterns. The entire medical image can be thus represented by a long sequence of patch representations and their cluster assignments. Then a memory-efficient clustering Vision Transformer is proposed to aggregate all the patches to predict mortality risk of patients and identify high-risk patterns. To demonstrate the effectiveness and generalizability of our model, we test the survival prediction performance of our method on two sets of patients with idiopathic pulmonary fibrosis (IPF), a chronic, progressive, and life-threatening interstitial pneumonia of unknown etiology. Moreover, by comparing the high-risk imaging patterns extracted by our model with existing imaging patterns utilised in clinical practice, we can identify a novel biomarker that may help clinicians improve risk stratification of IPF patients.CSC-UCL Joint Research Scholarship; UK EPSRC [M020533, R006032, R014019, V034537]; Wellcome Trust [UNS113739]; Wellcome Trust Clinical Research Career Development Fellowship [209,553/Z/17/Z]; NIHR UCLH Biomedical Research Centre, UKMICCAI SocAZ is supported by CSC-UCL Joint Research Scholarship. DCA is supported by UK EPSRC grants M020533, R006032, R014019, V034537, Wellcome Trust UNS113739. JJ is supported by Wellcome Trust Clinical Research Career Development Fellowship 209,553/Z/17/Z. DCA and JJ are supported by the NIHR UCLH Biomedical Research Centre, UK

Delineating associations of progressive pleuroparenchymal fibroelastosis in patients with pulmonary fibrosis

Background Computer quantification of baseline computed tomography (CT) radiological pleuroparenchymal fibroelastosis (PPFE) associates with mortality in idiopathic pulmonary fibrosis (IPF). We examined mortality associations of longitudinal change in computer-quantified PPFE-like lesions in IPF and fibrotic hypersensitivity pneumonitis (FHP). Methods Two CT scans 6-36 months apart were retrospectively examined in one IPF (n=414) and one FHP population (n=98). Annualised change in computerised upper-zone pleural surface area comprising radiological PPFE-like lesions (Delta-PPFE) was calculated.Delta-PPFE >1.25% defined progressive PPFE above scan noise. Mixed-effects models evaluated Delta-PPFE against change in visual CT interstitial lung disease (ILD) extent and annualised forced vital capacity (FVC) decline. Multivariable models were adjusted for age, sex, smoking history, baseline emphysema presence, antifibrotic use and diffusion capacity of the lung for carbon monoxide. Mortality analyses further adjusted for baseline presence of clinically important PPFE-like lesions and ILD change. Results Delta-PPFE associated weakly with ILD and FVC change. 22-26% of IPF and FHP cohorts demonstrated progressive PPFE-like lesions which independently associated with mortality in the IPF cohort (hazard ratio 1.25, 95% CI 1.16-1.34, p0.0001) and the FHP cohort (hazard ratio 1.16, 95% CI 1.00-1.35, p=0.045). Interpretation Progression of PPFE-like lesions independently associates with mortality in IPF and FHP but does not associate strongly with measures of fibrosis progression.This research was funded in whole or in part by the Wellcome Trust (209553/Z/17/Z). This project, J. Jacob, E. Gudmundsson, E. Denneny, J. Porter and S.M. Janes were also supported by the NIHR UCLH Biomedical Research Centre, UK. M.G. Jones, T. Wallis and C.J. Brereton acknowledge the support of the NIHR Southampton Biomedical Research Centre. Funding information for this article has been deposited with the Crossref Funder Registry.Wellcome Trust [209553/Z/17/Z]; NIHR UCLH Biomedical Research Centre, UK; NIHR Southampton Biomedical Research Centr

The importance of biobank and nationwide registry for lymphangioleiomyomatosis in a small sized country

Introduction: Patient registries combined with the collection of human specimens in biobanks are of great value for facilitating research on orphan diseases, allowing researchers to obtain valid and reliable epidemiological information. This results in an increase of knowledge on epidemiology, course of disease, clinical presentation and underlying biomarkers. The biobank in the St. Antonius Hospital in Nieuwegein, the Netherlands, is used for nationwide registry of patients with lymphangioleiomyomatosis (LAM), an ultra rare interstitial lung disease characterized by smooth muscle proliferation in the lungs. Areas covered: This paper provides a review of the global prevalence and disease characteristics of LAM. Additionally, disease characteristics of patients derived from the nationwide registry in the Netherlands will be described and compared with the literature. Expert opinion: A nationwide registry and biobank for ultra rare diseases such as LAM allows for country-specific insight in epidemiology and clinical characteristics. Active cooperation between health care professionals and patients is of utmost importance to increase general awareness and successful coverage ratio of inclusions. The Dutch LAM registry and biobank will now be able to participate in the global LAM registry of the LAM Foundation International Clinics program, with the purpose of identifying patients and availability for trials and bio-specimens for basic research worldwide, when possible

PATTERNS OF HEALTHCARE RESOURCE UTILIZATION IN PATIENTS WITH SARCOIDOSIS: A CROSS-SECTIONAL STUDY

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