scholarly journals Large-scale external validation and comparison of prognostic models: an application to chronic obstructive pulmonary disease

BMC Medicine ◽  
2018 ◽  
Vol 16 (1) ◽  
Author(s):  
Beniamino Guerra ◽  
◽  
Sarah R. Haile ◽  
Bernd Lamprecht ◽  
Ana S. Ramírez ◽  
...  
BMJ ◽  
2019 ◽  
pp. l5358 ◽  
Author(s):  
Vanesa Bellou ◽  
Lazaros Belbasis ◽  
Athanasios K Konstantinidis ◽  
Ioanna Tzoulaki ◽  
Evangelos Evangelou

Abstract Objective To map and assess prognostic models for outcome prediction in patients with chronic obstructive pulmonary disease (COPD). Design Systematic review. Data sources PubMed until November 2018 and hand searched references from eligible articles. Eligibility criteria for study selection Studies developing, validating, or updating a prediction model in COPD patients and focusing on any potential clinical outcome. Results The systematic search yielded 228 eligible articles, describing the development of 408 prognostic models, the external validation of 38 models, and the validation of 20 prognostic models derived for diseases other than COPD. The 408 prognostic models were developed in three clinical settings: outpatients (n=239; 59%), patients admitted to hospital (n=155; 38%), and patients attending the emergency department (n=14; 3%). Among the 408 prognostic models, the most prevalent endpoints were mortality (n=209; 51%), risk for acute exacerbation of COPD (n=42; 10%), and risk for readmission after the index hospital admission (n=36; 9%). Overall, the most commonly used predictors were age (n=166; 41%), forced expiratory volume in one second (n=85; 21%), sex (n=74; 18%), body mass index (n=66; 16%), and smoking (n=65; 16%). Of the 408 prognostic models, 100 (25%) were internally validated and 91 (23%) examined the calibration of the developed model. For 286 (70%) models a model presentation was not available, and only 56 (14%) models were presented through the full equation. Model discrimination using the C statistic was available for 311 (76%) models. 38 models were externally validated, but in only 12 of these was the validation performed by a fully independent team. Only seven prognostic models with an overall low risk of bias according to PROBAST were identified. These models were ADO, B-AE-D, B-AE-D-C, extended ADO, updated ADO, updated BODE, and a model developed by Bertens et al. A meta-analysis of C statistics was performed for 12 prognostic models, and the summary estimates ranged from 0.611 to 0.769. Conclusions This study constitutes a detailed mapping and assessment of the prognostic models for outcome prediction in COPD patients. The findings indicate several methodological pitfalls in their development and a low rate of external validation. Future research should focus on the improvement of existing models through update and external validation, as well as the assessment of the safety, clinical effectiveness, and cost effectiveness of the application of these prognostic models in clinical practice through impact studies. Systematic review registration PROSPERO CRD42017069247


2020 ◽  
Vol 48 (8) ◽  
pp. 030006052094550
Author(s):  
Jia Yang ◽  
Junchao Yang

Objective This study aimed to evaluate the effect of clearing heat and resolving phlegm for acute exacerbation of chronic obstructive pulmonary disease with the syndrome of phlegm-heat obstruction of the lung. Methods This was a real-world retrospective cohort study of inpatients at our institution from 1 January 2015 to 31 December 2017. The patients were divided into two groups according to whether they received oral traditional Chinese medicine (TCM) for clearing heat and resolving phlegm or routine treatment (controls). Efficacy and safety indicators were analyzed. Propensity score matching was used to control for confounding factors. Results Among 488 patients, 164 (82 pairs) were successfully matched. The changes in neutrophils (%) and C-reactive protein levels were more significant in the TCM group than in the control group. The duration of fever was significantly shorter in the TCM group than in the control group. Conclusions The therapy of clearing heat and resolving phlegm might effectively control the inflammatory reaction of acute exacerbation of chronic obstructive pulmonary disease in patients with the syndrome of phlegm-heat obstruction of the lung, especially for those with fever. Nevertheless, large-scale and prospective studies are required to provide a higher quality of evidence.


2020 ◽  
Vol 40 (6) ◽  
Author(s):  
Xi-Juan Zhang ◽  
Zhong-Hua Cui ◽  
Yan Dong ◽  
Xiu-Wen Liang ◽  
Yan-Xin Zhao ◽  
...  

Abstract Osteoporosis (OP) is significant and debilitating comorbidity of chronic obstructive pulmonary disease (COPD). We hypothesize that genetic variance identified with OP may also play roles in COPD. We have conducted a large-scale relation data analysis to explore the genes implicated with either OP or COPD, or both. Each gene linked to OP but not to COPD was further explored in a mega-analysis and partial mega-analysis of 15 independently collected COPD RNA expression datasets, followed by gene set enrichment analysis (GSEA) and literature-based pathway analysis to explore their functional linked to COPD. A multiple linear regression (MLR) model was built to study the possible influence of sample size, population region, and study date on the gene expression data in COPD. At the first step of the analysis, we have identified 918 genes associated with COPD, 581 with OP, and a significant overlap (P<2.30e-140; 210 overlapped genes). Partial mega-analysis showed that, one OP gene, GPNMB presented significantly increased expression in COPD patients (P-value = 0.0018; log fold change = 0.83). GPNMB was enriched in multiple COPD pathways and plays roles as a gene hub formulating multiple vicious COPD pathways included gene MMP9 and MYC. GPNMB could be a novel gene that plays roles in both COPD and OP. Partial mega-analysis is valuable in identify case-specific genes for COPD.


2018 ◽  
Vol 16 ◽  
pp. 147997311881569 ◽  
Author(s):  
Preyanate Wilairat ◽  
Kirati Kengkla ◽  
Chaiyawat Thayawiwat ◽  
Phongsathorn Phlaisaithong ◽  
Supakorn Somboonmee ◽  
...  

To examine clinical outcomes of theophylline use in patients with chronic obstructive pulmonary disease (COPD) receiving inhaled corticosteroids (ICS) and long-acting beta-2 agonists (LABA). Electronic data from five hospitals located in Northern Thailand between January 2011 and December 2015 were retrospectively collected. Propensity score (PS) matching (2:1 ratio) technique was used to minimize confounding factors. The primary outcome was overall exacerbations. Secondary outcomes were exacerbation not leading to hospital admission, hospitalization for exacerbation, hospitalization for pneumonia, and all-cause hospitalizations. Cox’s proportional hazards models were used to estimate adjusted hazard ratio (aHR) and 95% confidence interval (CI). After PS matching, of 711 patients with COPD (mean age: 70.1 years; 74.4% male; 60.8% severe airflow obstruction), 474 theophylline users and 237 non-theophylline users were included. Mean follow-up time was 2.26 years. Theophylline significantly increased the risk of overall exacerbation (aHR: 1.48, 95% CI: 1.11–1.96; p = 0.008) and exacerbation not leading to hospital admission (aHR: 1.47, 95% CI: 1.06–2.03; p = 0.020). Theophylline use did not significantly increase the risk of hospitalization for exacerbation (aHR: 1.11, 95% CI: 0.79–1.58; p = 0.548), hospitalization for pneumonia (aHR: 1.28, 95% CI: 0.89–1.84; p = 0.185), and all-cause hospitalizations (aHR: 1.03, 95% CI: 0.80–1.33; p = 0.795). Theophylline use as add-on therapy to ICS and LABA might be associated with an increased risk for overall exacerbation in patients with COPD. A large-scale prospective study of theophylline use investigating both safety and efficacy is warranted.


2019 ◽  
Author(s):  
Almaz Sharman ◽  
Baurzhan Zhussupov ◽  
Dana Sharman ◽  
Irina Kim

BACKGROUND Chronic obstructive pulmonary disease (COPD) is a global public health problem, and continuous monitoring is essential for both its management as well as the management of other chronic diseases. Telemonitoring using mobile health (mHealth) devices has the potential to promote self-management, improve control, increase quality of life, and prevent hospital admissions. OBJECTIVE This study aims to demonstrate whether a large-scale study assessing the use of mHealth devices to improve the treatment, assessment, compliance, and outcomes of chronic diseases, particularly COPD and cardio-metabolic syndrome, is feasible. This will allow our team to select the appropriate design and characteristics for our large-scale study. METHODS A total of 3 cohorts, with 9 participants in each, will use mHealth devices for 90 days while undergoing the current standard of care. These groups are: 9 “non-COPD,” otherwise healthy, smokers; 9 “grey zone” smokers (forced expiratory volume in 1 second/ forced vital capacity ≥0.70 after bronchodilator treatment; COPD Assessment Test ≥10); and 9 smokers diagnosed with Stage 1-3 COPD. Rates of recruitment, retention, and adherence will be measured. Overall, two mHealth devices will be utilized in the study: the AnaMed Original Equipment Manufacturer device (measures distance, energy expenditure, heart rate, and heart rate variability) and the Air Next mobile spirometry device. The mHealth devices will be compared against industry standards. Additionally, a questionnaire will be administered to assess the participants’ perceptions of the mHealth technologies used. RESULTS The inclusion of participants started in June 2019. Study results will be published in peer-reviewed scientific journals. CONCLUSIONS This study will demonstrate whether a large-scale study to assess the use of mHealth devices to improve the treatment, assessment, compliance, and outcomes of chronic diseases, particularly COPD and cardio-metabolic syndrome, is feasible. It will also allow the research team to select the appropriate design and characteristics for the large-scale study. CLINICALTRIAL ClinicalTrials.gov NCT04081961; https://clinicaltrials.gov/ct2/show/NCT04081961 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/16461


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