Pragmatic and Observational Research最新文献

{"title":"Real-World Effectiveness of First Line Lenvatinib Therapy in Advanced Hepatocellular Carcinoma: Current Insights.","authors":"Tiago Biachi de Castria, Richard D Kim","doi":"10.2147/POR.S395974","DOIUrl":"10.2147/POR.S395974","url":null,"abstract":"<p><p>Lenvatinib received its initial approval in 2018 for the treatment of advanced hepatocellular carcinoma. It has since emerged as the preferred first line agent, supported by non-inferiority data from the REFLECT trial. Notably, lenvatinib exhibits a more favorable toxicity profile and a higher response rate compared to sorafenib. Despite the approval of immunotherapy in 2020, specifically the combination of atezolizumab and bevacizumab following the IMbrave150 trial, tyrosine kinase inhibitors remain an indispensable class of agents in the landscape of hepatocellular carcinoma treatment. This comprehensive review delves into various facets of lenvatinib utilization in hepatocellular carcinoma, shedding light on real-world data, addressing challenges, and providing insights into strategies to overcome these obstacles.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"79-87"},"PeriodicalIF":8.9,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178097/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141332914","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Using Claims Data to Predict Pre-Operative BMI Among Bariatric Surgery Patients: Development of the BMI Before Bariatric Surgery Scoring System (B3S3).","authors":"Jenna Wong, Xiaojuan Li, David E Arterburn, Dongdong Li, Elizabeth Messenger-Jones, Rui Wang, Sengwee Toh","doi":"10.2147/POR.S450229","DOIUrl":"10.2147/POR.S450229","url":null,"abstract":"<p><strong>Background: </strong>Lack of body mass index (BMI) measurements limits the utility of claims data for bariatric surgery research, but pre-operative BMI may be imputed due to existence of weight-related diagnosis codes and BMI-related reimbursement requirements. We used a machine learning pipeline to create a claims-based scoring system to predict pre-operative BMI, as documented in the electronic health record (EHR), among patients undergoing a new bariatric surgery.</p><p><strong>Methods: </strong>Using the Optum Labs Data Warehouse, containing linked de-identified claims and EHR data for commercial or Medicare Advantage enrollees, we identified adults undergoing a new bariatric surgery between January 2011 and June 2018 with a BMI measurement in linked EHR data ≤30 days before the index surgery (n=3226). We constructed predictors from claims data and applied a machine learning pipeline to create a scoring system for pre-operative BMI, the B3S3. We evaluated the B3S3 and a simple linear regression model (benchmark) in test patients whose index surgery occurred concurrent (2011-2017) or prospective (2018) to the training data.</p><p><strong>Results: </strong>The machine learning pipeline yielded a final scoring system that included weight-related diagnosis codes, age, and number of days hospitalized and distinct drugs dispensed in the past 6 months. In concurrent test data, the B3S3 had excellent performance (R² 0.862, 95% confidence interval [CI] 0.815-0.898) and calibration. The benchmark algorithm had good performance (R² 0.750, 95% CI 0.686-0.799) and calibration but both aspects were inferior to the B3S3. Findings in prospective test data were similar.</p><p><strong>Conclusion: </strong>The B3S3 is an accessible tool that researchers can use with claims data to obtain granular and accurate predicted values of pre-operative BMI, which may enhance confounding control and investigation of effect modification by baseline obesity levels in bariatric surgery studies utilizing claims data.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"65-78"},"PeriodicalIF":8.9,"publicationDate":"2024-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10981874/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140336646","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trends in Systemic Glucocorticoid Utilization in the United Kingdom from 1990 to 2019: A Population-Based, Serial Cross-Sectional Analysis.","authors":"Andrew N Menzies-Gow, Trung N Tran, Brooklyn Stanley, Victoria Ann Carter, Josef S Smolen, Arnaud Bourdin, J Mark Fitzgerald, Tim Raine, Jatin Chapaneri, Benjamin Emmanuel, David J Jackson, David B Price","doi":"10.2147/POR.S442959","DOIUrl":"https://doi.org/10.2147/POR.S442959","url":null,"abstract":"<p><strong>Purpose: </strong>Associations between systemic glucocorticoid (SGC) exposure and risk for adverse outcomes have spurred a move toward steroid-sparing treatment strategies. Real-world changes in SGC exposure over time, after the introduction of steroid-sparing treatment strategies, reveal areas of successful risk mitigation as well as unmet needs.</p><p><strong>Patients and methods: </strong>A population-based ecological study was performed from the Optimum Patient Care Research Database to describe SGC prescribing trends of steroid-sparing treatment strategies in primary care practices before and after licensure of biologics in the United Kingdom from 1990 to 2019. Each analysis year included patients aged ≥5 years who were registered for ≥1 year with a participating primary care practice. The primary analysis was SGC exposure, defined as total cumulative SGC dose per patient per year, for asthma, severe asthma, chronic obstructive pulmonary disease (COPD), nasal polyps, Crohn's disease, rheumatoid arthritis, ulcerative colitis, and systemic lupus erythematosus. Secondary outcomes were percentages of patients prescribed SGCs and number of SGC prescriptions per patient per year.</p><p><strong>Results: </strong>The number of patients who met study inclusion criteria ranged from 219,862 (1990) to 1,261,550 (2019). At the population level, patients with asthma or COPD accounted for 67.7% to 73.2% of patients per year with an SGC prescription. Over three decades, decreases in SGC total yearly dose ≥1000 mg have been achieved in multiple conditions. Patients with COPD prescribed SGCs increased from 5.8% (1990) to 34.8% (2017). SGC prescribing trends for severe asthma, Crohn's disease, and ulcerative colitis show decreased prescribing trends after the introduction of biologics.</p><p><strong>Conclusion: </strong>Decreases in total yearly SGC doses have been shown in multiple conditions; however, for conditions such as severe asthma and COPD, an unmet need remains for increased awareness of SGC burden and the adoption or development of SGC-sparing alternatives to reduce overuse.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"53-64"},"PeriodicalIF":8.9,"publicationDate":"2024-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10949995/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140176122","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Difficult-To-Treat and Severe Asthma: Can Real-World Studies On Effectiveness of Biological Treatments Change the Lives of Patients?","authors":"Corrado Pelaia, Antonio Giacalone, Gianluca Ippolito, Daniela Pastore, Angelantonio Maglio, Giovanna Lucia Piazzetta, Nadia Lobello, Nicola Lombardo, Alessandro Vatrella, Girolamo Pelaia","doi":"10.2147/POR.S396799","DOIUrl":"10.2147/POR.S396799","url":null,"abstract":"<p><p>Many different phenotypes that characterize severe asthma are supported by intricate pathomechanisms called endotypes. The latter are driven by molecular interactions, mediated by intercellular networks. With regard to the biological treatments of either allergic or non-allergic eosinophilic type 2 asthma, real-world studies have confirmed the positive effects of currently available antibodies directed against immunoglobulins E (IgE), interleukin-5 (IL-5) and its receptor, as well as the receptors of interleukins-4 (IL-4) and 13 (IL-13). The best way to treat severe asthma should be chosen based on the peculiar phenotypic and endotypic traits of each patient. This will lead to relevant improvements in both clinical and functional outcomes. In particular, biological therapies can change the lives of asthma patients with a strong impact on quality of life. Unfortunately, patients with severe non-type-2 asthma, who continue to have pertinent unmet needs, are not receiving satisfactory advances within the context of biological treatments. It is also hopeful that in the next future new therapeutic strategies will be specifically implemented for these people, perhaps offering them the opportunity to improve their current, mostly inadequate asthma management.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"45-51"},"PeriodicalIF":8.9,"publicationDate":"2024-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10941791/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140143964","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Obesity Paradox in Heart Failure with Mildly Reduced Ejection Fraction.","authors":"Marielen Reinhardt, Tobias Schupp, Mohammad Abumayyaleh, Felix Lau, Alexander Schmitt, Noah Abel, Muharrem Akin, Jonas Rusnak, Ibrahim Akin, Michael Behnes","doi":"10.2147/POR.S444361","DOIUrl":"10.2147/POR.S444361","url":null,"abstract":"<p><strong>Objective: </strong>The study investigates the prognostic impact of body mass index (BMI) in patients hospitalized with heart failure with mildly reduced ejection fraction (HFmrEF).</p><p><strong>Background: </strong>Limited data regarding the prognostic impact of BMI in patients with HFmrEF is available.</p><p><strong>Methods: </strong>Consecutive patients with HFmrEF (ie, left ventricular ejection fraction 41-49% and signs and/or symptoms of HF) were retrospectively included at one institution from 2016 to 2022. Risk stratification was performed according to WHO-defined BMI groups. The primary endpoint was all-cause mortality at 30 months (median follow-up). Kaplan-Meier, uni- and multivariable Cox proportional regression analyses were applied for statistics.</p><p><strong>Results: </strong>1832 consecutive patients with HFmrEF were included with a median BMI of 26.7 kg/m² (IQR 24.0-30.8 kg/m²). Patients with lowest BMI (ie, 18.5-24.9 kg/m²) were associated with highest risk of all-cause mortality at 30 months compared to patients with higher BMI values (40.0% vs 29.0% vs 21.4% vs 20.9%; log rank p = 0.001; HR = 0.721; 95% CI 0.656-0.793; p = 0.001). Even after multivariable adjustment, higher BMI values were associated with improved survival at 30 months (HR = 0.963; 95% CI 0.943-0.985; p = 0.001). In contrast, the risk of HF- related rehospitalization at 30 months was not affected by BMI (log rank p = 0.064).</p><p><strong>Conclusion: </strong>In patients hospitalized with HFmrEF, lower BMI was associated with increased risk of all-cause mortality at 30 months, suggesting an obesity paradox in HFmrEF.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"31-43"},"PeriodicalIF":8.9,"publicationDate":"2024-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10933520/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140120427","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Drug Repurposing in Crohn's Disease Using Danish Real-World Data.","authors":"Saeed Shakibfar, Kristine H Allin, Tine Jess, Maria Antonietta Barbieri, Vera Battini, Eva Simoncic, Julien Kirchgesner, Trond Ulven, Maurizio Sessa","doi":"10.2147/POR.S444569","DOIUrl":"https://doi.org/10.2147/POR.S444569","url":null,"abstract":"<p><strong>Aim: </strong>Drug repurposing, utilizing electronic healthcare records (EHRs), offers a promising alternative by repurposing existing drugs for new therapeutic indications, especially for patients lacking effective therapies. Intestinal fibrosis, a severe complication of Crohn's disease (CD), poses significant challenges, increasing morbidity and mortality without available pharmacological treatments. This article focuses on identifying medications associated with an elevated or reduced risk of fibrosis in CD patients through a population-wide real-world data and artificial intelligence (AI) approach.</p><p><strong>Methods: </strong>Patients aged 65 or older with a diagnosis of CD from 1996 to 2019 in the Danish EHRs were followed for up to 24 years. The primary outcome was the need of specific surgical procedures, namely proctocolectomy with ileostomy and ileocecal resection as proxies of intestinal fibrosis. The study explored drugs linked to an increased or reduced risk of the study outcome through machine-learning driven survival analysis.</p><p><strong>Results: </strong>Among the 9179 CD patients, 1029 (11.2%) underwent surgery, primarily men (58.5%), with a mean age of 76 years, 10 drugs were linked to an elevated risk of surgery for proctocolectomy with ileostomy and ileocecal resection. In contrast, 10 drugs were associated with a reduced risk of undergoing surgery for these conditions.</p><p><strong>Conclusion: </strong>This study focuses on repurposing existing drugs to prevent surgery related to intestinal fibrosis in CD patients, using Danish EHRs and advanced statistical methods. The findings offer valuable insights into potential treatments for this condition, addressing a critical unmet medical need. Further research and clinical trials are warranted to validate the effectiveness of these repurposed drugs in preventing surgery related to intestinal fibrosis in CD patients.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"15 ","pages":"17-29"},"PeriodicalIF":8.9,"publicationDate":"2024-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10894518/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139973158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prediction Model with Validation for Polioseronegativity in Malnourished Children from Poliomyelitis Transmission High-Risk Area of the Democratic Republic of the Congo (DRC).","authors":"Guillaume Ngoie Mwamba, Michel Kabamba, Nicole A Hoff, Patrick K Mukadi, Kamy Kaminye Musene, Sue K Gerber, Megan Halbrook, Cyrus Sinai, Trevon Fuller, Arie Voorman, Paul Makan Mawaw, Oscar Luboya Numbi, Emile Okitolonda Wemakoy, Patricia N Mechael, Jean Jacques Muyembe Tamfum, Mala Ali Mapatano, Anne W Rimoin, Paul-Samson Lusamba Dikassa","doi":"10.2147/POR.S437485","DOIUrl":"10.2147/POR.S437485","url":null,"abstract":"<p><strong>Background: </strong>Malnutrition is identified as a risk factor for insufficient polio seroconversion in the context of a vaccine-derived poliovirus (VDPV) outbreak-prone region. In the Democratic Republic of Congo (DRC), underweight decreased from 31% (in 2001) to 26% (in 2018). Since 2004, VDPV serotype 2 outbreaks (cVDPV2) have been documented and were geographically limited around the Haut-Lomami and Tanganyika Provinces.</p><p><strong>Methods: </strong>To develop and validate a predictive model for poliomyelitis vaccine response in malnourished infants, a cross-sectional household study was carried out in the Haut-Lomami and Tanganyika provinces. Healthy children aged 6 to 59 months (n=968) were enrolled from eight health zones (HZ) out of 27, in March 2018. We performed a bivariate and multivariate logistics analysis. Final models were selected using a stepwise Wald method, and variables were selected based on the criterion p < 0.05. The association between nutritional variables, explaining polio seronegativity for the three serotypes, was assessed using the receiver operating characteristic curve (ROC curve).</p><p><strong>Results: </strong>Factors significantly associated with seronegativity to the three polio serotypes were underweight, non-administration of vitamin A, and the age group of 12 to 59 months. The sensitivity was 10.5%, and its specificity was 96.4% while the positive predictive values (PPV) and negative (PNV) were 62.7% and 65.3%, respectively. We found a convergence of the curves of the initial sample and two split samples. Based on the comparison of the overlapping confidence intervals of the ROC curve, we concluded that our prediction model is valid.</p><p><strong>Conclusion: </strong>This study proposed the first tool which variables are easy to collect by any health worker in charge of vaccination or in charge of nutrition. It will bring on top, the collaboration between the Immunization and the Nutritional programs in DRC integration policy, and its replicability in other low- and middle-income countries with endemic poliovirus.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"14 ","pages":"155-165"},"PeriodicalIF":2.3,"publicationDate":"2023-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10749540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139037944","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Avelumab for Advanced Merkel Cell Carcinoma: Global Real-World Data on Patient Response and Survival.","authors":"Rishabh Lohray, Kritin K Verma, Leo L Wang, Dylan Haynes, Daniel J Lewis","doi":"10.2147/POR.S398151","DOIUrl":"10.2147/POR.S398151","url":null,"abstract":"<p><strong>Introduction: </strong>Avelumab is a programmed cell death-ligand 1 (PD-L1) inhibitor approved by the Food and Drug Administration for advanced Merkel cell carcinoma (MCC). Studies conducted in real-world settings have shed light on its effectiveness and safety in clinical settings.</p><p><strong>Areas covered: </strong>Real-world studies on avelumab for MCC from North and South America, Europe, and Asia have been presented in this review. Most studies are on patients over age 70 and have a male-predominant sex ratio. Overall response rates range from 29.1% to 72.1%, (disease control rate: 60.0-72.7%; complete response rate: 15.8%-37.2%; partial rate: 18.2-42.1%; stable disease: 7.1-30.9%; progressive disease: 7.1-40.0%) and median progression free survival ranges from 8.1 to 24.1 months depending on the population studied. Immunosuppressed patients appear to benefit from avelumab as well, with response rates equivalent to the general population. Patients receiving avelumab as a first-line agent tend to have better outcomes than those using it as a second-line therapy. Fatigue, infusion-related reactions, and dyspnea were some of the most common adverse events identified in real-world studies. Autoimmune hepatitis and thyroiditis were also observed.</p><p><strong>Conclusion: </strong>The use of avelumab as a safe and effective treatment option for advanced MCC is supported by real-world data, although additional study is required to assess long-term efficacy and safety outcomes.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"14 ","pages":"149-154"},"PeriodicalIF":2.3,"publicationDate":"2023-11-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10658947/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138462185","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adult Severe Asthma Registries: A Global and Growing Inventory.","authors":"Breda Cushen,&nbsp;Mariko Siyue Koh,&nbsp;Trung N Tran,&nbsp;Neil Martin,&nbsp;Ruth Murray,&nbsp;Thendral Uthaman,&nbsp;Celine Yun Yi Goh,&nbsp;Rebecca Vella,&nbsp;Neva Eleangovan,&nbsp;Lakmini Bulathsinhala,&nbsp;Jorge F Maspero,&nbsp;Matthew J Peters,&nbsp;Florence Schleich,&nbsp;Paulo Pitrez,&nbsp;George Christoff,&nbsp;Mohsen Sadatsafavi,&nbsp;Carlos A Torres-Duque,&nbsp;Celeste Porsbjerg,&nbsp;Alan Altraja,&nbsp;Lauri Lehtimäki,&nbsp;Arnaud Bourdin,&nbsp;Christian Taube,&nbsp;Nikolaos G Papadopoulos,&nbsp;Csoma Zsuzsanna,&nbsp;Unnur Björnsdóttir,&nbsp;Sundeep Salvi,&nbsp;Enrico Heffler,&nbsp;Takashi Iwanaga,&nbsp;Mona Al-Ahmad,&nbsp;Désirée Larenas-Linnemann,&nbsp;Job F M van Boven,&nbsp;Bernt Bøgvald Aarli,&nbsp;Piotr Kuna,&nbsp;Cláudia Chaves Loureiro,&nbsp;Riyad Al-Lehebi,&nbsp;Jae Ha Lee,&nbsp;Nuria Marina,&nbsp;Leif Bjermer,&nbsp;Chau-Chyun Sheu,&nbsp;Bassam Mahboub,&nbsp;John Busby,&nbsp;Andrew Menzies-Gow,&nbsp;Eileen Wang,&nbsp;David B Price","doi":"10.2147/POR.S399879","DOIUrl":"10.2147/POR.S399879","url":null,"abstract":"<p><strong>Aim: </strong>The International Severe Asthma Registry (ISAR; http://isaregistries.org/) uses standardised variables to enable multi-country and adequately powered research in severe asthma. This study aims to look at the data countries within ISAR and non-ISAR countries reported collecting that enable global research that support individual country interests.</p><p><strong>Methods: </strong>Registries were identified by online searches and approaching severe asthma experts. Participating registries provided data collection specifications or confirmed variables collected. Core variables (results from ISAR's Delphi study), steroid-related comorbidity variables, biologic safety variables (serious infection, anaphylaxis, and cancer), COVID-19 variables and additional variables (not belonging to the aforementioned categories) that registries reported collecting were summarised.</p><p><strong>Results: </strong>Of the 37 registries identified, 26 were ISAR affiliates and 11 non-ISAR affiliates. Twenty-five ISAR-registries and 4 non-ISAR registries reported collecting >90% of the 65 core variables. Twenty-three registries reported collecting all optional steroid-related comorbidity variables. Twenty-nine registries reported collecting all optional safety variables. Ten registries reported collecting COVID-19 variables. Twenty-four registries reported collecting additional variables including data from asthma questionnaires (10 Asthma Control Questionnaire, 20 Asthma Control Test, 11 Asthma Quality of Life Questionnaire, and 4 EuroQol 5-dimension 5-level Questionnaire). Eight registries are linked to databases such as electronic medical records and national claims or disease databases.</p><p><strong>Conclusion: </strong>Standardised data collection has enabled individual severe asthma registries to collect unified data and increase statistical power for severe asthma research irrespective of ISAR affiliations.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"14 ","pages":"127-147"},"PeriodicalIF":8.9,"publicationDate":"2023-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/48/7f/por-14-127.PMC10595155.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"50162638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of an Asthma Exacerbation Risk Prediction Model for Conversational Use by Adults in England.","authors":"Constantinos Kallis,&nbsp;Rafael A Calvo,&nbsp;Bjorn Schuller,&nbsp;Jennifer K Quint","doi":"10.2147/POR.S424098","DOIUrl":"10.2147/POR.S424098","url":null,"abstract":"<p><strong>Background: </strong>Improving accurate risk assessment of asthma exacerbations, and reduction via relevant behaviour change among people with asthma could save lives and reduce health care costs. We developed a simple personalised risk prediction model for asthma exacerbations using factors collected in routine healthcare data for use in a risk modelling feature for automated conversational systems.</p><p><strong>Methods: </strong>We used pseudonymised primary care electronic healthcare records from the Clinical Practice Research Datalink (CPRD) Aurum database in England. We combined variables for prediction of asthma exacerbations using logistic regression including age, gender, ethnicity, Index of Multiple Deprivation, geographical region and clinical variables related to asthma events.</p><p><strong>Results: </strong>We included 1,203,741 patients divided into three cohorts to implement temporal validation: 898,763 (74.7%) in the training sample, 226,754 (18.8%) in the testing sample and 78,224 (6.5%) in the validation sample. The Area under the ROC curve (AUC) for the full model was 0.72 and for the restricted model was 0.71. Using a cut-off point of 0.1, approximately 27 asthma reviews by clinicians per 100 patients would be prevented compared with a strategy that all patients are regarded as high risk. Compared with patients without an exacerbation, patients who exacerbated were older, more likely to be female, prescribed more SABA and ICS in the preceding 12 months, have history of GORD, COPD, anxiety, depression, live in very deprived areas and have more severe disease.</p><p><strong>Conclusion: </strong>Using information available from routinely collected electronic healthcare record data, we developed a model that has moderate ability to separate patients who had an asthma exacerbation within 3 months from their index date from patients who did not. When comparing this model with a simplified model with variables that can easily be self-reported through a WhatsApp chatbot, we have shown that the predictive performance of the model is not substantially different.</p>","PeriodicalId":20399,"journal":{"name":"Pragmatic and Observational Research","volume":"14 ","pages":"111-125"},"PeriodicalIF":8.9,"publicationDate":"2023-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10560745/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41210162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}