{"title":"Upper gastrointestinal Crohn's disease: What are we talking about?","authors":"Luisa Bertin, Brigida Barberio, Edoardo Vincenzo Savarino","doi":"10.1002/ueg2.12664","DOIUrl":"https://doi.org/10.1002/ueg2.12664","url":null,"abstract":"","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142606623","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Mucosal Healing in Inflammatory Bowel Diseases: Still too Many Irons on the Fire.","authors":"Gabrio Bassotti, Vincenzo Villanacci","doi":"10.1002/ueg2.12707","DOIUrl":"https://doi.org/10.1002/ueg2.12707","url":null,"abstract":"","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142584402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Early colorectal cancer diagnosis: A novel methylated stool DNA model enhanced the diagnostic efficiency.","authors":"Peng Yun, Kamila Kulaixijiang, Jiang Pan, Luping Yang, Nengzhuang Wang, Zheng Xu, Yaodong Zhang, Haifang Cai, Zi-Ye Zhao, Min Zhu, Hongli Yan","doi":"10.1002/ueg2.12696","DOIUrl":"https://doi.org/10.1002/ueg2.12696","url":null,"abstract":"<p><strong>Background: </strong>Methylated stool DNA (sDNA) is a reliable noninvasive biomarker for early colorectal cancer (CRC) diagnosis. However, there are barely any diagnostic panels that can achieve both a sensitivity and specificity exceeding 90% simultaneously.</p><p><strong>Objective: </strong>We aimed to identify a novel methylated sDNA panel and model for the early diagnosis of CRC.</p><p><strong>Methods: </strong>We conducted methyl-CpG binding domain isolated genome sequencing (MiGS) on CpG island methylation phenotype (CIMP)-positive (n = 3) and CIMP-negative CRC tissues (n = 3) and their corresponding normal adjacent tissues. Subsequently, by utilizing both the aforementioned data and public datasets, we identified a set of promising methylated sDNA markers for CRC. Next, we validated 5 of these genes using pyrosequencing in CRC patients (n = 31). Then, we developed a combined diagnostic model (CDM) for CRC based on the methylation status of PRDM12, FOXE1, and SDC2 by a Training cohort (n = 231). Finally, the performance of CDM was evaluated in an independent multicenter Validation cohort (n = 800).</p><p><strong>Results: </strong>A total of 1062 participants were included in this study. The area under the curve (AUC) of the CDM was 0.979 (95% CI: 0.960-0.997), and the optimal sensitivity and specificity were 97.35% and 99.05%, respectively, in the training cohort (n = 231). In the independent validation cohort (n = 800), the AUC was 0.950 (95% CI: 0.927-0.973), along with the optimal sensitivity of 92.75% and specificity of 97.21%. When CRC and advanced adenoma (AAD) were used as diagnostic targets, the model AUC was 0.945 (95% CI: 0.922-0.969), with an optimal sensitivity of 91.89% and a specificity of 95.21%. The model sensitivity for nonadvanced adenoma patients was 68.66%.</p><p><strong>Conclusion: </strong>The sDNA diagnostic model CDM, developed from both CIMP-P and CIMP-N, exhibited exceptional performance in CRC and could serve as a potential alternative strategy for CRC screening.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-11-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Stefan Schreiber, Brian G Feagan, Edouard Louis, Tadakazu Hisamatsu, Toshifumi Hibi, Louis Dron, Corinne Jamoul, Haridarshan Patel, Kristina Harris, Virginia Taliadouros, Alessandra Oortwijn, Laurent Peyrin-Biroulet
{"title":"Distinct trajectories of symptomatic response in ulcerative colitis during filgotinib therapy: A post hoc analysis from the SELECTION study.","authors":"Stefan Schreiber, Brian G Feagan, Edouard Louis, Tadakazu Hisamatsu, Toshifumi Hibi, Louis Dron, Corinne Jamoul, Haridarshan Patel, Kristina Harris, Virginia Taliadouros, Alessandra Oortwijn, Laurent Peyrin-Biroulet","doi":"10.1002/ueg2.12686","DOIUrl":"10.1002/ueg2.12686","url":null,"abstract":"<p><strong>Background: </strong>Filgotinib is an oral, once-daily, Janus kinase 1 preferential inhibitor approved for treatment of ulcerative colitis (UC) following the phase 2b/3 SELECTION trial. Identification of patient populations and factors associated with long-term treatment response trajectories may improve UC management.</p><p><strong>Objective: </strong>We aimed to identify and describe distinct patient subgroups of response to filgotinib based on partial Mayo Clinic Score (pMCS) trajectories over time.</p><p><strong>Methods: </strong>In these post hoc analyses of SELECTION, group-based trajectory modeling (GBTM) was applied to pMCS to describe groups of distinct, symptom-based patient trajectories using data from patients who responded to filgotinib 200 or 100 mg and continued receiving filgotinib up to week 58. Patient demographics, disease characteristics, and week 10 response were compared between the groups. Achievement of a patient-level multi-component endpoint of comprehensive disease control (CDC) was assessed in each group.</p><p><strong>Results: </strong>GBTM identified five distinct patient populations with different response trajectories; 67.5% of patients had beneficial trajectories. The beneficial trajectory groups generally had higher proportions of patients who were recently diagnosed (<1 year), were receiving filgotinib 200 mg and were biologic-naive versus the relapsing trajectory groups (4%-9% vs. 4%-5%; 43%-65% vs. 36%-46%; 54%-70% vs. 35%-58%, respectively). Furthermore, 55.4% of patients had sustained beneficial trajectories, with low baseline endoscopic subscores (≥43% of patients had a subscore of 2) and strong week 10 FCP responses (≥61% of patients with >50% decrease in FCP from baseline). Sustained beneficial trajectory groups had a higher probability of achieving CDC at week 58 than other groups (31%-32% vs. 0%-7%).</p><p><strong>Conclusions: </strong>Beneficial long-term response trajectories and achievement of CDC with filgotinib were associated with being biologic-naive and having less severe disease at baseline. Early estimation of sustained and CDC may facilitate patient identification and development of personalized management strategies in UC.</p><p><strong>Clinicaltrials: </strong></p><p><strong>Gov identifier: </strong>NCT02914522.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1243-1255"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508924","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marjolijn Duijvestein, Reena Sidhu, Katharina Zimmermann, Emma V Carrington, Alexander Hann, Paula Sousa, Hugo R W Touw, Jeanin E van Hooft, Martina Müller
{"title":"The United European Gastroenterology green paper-climate change and gastroenterology.","authors":"Marjolijn Duijvestein, Reena Sidhu, Katharina Zimmermann, Emma V Carrington, Alexander Hann, Paula Sousa, Hugo R W Touw, Jeanin E van Hooft, Martina Müller","doi":"10.1002/ueg2.12698","DOIUrl":"10.1002/ueg2.12698","url":null,"abstract":"<p><p>Climate change, described by the World Health Organization (WHO) in 2021 as 'the single biggest health threat facing humanity', causes extreme weather, disrupts food supplies, and increases the prevalence of diseases, thereby affecting human health, medical practice, and healthcare stability. Greener Gastroenterology is an important movement that has the potential to make a real difference in reducing the impact of the delivery of healthcare, on the environment. The WHO defines an environmentally sustainable health system as one which would improve, maintain or restore health while minimizing negative environmental impacts. Gastroenterologists encounter the impacts of climate change in daily patient care. Alterations in the gut microbiome and dietary habits, air pollution, heat waves, and the distribution of infectious diseases result in changed disease patterns affecting gastrointestinal and hepatic health, with particularly severe impacts on vulnerable groups such as children, adolescents, and the elderly. Additionally, women are disproportionally affected, since climate change can exacerbate gender inequalities. Paradoxically, while healthcare aims to improve health, the sector is responsible for 4.4% of global carbon emissions. Endoscopy is a significant waste producer in healthcare, being the third highest generator with 3.09 kg of waste per day per bed, contributing to the carbon footprint of the GI sector. Solutions to the climate crisis can offer significant health co-benefits. Steps to reduce our carbon footprint include fostering a Planetary Health Diet and implementing measures for greener healthcare, such as telemedicine, digitalization, education, and research on sustainable healthcare practices. Adhering to the principles of 'reduce, reuse, recycle' is crucial. Reducing unnecessary procedures, which constitute a significant portion of endoscopies, can significantly decrease the carbon footprint and enhance sustainability. This position paper by the United European Gastroenterology aims to raise awareness and outline key principles that the GI workforce can adopt to tackle the climate crisis together.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1292-1305"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578853/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508941","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Nikhilesh R Mazumder, Filip Jezek, Sardar Ansari, Elliot B Tapper, Anna S Lok
{"title":"The physiological determinants of symptom burden in cirrhosis with ascites.","authors":"Nikhilesh R Mazumder, Filip Jezek, Sardar Ansari, Elliot B Tapper, Anna S Lok","doi":"10.1002/ueg2.12675","DOIUrl":"10.1002/ueg2.12675","url":null,"abstract":"<p><strong>Background & aims: </strong>Paracentesis is commonly used to manage patient discomfort due to ascites. The relationship between ascites pressure, ascites volume, and patient discomfort has not been elucidated.</p><p><strong>Methods: </strong>We prospectively enrolled adult patients with non-malignant ascites undergoing outpatient therapeutic paracenteses from 2021 to 2024 at a tertiary care hospital. Patients completed a validated symptom questionnaire (ASI-7, maximum score 35) before, immediately after, and 1 week after paracentesis. An open-ended manometer was used to measure ascites pressure at the beginning and end of paracentesis. Mixed effect linear regression was performed to evaluate the relationships between patient characteristics, pressure, volume, and symptoms.</p><p><strong>Results: </strong>One hundred and fifty paracentesis procedures among 48 unique patients with an average Model for End Stage Liver Disease-Sodium 3.0 of 16.7 were included. An average of 6.5 L was drained, which reduced abdominal pressure from a mean of 13.7 to 6.0 cm H<sub>2</sub>O (10.1 to 4.4 mmHg, p < 0.001) and mean symptom score from 22.6 to 6.5 (p < 0.001). Regression models identified that symptoms and abdominal pressure linearly correlated above a pressure of 6 cm H<sub>2</sub>O or ASI-7 score of 16 (p < 0.01). Taller patients required about 670 ml additional drainage per inch above the cohort mean height (5'8″) to achieve the same symptom relief.</p><p><strong>Conclusions: </strong>Pressure measured at the bedside can be used to explore changes in abdominal pressure during paracentesis. Pressure, volume, and patient level factors such as height contribute to patient symptoms but cannot fully explain discomfort associated with ascites and relief after paracentesis.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1222-1229"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578835/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142393723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Addressing the overlooked psychological and social impact of fecal incontinence in inflammatory bowel disease patients.","authors":"Xinli Chen, Wenjun Wang, Jianyu Lv","doi":"10.1002/ueg2.12687","DOIUrl":"10.1002/ueg2.12687","url":null,"abstract":"","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1308-1309"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578833/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475868","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Katharina Mitzlaff, Martha M Kirstein, Christian Müller, Marino Venerito, Alexander Olkus, Michael T Dill, Arndt Weinmann, Lorenz Kocheise, Alina Busch, Kornelius Schulze, Gabriel Allo, Dirk-Thomas Waldschmidt, Maryam Barsch, Bertram Bengsch, Michael Quante, Maria A Gonzalez-Carmona, Vera Himmelsbach, Fabian Finkelmeier, Roman Kloeckner, Peter Schirmacher, Jens U Marquardt, Carolin Zimpel
{"title":"Efficacy, safety and differential outcomes of immune-chemotherapy with gemcitabine, cisplatin and durvalumab in patients with biliary tract cancers: A multicenter real world cohort.","authors":"Katharina Mitzlaff, Martha M Kirstein, Christian Müller, Marino Venerito, Alexander Olkus, Michael T Dill, Arndt Weinmann, Lorenz Kocheise, Alina Busch, Kornelius Schulze, Gabriel Allo, Dirk-Thomas Waldschmidt, Maryam Barsch, Bertram Bengsch, Michael Quante, Maria A Gonzalez-Carmona, Vera Himmelsbach, Fabian Finkelmeier, Roman Kloeckner, Peter Schirmacher, Jens U Marquardt, Carolin Zimpel","doi":"10.1002/ueg2.12656","DOIUrl":"10.1002/ueg2.12656","url":null,"abstract":"<p><strong>Background: </strong>Combined Immuno-chemotherapy consisting of gemcitabine, cisplatin and the programmed death-ligand one inhibitor durvalumab (GCD) is the new standard of care for patients with biliary tract cancers (BTC) based on positive results of the TOPAZ-1 study.</p><p><strong>Objective: </strong>We here evaluated the efficacy and safety of GCD for BTC in a German multicenter real-world patient cohort.</p><p><strong>Methods: </strong>Patients with BTC treated with GCD from 9 German centers were included. Clinicopathological baseline parameters, overall survival (OS), response rate and adverse events (AEs) were retrospectively analyzed. The prognostic impact was determined by Kaplan-Meier analyses and Cox regression models.</p><p><strong>Results: </strong>A total of 165 patients treated with GCD between 2021 and 2024 were included in the study. Median OS and median progression-free survival were 14 months (95% CI 10.3-17.7) and 8 months (95% CI 6.8-9.2), respectively. The best overall response rate was 28.5% and disease control rate was 65.5%. While extrahepatic and intrahepatic BTC showed similar outcomes, mOS was significantly shorter in patients with gall bladder cancer (GB-CA) with 9 months (95% CI 5.5-12.4; p = 0.02). In univariate analyses age ≥70 years, Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≥1, status post cholecystectomy, GB-CA and high baseline CRP values were significantly associated with OS. ECOG PS ≥ 1 and GB-CA remained independent prognostic factors for OS in multivariable cox regression analysis. AEs have been reported in 130 patients (78.8%), including 149 grade 3-4 AEs (25.5%). One patient died of severe infectious pneumonia. Immune-related (ir)AEs occurred in 17 patients (10.3%), including 9 grade 3-4 irAEs (2.2%), which led to treatment interruption in 4 patients.</p><p><strong>Conclusions: </strong>Immuno-chemotherapy in patients with BTC was feasible, effective and safe in a real-life scenario. Our results were comparable to the phase 3 clinical trial results (TOPAZ-1). Reduced efficacy was noted in patients with GB-CA and/or a reduced performance status that warrants further investigation.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1230-1242"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578849/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142296508","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mark Löwenberg, Marit van Barreveld, Adriaan Volkers, Sara van Gennep, Marjolijn Duijvestein, Adriaan A van Bodegraven, Melanie S Hulshoff, Jeroen M Jansen, Dirk van Asseldonk, Rachel West, Geert D'Haens, Nanne de Boer, Marcel G W Dijkgraaf
{"title":"Cost-effectiveness and cost-utility of optimized mercaptopurine treatment versus placebo in ulcerative colitis patients: The randomized controlled OPTIC trial.","authors":"Mark Löwenberg, Marit van Barreveld, Adriaan Volkers, Sara van Gennep, Marjolijn Duijvestein, Adriaan A van Bodegraven, Melanie S Hulshoff, Jeroen M Jansen, Dirk van Asseldonk, Rachel West, Geert D'Haens, Nanne de Boer, Marcel G W Dijkgraaf","doi":"10.1002/ueg2.12661","DOIUrl":"10.1002/ueg2.12661","url":null,"abstract":"<p><strong>Background and aims: </strong>We assessed the cost-effectiveness and cost-utility of therapeutic drug monitoring (TDM)-guided mercaptopurine treatment compared with placebo in ulcerative colitis (UC) patients failing 5-aminosalicylates.</p><p><strong>Methods: </strong>Data were gathered alongside the randomized controlled OPTIC trial (EudraCT: 2015-005260-41). The evaluation was performed from a health care and societal perspective as cost-effectiveness and cost-utility analyses with a time horizon of one year. Volumes and costs of in-hospital care, out-of-hospital care, out-of-pocket expenses and productivity loss were assessed. The main outcomes were the extra costs per additional patient who achieved clinical remission and endoscopic improvement at 52 weeks (responders) and extra costs per quality-adjusted life-year (QALY) gained.</p><p><strong>Results: </strong>In total, 59 patients were randomized to the intervention (n = 29) and control (n = 30) group. Non-significant differences in costs were €63 (-€1267 to €1434; P = 0.93) in favour of placebo from a health care perspective and -€742 (-€3683 to €2016; P = 0.64) in favour of mercaptopurine from a societal perspective. The higher proportion of responders and a non-significant QALY difference of 0.0475 (-0.024-0.117) (P = 0.184) favouring patients on mercaptopurine treatment resulted in €165 extra costs per additional responder and €1326 extra costs per QALY gained from a health care perspective. From a societal perspective, dominance over placebo was observed with cost savings of €1937 per additional responder and €15,621 per QALY gained. The probability of optimised mercaptopurine treatment being cost-effective was 0.80 at a willingness to pay per additional QALY of €20,000.</p><p><strong>Conclusions: </strong>TDM-based mercaptopurine treatment in UC patients failing 5-aminosalicylates is a cost-effective strategy from a societal perspective.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1256-1265"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578840/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Paula Leão Moreira, Axel Dignass, Maria Manuela Estevinho, Francisco Portal, João Mendes, Mafalda Santiago, Walter Reinisch, Bruce E Sands, Geert D'Haens, Gerassimos J Mantzaris, Silvio Danese, Laurent Peyrin-Biroulet, Vipul Jairath, Iris Dotan, Fernando Magro
{"title":"Assessment of outcomes in Crohn's disease: A systematic review of randomized clinical trials to inform a multiple outcome framework.","authors":"Paula Leão Moreira, Axel Dignass, Maria Manuela Estevinho, Francisco Portal, João Mendes, Mafalda Santiago, Walter Reinisch, Bruce E Sands, Geert D'Haens, Gerassimos J Mantzaris, Silvio Danese, Laurent Peyrin-Biroulet, Vipul Jairath, Iris Dotan, Fernando Magro","doi":"10.1002/ueg2.12679","DOIUrl":"10.1002/ueg2.12679","url":null,"abstract":"<p><p>Longstanding disease control in Crohn's disease (CD) is challenging and requires understanding treatment efficacy and outcomes assessment. With multiple novel therapeutic options, rigorous evaluation of outcomes in randomized controlled trials (RCTs) is crucial to inform clinical practice. This study systematically reviewed RCTs focusing on CD outcomes to elucidate the breadth and depth of reported outcomes and measurement instruments. A systematic search was conducted on MEDLINE and Scopus for RCTs published from 1 January 2000 to 31 January 2023. Eligible studies included full-text articles with at least 50 adult CD patients. Primary and secondary outcomes, along with their measurement instruments, were categorized according to the Outcome Measures in Rheumatology Filter 2.1 framework. From 88 included studies, 393 outcomes were analyzed. Clinical outcomes, such as clinical remission and response, were the most prevalent (50.6%); biomarkers (11.5%) and patient-reported outcomes (10.2%) were also assessed. Other outcomes included disease behavior and complications (2%), endoscopy (10.4%), histology (0.5%), radiology (1.3%), healthcare utilization (3.8%), and therapy-related safety (6.9%). Composite outcomes showed an increasing trend, reflecting a shift toward comprehensive evaluations. Coprimary endpoints, including clinical symptoms and mucosal inflammation, were reported in 21 of 88 studies. This review highlights the evolving landscape of outcome assessment in CD RCTs, emphasizing the increasing complexity of outcomes. The prominence of composite outcomes underscores efforts to capture the multidimensional nature of CD. These findings will inform the second stage of a two-round e-Delphi aimed at prioritizing key domains and outcomes for developing a multiple-component outcome for RCTs in CD research.</p>","PeriodicalId":23444,"journal":{"name":"United European Gastroenterology Journal","volume":" ","pages":"1280-1291"},"PeriodicalIF":5.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11578837/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401457","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}