Clinical and Translational Gastroenterology最新文献

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Disaccharidase Enzyme Deficiency in Adult Patients With Gas and Bloating. 成人气胀患者双糖酶缺乏症。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000809
Brendan Kemple, Satish S C Rao
{"title":"Disaccharidase Enzyme Deficiency in Adult Patients With Gas and Bloating.","authors":"Brendan Kemple, Satish S C Rao","doi":"10.14309/ctg.0000000000000809","DOIUrl":"10.14309/ctg.0000000000000809","url":null,"abstract":"<p><strong>Introduction: </strong>Disaccharidases produced by the small intestinal brush border facilitate digestion of dietary carbohydrates. If deficient, they can cause carbohydrate malabsorption, resulting in several abdominal symptoms. Our aim was to examine the prevalence of disaccharidase deficiency and correlate this with abdominal symptoms in adult patients with chronic abdominal symptoms.</p><p><strong>Methods: </strong>In a retrospective study, patients with gas and bloating and normal endoscopy and computed tomography scan were assessed for lactase, sucrase, maltase, palatinase, and glucoamylase activity. Nine common symptoms such as pain, cramping, constipation, belching, bloating, fullness, indigestion, nausea, diarrhea, vomiting, and gas were assessed for their frequency, intensity, and duration using a validated scale, and a total symptom index was calculated and compared. K-means cluster analysis was performed on lactase-deficient and pandeficient patients with deficiency in 3 or more enzymes.</p><p><strong>Results: </strong>Four hundred ninety-six patients (78.4% female) were enrolled of whom 143 (28.8%) had single enzyme deficiency, 9 (1.8%) had double enzyme deficiency, and 48 (9.7%) were pandeficient. The mean symptom prevalence and its severity were not significantly different between those with or without disaccharidase deficiency. Patients with pandeficiency did not have worse symptoms than those with single or double enzyme deficiency. No single symptom was more prevalent in patients with confirmed enzyme deficiency than those without. Three groups were identified in cluster analysis of pandeficient patients with one group demonstrating significantly lower average symptoms of cramping, indigestion, and nausea.</p><p><strong>Discussion: </strong>Disaccharidase deficiency is common in adults presenting with gas, bloating, distention, and pain. Because these deficiencies are treatable with enzyme supplements or diet, an evaluation for disaccharidase deficiency should be routinely considered.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00809"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932595/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945647","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Vidofludimus Calcium in Patients With Moderate-to-Severe Ulcerative Colitis: A Randomized, Placebo-Controlled, Phase 2 Trial. Vidofludimus钙治疗中重度溃疡性结肠炎:一项随机、安慰剂对照的2期试验
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000813
Geert D'Haens, Kalina Grivcheva Stardelova, Edite Sadiku, Natallia Kizlova, Syitlana Skybalo, Yulia Shehovtsova, Mirela Abramescu, Daniel Vitt, Hella Kohlhof, Andreas Muehler
{"title":"Vidofludimus Calcium in Patients With Moderate-to-Severe Ulcerative Colitis: A Randomized, Placebo-Controlled, Phase 2 Trial.","authors":"Geert D'Haens, Kalina Grivcheva Stardelova, Edite Sadiku, Natallia Kizlova, Syitlana Skybalo, Yulia Shehovtsova, Mirela Abramescu, Daniel Vitt, Hella Kohlhof, Andreas Muehler","doi":"10.14309/ctg.0000000000000813","DOIUrl":"10.14309/ctg.0000000000000813","url":null,"abstract":"<p><strong>Introduction: </strong>Vidofludimus calcium (VidoCa) is a dihydroorotate dehydrogenase inhibitor that demonstrated efficacy in immune-related diseases. This study assessed the safety and efficacy of VidoCa in patients with active ulcerative colitis (UC).</p><p><strong>Methods: </strong>This placebo-controlled, phase 2 trial randomized adults with moderate-to-severe UC to receive once-daily VidoCa (10, 30, or 45 mg) or placebo for 10 weeks (induction); patients with symptomatic remission were rerandomized to VidoCa 10, 30 mg, or placebo once daily for an additional 40 weeks (maintenance). The primary endpoint was clinical remission at week 10. Secondary endpoints included symptomatic remission, endoscopic healing, and symptomatic response. The study is registered with ClinicalTrials.gov (NCT03341962) and EudraCT (2017-003703-22).</p><p><strong>Results: </strong>Two hundred sixty-three patients were randomized to induction treatment with VidoCa (10 mg [n = 67], 30 mg [n = 66], and 45 mg [n = 66]) or placebo (n = 64). Sixteen (14%) patients treated with VidoCa (30 mg or 45 mg) achieved the primary endpoint compared with 8 (14%) with placebo. In patients without concomitant corticosteroids, 7 (12%) treated with VidoCa achieved clinical remission at week 10 vs 1 (4%) with placebo. At week 50, dose-dependent increases in the rate of clinical remission ( P = 0.0358), steroid-free clinical remission, and endoscopic healing were observed. Common adverse events (AEs) were headache (4 [6%]), anemia (3 [6%]), vomiting (3 [5%]), and hypertension (3 [5%]) with incidence similar between placebo and VidoCa. Hematuria (4 [6%]) was a treatment-related AE with VidoCa 45 mg only. The incidence of serious AEs was low.</p><p><strong>Discussion: </strong>VidoCa was safe, well-tolerated, and demonstrated proof-of-concept for dihydroorotate dehydrogenase inhibition to treat UC.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00813"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932581/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment of Bile Acid Diarrhea With Glucagon-Like Peptide 1 Receptor Agonists: A Promising Yet Understudied Approach. GLP-1受体激动剂治疗胆汁酸性腹泻:一种有希望但研究不足的方法。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000815
Anne-Marie Ellegaard, Martin L Kårhus, Matilde Winther-Jensen, Asger B Lund, Filip K Knop
{"title":"Treatment of Bile Acid Diarrhea With Glucagon-Like Peptide 1 Receptor Agonists: A Promising Yet Understudied Approach.","authors":"Anne-Marie Ellegaard, Martin L Kårhus, Matilde Winther-Jensen, Asger B Lund, Filip K Knop","doi":"10.14309/ctg.0000000000000815","DOIUrl":"10.14309/ctg.0000000000000815","url":null,"abstract":"<p><p>Bile acid diarrhea (BAD) is a chronic and socially debilitating disease characterized by abdominal pain, diarrhea, urgency, and fecal incontinence. Recently, in a 6-week randomized controlled trial, we showed that the glucagon-like peptide 1 receptor agonist (GLP-1RA) liraglutide is superior to bile acid sequestration (considered standard-of-care) using colesevelam in reducing BAD symptoms. The emergence of new, more potent, and longer-acting GLP-1RAs has spurred an interest in these treatments in BAD management. Here, we review the literature on different GLP-1RAs in BAD treatment and outline their potential mode of actions, highlight knowledge gaps, and outline the need for further clinical evidence generation.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00815"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142977890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bilingual Perspectives of Functional Dyspepsia Management in People From Underserved Areas. 欠发达地区功能性消化不良管理的双语视角。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000817
Christopher Vélez, Isabelle Garcia-Fischer, Mary Paz, Alisa Regassa, Ingrid Guerrero-López, April Mendez, Hannah Konkel, Nir Bar, Esteban A Barreto, Joseph Betancourt, Helen Burton-Murray, Kyle Staller, Braden Kuo
{"title":"Bilingual Perspectives of Functional Dyspepsia Management in People From Underserved Areas.","authors":"Christopher Vélez, Isabelle Garcia-Fischer, Mary Paz, Alisa Regassa, Ingrid Guerrero-López, April Mendez, Hannah Konkel, Nir Bar, Esteban A Barreto, Joseph Betancourt, Helen Burton-Murray, Kyle Staller, Braden Kuo","doi":"10.14309/ctg.0000000000000817","DOIUrl":"10.14309/ctg.0000000000000817","url":null,"abstract":"<p><strong>Introduction: </strong>Disorders of gut-brain interaction, such as functional dyspepsia (FD), are prevalent and challenging conditions. In other gastrointestinal (GI) disorders, individuals from underserved areas (UAs) have difficulty accessing care. Little is known about UA FD patient perspectives of their care, especially in those with limited English proficiency. We aimed to characterize patients' experiences with FD management with the goal of informing future studies targeting disorders of gut-brain interaction management in potentially vulnerable communities residing in UAs.</p><p><strong>Methods: </strong>Participants meeting FD criteria were identified in 2 community health centers affiliated with a large academic medical center in the Northeastern United States. Semistructured interviews were conducted in English and Spanish. Transcripts were reviewed by a bilingual panel of investigators using the constant comparative method of iterative data acquisition. Psychosocial stressors and GI symptom severity were assessed.</p><p><strong>Results: </strong>A total of 26 participants were interviewed (12 English-speaking and 14 Spanish-speaking). Broadly, GI symptoms were mild and there was mild-to-moderate psychological distress present. Adverse social determinants of health were highly prevalent. Despite mild symptom severity on objective scales, FD severely affected quality of life and interfered with physical, psychological, and social well-being, including avoidance of certain foods and professional/social situations. Study participants (particularly those with limited English proficiency status) reported difficulty in receiving care. Thematic saturation was achieved.</p><p><strong>Discussion: </strong>Even when symptoms were mild, interviewees from UAs reported significant FD-related impairment, along with psychological distress. Education interventions targeting FD-related care in UAs should be designed to improve shared decision making in FD, sensitive to the burden of social determinants of health.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00817"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932584/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142969821","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Iodine Staining With Distance Countdown Improving the Safety for Reduction of Adverse Events: A Randomized Controlled Trial. 碘染色与距离倒计时提高安全性,减少不良事件:一项随机对照试验。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000822
Mingjia Xi, Xinyue Luo, Feifan Chen, Zhu Wang, Xue Xiao, Binyang Luo, Mo Chen, Tao Gan, Jinlin Yang, Kai Deng
{"title":"Iodine Staining With Distance Countdown Improving the Safety for Reduction of Adverse Events: A Randomized Controlled Trial.","authors":"Mingjia Xi, Xinyue Luo, Feifan Chen, Zhu Wang, Xue Xiao, Binyang Luo, Mo Chen, Tao Gan, Jinlin Yang, Kai Deng","doi":"10.14309/ctg.0000000000000822","DOIUrl":"10.14309/ctg.0000000000000822","url":null,"abstract":"<p><strong>Introduction: </strong>Lugol chromoendoscopy (LCE) is valuable, cost-effective, and widely used in early esophageal cancer screening, yet it suffers from low compliance because of adverse events after LCE. In addition, the reflux of iodine during iodine staining in the upper esophagus brings the risk of bucking and aspiration. We introduced a new model called distance countdown (DC) aimed to reduce reflux during iodine staining in upper esophageal LCE.</p><p><strong>Methods: </strong>In this randomized controlled trial, 204 patients were randomized into the DC and No-DC groups. The primary end point was the difference in the incidence of positive starch reagent reaction (iodine solution reflux) between the 2 groups. The secondary end points were the comparisons of the incidence of other adverse events after LCE between the 2 groups.</p><p><strong>Results: </strong>The rate of iodine solution reflux was 1.0% in the DC group and 26.5% in the No-DC group ( P < 0.001). Furthermore, the incidences of bucking between the 2 groups were 1.0% and 9.8% ( P = 0.005). LCE satisfaction rates were 78.4% and 76.5% in the DC and No-DC groups ( P = 0.363), respectively. Concerning symptoms after LCE, incidences of sore throat, pharyngeal discomfort or odor, bitter taste, and heartburn were also reduced in the DC group (all P < 0.05).</p><p><strong>Discussion: </strong>Adding DC as an auxiliary effect during LCE would reduce the risk of iodine solution reflux, as well as other adverse events after LCE. Implementing this measure could be beneficial in improving the safety of LCE in early esophageal cancer screening.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00822"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932585/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143001143","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Noninvasive Assessment of Vascular Endothelial Growth Factor and Prognosis in Gastric Cancer Through Radiomic Features. 通过放射学特征无创评估胃癌VEGF与预后。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000802
Hao Feng, Kangneng Zhou, Qingyu Yuan, Zhiwei Liu, Taojun Zhang, Hao Chen, Benjamin Xu, Zepang Sun, Zhen Han, Hao Liu, Shitong Yu, Tao Chen, Guoxin Li, Wenlan Zhou, Jiang Yu, Weicai Huang, Yuming Jiang
{"title":"Noninvasive Assessment of Vascular Endothelial Growth Factor and Prognosis in Gastric Cancer Through Radiomic Features.","authors":"Hao Feng, Kangneng Zhou, Qingyu Yuan, Zhiwei Liu, Taojun Zhang, Hao Chen, Benjamin Xu, Zepang Sun, Zhen Han, Hao Liu, Shitong Yu, Tao Chen, Guoxin Li, Wenlan Zhou, Jiang Yu, Weicai Huang, Yuming Jiang","doi":"10.14309/ctg.0000000000000802","DOIUrl":"10.14309/ctg.0000000000000802","url":null,"abstract":"<p><strong>Introduction: </strong>Gastric cancer (GC) is a leading cause of cancer-related deaths worldwide, with delayed diagnosis often limiting effective treatment options. This study introduces a novel, noninvasive radiomics-based approach using [18F] FDG PET/CT (fluorodeoxyglucose positron emission tomography/computed tomography) to predict vascular endothelial growth factor (VEGF) status and survival in patients with GC. The ability to noninvasively assess these parameters can significantly influence therapeutic decisions and outcomes.</p><p><strong>Methods: </strong>We conducted a retrospective study involving patients diagnosed with GC, stratified into training, validation, and test groups. Each patient underwent a [18F] FDG PET/CT scan, and radiomic features were extracted using dedicated software. A Radiomics Score (RS) was calculated, serving as a predictor for VEGF status. Statistical analyses included logistic regression and Cox proportional hazards models to evaluate the predictive power of RS on survival outcomes.</p><p><strong>Results: </strong>The developed radiomics model demonstrated high predictive accuracy, with the RS formula achieving an area under the receiver operating characteristic curve of 0.861 in the training cohort and 0.857 in the validation cohort for predicting VEGF status. The model also identified RS as an independent prognostic factor for survival, where higher RS values correlated with poorer survival rates.</p><p><strong>Discussion: </strong>The findings underscore the potential of [18F] FDG PET/CT radiomics in transforming the management of GC by providing a noninvasive means to assess tumor aggressiveness and prognosis through VEGF status. This model could facilitate earlier and more tailored therapeutic interventions, potentially improving survival outcomes in a disease marked by typically late diagnosis and limited treatment success.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00802"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932601/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945630","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Serial Procalcitonin Measurements for Determining Bacterial Infection and Mortality in Cirrhotic Patients With Systemic Inflammatory Response Syndrome. 一系列降钙素原测量测定系统性炎症反应综合征肝硬化患者的细菌感染和死亡率。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000810
Phunchai Charatcharoenwitthaya, Pisit Apisophonsiri, Kamonthip Sukonrut, Kraisingh Kuljiratitikal, Ronnakorn Kongsakon, Siwaporn Chainuvati
{"title":"Serial Procalcitonin Measurements for Determining Bacterial Infection and Mortality in Cirrhotic Patients With Systemic Inflammatory Response Syndrome.","authors":"Phunchai Charatcharoenwitthaya, Pisit Apisophonsiri, Kamonthip Sukonrut, Kraisingh Kuljiratitikal, Ronnakorn Kongsakon, Siwaporn Chainuvati","doi":"10.14309/ctg.0000000000000810","DOIUrl":"10.14309/ctg.0000000000000810","url":null,"abstract":"<p><strong>Introduction: </strong>The utility of serial procalcitonin (PCT) measurements in cirrhotic patients with systemic inflammatory response syndrome (SIRS) is not well understood. The aim of this study was to assess the effectiveness of serial PCT measurements for diagnosing bacterial infections and predicting 30-day mortality in this population.</p><p><strong>Methods: </strong>We prospectively studied 120 cirrhotic patients with SIRS, 64.2% of whom had bacterial infections. Serial PCT levels were measured within the first 72 hours of admission.</p><p><strong>Results: </strong>Patients with bacterial infections had significantly higher PCT levels at admission, 24 hours, and 72 hours compared with those without infections. PCT values >0.5 ng/mL within 72 hours demonstrated high sensitivity (81.8-87.5%) but moderate specificity (27.9-44.2%) for diagnosing bacterial infections. Serial PCT monitoring, including the 72-hr/baseline ratio and changes in PCT over 72 hours, provided insights into the evolution of bacterial infections and short-term mortality. Patients with a PCT 72-hour/baseline ratio >0.8 had higher 30-day mortality than those with a ratio <0.5 (50.0% vs 25.6%; odds ratio 3.91, 95% CI 1.40-10.97). Patients whose PCT levels decreased by >50% had lower 30-day mortality than those with increasing levels (23.3% vs 46.7%; odds ratio 0.25, 95% CI 0.08-0.74). Patients with Model for End-Stage Liver Disease scores >15 and bacterial infections who experienced a PCT decrease of <50% had higher 30-day mortality than those with greater reductions (57.7% vs 25.0%, P = 0.021).</p><p><strong>Discussion: </strong>Serial PCT measurements within 72 hours of admission are useful for determining bacterial infections and mortality in cirrhotic patients with SIRS. PCT monitoring may optimize antibiotic use and enhance early risk stratification, potentially improving patient outcomes.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00810"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932589/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Tenascin-X Deficiency Causing Classical-Like Ehlers-Danlos Syndrome Type 1 in Humans is a Significant Risk Factor of Gastrointestinal and Tracheal Ruptures. Tenascin-X缺乏导致人类典型的Ehlers - Danlos综合征1型,是胃肠道和气管破裂的重要危险因素。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000821
Jonneke E van Gurp, Rosan L Lechner, Dimitra Micha, Alessandra Maugeri, Eelco Dulfer, Fleur S van Dijk, Daniel Keszthelyi, Edoardo Malfatti, Akiharu Kubo, Nicol C Voermans, Serwet Demirdas
{"title":"Tenascin-X Deficiency Causing Classical-Like Ehlers-Danlos Syndrome Type 1 in Humans is a Significant Risk Factor of Gastrointestinal and Tracheal Ruptures.","authors":"Jonneke E van Gurp, Rosan L Lechner, Dimitra Micha, Alessandra Maugeri, Eelco Dulfer, Fleur S van Dijk, Daniel Keszthelyi, Edoardo Malfatti, Akiharu Kubo, Nicol C Voermans, Serwet Demirdas","doi":"10.14309/ctg.0000000000000821","DOIUrl":"10.14309/ctg.0000000000000821","url":null,"abstract":"<p><strong>Introduction: </strong>Classical-like Ehlers-Danlos syndrome type 1 (clEDS1) is a very rare form of Ehlers-Danlos syndrome caused by tenascin-X deficiency, with only 56 individuals reported in medical literature. Tenascin-X is an extracellular matrix protein needed for collagen stability. Previous publications propose that individuals with clEDS1 might be at risk of gastrointestinal (GI) tract perforations and/or tracheal ruptures. The aim of this study was to characterize complications resulting from perforations of the GI tract and/or tracheal rupture in an international case series of individuals with clEDS1 due to disease-related tissue fragility.</p><p><strong>Methods: </strong>This case series includes individuals with confirmed clEDS1 and GI perforations and/or tracheal ruptures from participating centers. Researchers who previously reported such individuals were contacted for additional information. A retrospective assessment of clinical features was performed.</p><p><strong>Results: </strong>Fifteen individuals were included. Ten had spontaneous GI perforations, 7 of whom had multiple GI perforations. Almost all had severe diverticulosis. Three individuals experienced iatrogenic tracheal ruptures.</p><p><strong>Discussion: </strong>Severe GI complications, such as perforation, and tracheal rupture were observed in a substantial number of individuals with clEDS1. As these features seem significantly more common in clEDS1 than in the average population, we advise vigilance during intubation and GI endoscopic interventions of individuals with clEDS1. Routine referrals to clinical geneticists are recommended for patients with symptoms indicative of clEDS1, especially with unexplained GI perforations and connective tissue symptoms. Our findings offer valuable insights for the clinical management of clEDS1 and underscore the importance of specialized care, providing a foundation for improved clinical guidelines and preventive strategies.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00821"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932583/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142977886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Higher Rates of Delay in Starting Advanced Inflammatory Bowel Disease Therapies Linked to Insurance Delays, Intravenous Infusions, and Lack of Pharmacy Support. 延迟开始晚期炎症性肠病治疗的较高比率与保险延迟、静脉输液和缺乏药房支持有关。
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000808
Sara Gottesman, Karen Xiao, Hang P Nguyen, Elizabeth Hernandez, Emily Saweris, Priyanka Jagannathan, Faraz Jafri, Jonathan Davis, Kimhouy Tong, Zhouwen Tang, Jill K J Gaidos, Linda A Feagins
{"title":"Higher Rates of Delay in Starting Advanced Inflammatory Bowel Disease Therapies Linked to Insurance Delays, Intravenous Infusions, and Lack of Pharmacy Support.","authors":"Sara Gottesman, Karen Xiao, Hang P Nguyen, Elizabeth Hernandez, Emily Saweris, Priyanka Jagannathan, Faraz Jafri, Jonathan Davis, Kimhouy Tong, Zhouwen Tang, Jill K J Gaidos, Linda A Feagins","doi":"10.14309/ctg.0000000000000808","DOIUrl":"10.14309/ctg.0000000000000808","url":null,"abstract":"<p><strong>Introduction: </strong>Because biologic and small molecule therapy is expensive, payors have mandated preauthorizations for these medications, often resulting in a lengthy approval process. The aims of this study were to assess the frequency of and risk factors for delays in starting advanced therapies assessing insurance, care team, and patient-related factors.</p><p><strong>Methods: </strong>Retrospective, multicenter study of patients with adult inflammatory bowel disease with prescriptions for an advanced therapy in 2 geographically distinct academic gastroenterology practices: one with and the other without a dedicated pharmacist. A priori , we defined a delay in starting therapy as >14 days between prescription and the first dose. Logistic regression analysis was performed to assess for risk factors for delay.</p><p><strong>Results: </strong>A total of 388 patients were prescribed advanced therapies with 46.6% receiving their first dose within 14 days. Patients who were on time vs delayed were similar in baseline demographics, disease characteristics, and disease activity. After adjusting for confounders, 3 independent risk factors remained significant as predictors for delay: study site (OR = 5.2, 95% CI 2.894, 9.333), intravenous drug delivery as opposed to subcutaneous or oral (OR = 3.07, 95% CI 1.845, 5.099), and insurance denial (OR = 2.72, 95% CI 1.082, 6.825).</p><p><strong>Discussion: </strong>In a multicenter study, we found that a delay between prescription and administration of the first dose of an advanced therapy is common, with > 50% of patients having the first dose delayed by > 2 weeks. Delays in starting therapy were significantly more likely if denied by insurance, given by intravenously induction, or at a study site without a dedicated pharmacist.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00808"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142881291","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Tenofovir vs Entecavir on the Prognosis of Patients With Hepatitis B Virus-Related Hepatocellular Carcinoma After Liver Resection: The Role of HBsAg Levels. 替诺福韦与恩替卡韦对乙型肝炎病毒相关性肝癌肝切除术后预后的影响:HBsAg水平的作用
IF 3 3区 医学
Clinical and Translational Gastroenterology Pub Date : 2025-03-01 DOI: 10.14309/ctg.0000000000000814
Zhancheng Qiu, Yueqing Xu, Weili Qi, Junyi Shen, Tianfu Wen, Chuan Li
{"title":"Tenofovir vs Entecavir on the Prognosis of Patients With Hepatitis B Virus-Related Hepatocellular Carcinoma After Liver Resection: The Role of HBsAg Levels.","authors":"Zhancheng Qiu, Yueqing Xu, Weili Qi, Junyi Shen, Tianfu Wen, Chuan Li","doi":"10.14309/ctg.0000000000000814","DOIUrl":"10.14309/ctg.0000000000000814","url":null,"abstract":"<p><strong>Introduction: </strong>Our study aimed to explore whether hepatitis B surface antigen (HBsAg) levels affected the role of nucleot(s)ide analog treatment (entecavir [ETV] and tenofovir disoproxil fumarate [TDF]) in improving the prognosis of patients with hepatitis B virus (HBV)-related hepatocellular carcinoma (HCC) after liver resection.</p><p><strong>Methods: </strong>A total of 865 patients with HBV-related HCC after hepatectomy treated with TDF or ETV were included in our study. Patients were divided into the high HBsAg cohort (n = 681) and the low HBsAg cohort (n = 184). Propensity score matching (PSM) analysis was used to reduce the impact of potential confounding factors. Kaplan-Meier method and competing risk analysis were used to compare the survival outcomes.</p><p><strong>Results: </strong>In the high HBsAg cohort, patients in the TDF group had better recurrence-free survival (RFS) and overall survival (OS) compared with patients in the ETV group both before (RFS: P < 0.001; OS: P < 0.001) and after (RFS: P = 0.005; OS: P = 0.035) PSM. TDF treatment was a favorable factor independently associated with RFS (hazard ratio: 0.58, 95% confidence interval: 0.45-0.75, P < 0.001) and OS (hazard ratio: 0.43, 95% confidence interval: 0.28-0.66, P < 0.001). In the low HBsAg cohort, no difference was observed in RFS and OS between the TDF group and the ETV group both before (RFS: P = 0.140; OS: P = 0.640) and after (RFS: P = 0.480; OS: P = 0.920) PSM. TDF treatment remained superiority after controlling for competing events by competing risk analysis in the high HBsAg cohort.</p><p><strong>Discussion: </strong>TDF treatment was superior to ETV treatment in improving RFS and OS of HBV-related HCC patients with high HBsAg level after liver resection. Even after controlling for survival competing events, the advantage of TDF treatment remained. Our findings may better help clinicians to assign individualized antiviral regimens to patients with HBV-related HCC after liver resection.</p>","PeriodicalId":10278,"journal":{"name":"Clinical and Translational Gastroenterology","volume":" ","pages":"e00814"},"PeriodicalIF":3.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11932590/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142945651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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