Pub Date : 2025-08-01Epub Date: 2025-07-22DOI: 10.1056/EVIDe2500127
Jacque L Duncan
{"title":"Ciliary Neurotrophic Factor - A Promising New Therapy for Macular Telangiectasia Type 2.","authors":"Jacque L Duncan","doi":"10.1056/EVIDe2500127","DOIUrl":"https://doi.org/10.1056/EVIDe2500127","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDe2500127"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-01Epub Date: 2025-06-23DOI: 10.1056/EVIDe2500178
Clifford J Rosen
{"title":"Semaglutide for Type I Diabetes - A New Twist on an Old Story.","authors":"Clifford J Rosen","doi":"10.1056/EVIDe2500178","DOIUrl":"10.1056/EVIDe2500178","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":" ","pages":"EVIDe2500178"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478147","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-01Epub Date: 2025-07-22DOI: 10.1056/EVIDstat2500187
Christos P Kotanidis, Sarah Gorey, Daniel Müller, Christopher Twichell, Adam Straus, Sharon-Lise Normand, Chana A Sacks, C Corey Hardin
{"title":"How Win Ratios Work.","authors":"Christos P Kotanidis, Sarah Gorey, Daniel Müller, Christopher Twichell, Adam Straus, Sharon-Lise Normand, Chana A Sacks, C Corey Hardin","doi":"10.1056/EVIDstat2500187","DOIUrl":"https://doi.org/10.1056/EVIDstat2500187","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDstat2500187"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-01Epub Date: 2025-07-22DOI: 10.1056/EVIDccon2400446
Joseph Benjamen, Michael Fralick
AbstractRandomized controlled trials have demonstrated a reduction in the composite of heart failure events and death from cardiovascular causes with use of SGLT2 inhibitors, regardless of whether a patient has type 2 diabetes mellitus. This evidence led to a class 1A indication for SGLT2i for adults with heart failure. However, recent data demonstrate that the adoption of SGLT2i into practice has been slow. This article describes benefits, risks, pivotal trials, prescribing pearls, and areas of uncertainty related to the use of SGLT2i for adults with heart failure.
{"title":"Which Patients with Heart Failure Benefit from an SGLT2 Inhibitor?","authors":"Joseph Benjamen, Michael Fralick","doi":"10.1056/EVIDccon2400446","DOIUrl":"https://doi.org/10.1056/EVIDccon2400446","url":null,"abstract":"<p><p>AbstractRandomized controlled trials have demonstrated a reduction in the composite of heart failure events and death from cardiovascular causes with use of SGLT2 inhibitors, regardless of whether a patient has type 2 diabetes mellitus. This evidence led to a class 1A indication for SGLT2i for adults with heart failure. However, recent data demonstrate that the adoption of SGLT2i into practice has been slow. This article describes benefits, risks, pivotal trials, prescribing pearls, and areas of uncertainty related to the use of SGLT2i for adults with heart failure.</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDccon2400446"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692670","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-01Epub Date: 2025-07-22DOI: 10.1056/EVIDoa2400424
Alejandro A Diaz, Pallavi P Balte, MeiLan Han, Ravi Kalhan, Surya P Bhatt, David J Couper, David R Jacobs, Martha L Daviglus, Laura R Loehr, Stephanie J London, George T O'Connor, Joseph E Schwartz, Sina A Gharib, Paulo H M Chaves, Tiffany R Sanchez, Sachin Yende, Rebeca Bao, Benjamin M Smith, Wendy B White, Fernando J Martinez, Elizabeth C Oelsner
Background: Identification of chronic obstructive pulmonary disease (COPD) diagnosed before 50 years of age ("young COPD") will help enable the study of preventive and therapeutic interventions for classically diagnosed COPD in later life. However, there remains uncertainty about the definition of young COPD and its prognostic significance.
Methods: We assessed the prevalence of young COPD, defined here as spirometric airflow obstruction plus symptoms of cough, phlegm, and dyspnea or 10 or more pack-years of smoking, among 18-to-49-year-old participants from four pooled, prospective U.S. cohorts. We evaluated the association of young COPD with premature mortality and respiratory and cardiovascular events over follow-up, using multivariable-adjusted proportional hazards models.
Results: Among 10,680 participants (median age, 40 years; 56.8% women; 41.7% Black; 51.1% unexposed to smoking), the prevalence of people meeting our case definition of young COPD was 4.5%. Compared with nonobstructed participants, the adjusted hazard ratio (an adjusted hazard ratio greater than unity indicates more incident cases) for participants with young COPD for death before 75 years of age was 1.43 (95% confidence interval [CI], 1.19 to 1.73; P<0.001); for incident hospitalization or death due to chronic lower respiratory disease, the adjusted hazard ratio was 2.56 (95% CI, 2.05 to 3.20); for coronary heart disease, the adjusted hazard ratio was 1.12 (95% CI, 0.85 to 1.47); and for heart failure, the adjusted hazard ratio was 1.72 (95%CI, 1.26 to 2.35). The hazards of the clinical outcomes in participants with simple obstruction (spirometric obstruction without symptoms and <10 pack-years; prevalence, 2.4%) were similar to those of nonobstructed participants.
Conclusions: Young COPD was present in 4.5% of adults under 50 years of age in the cohorts examined. The diagnosis was associated with premature mortality as well as respiratory and heart-failure events. (Funded by the National Heart, Lung, and Blood Institute and others.).
{"title":"Prevalence and Prognostic Significance of COPD in Adults Younger than 50 Years of Age.","authors":"Alejandro A Diaz, Pallavi P Balte, MeiLan Han, Ravi Kalhan, Surya P Bhatt, David J Couper, David R Jacobs, Martha L Daviglus, Laura R Loehr, Stephanie J London, George T O'Connor, Joseph E Schwartz, Sina A Gharib, Paulo H M Chaves, Tiffany R Sanchez, Sachin Yende, Rebeca Bao, Benjamin M Smith, Wendy B White, Fernando J Martinez, Elizabeth C Oelsner","doi":"10.1056/EVIDoa2400424","DOIUrl":"10.1056/EVIDoa2400424","url":null,"abstract":"<p><strong>Background: </strong>Identification of chronic obstructive pulmonary disease (COPD) diagnosed before 50 years of age (\"young COPD\") will help enable the study of preventive and therapeutic interventions for classically diagnosed COPD in later life. However, there remains uncertainty about the definition of young COPD and its prognostic significance.</p><p><strong>Methods: </strong>We assessed the prevalence of young COPD, defined here as spirometric airflow obstruction plus symptoms of cough, phlegm, and dyspnea or 10 or more pack-years of smoking, among 18-to-49-year-old participants from four pooled, prospective U.S. cohorts. We evaluated the association of young COPD with premature mortality and respiratory and cardiovascular events over follow-up, using multivariable-adjusted proportional hazards models.</p><p><strong>Results: </strong>Among 10,680 participants (median age, 40 years; 56.8% women; 41.7% Black; 51.1% unexposed to smoking), the prevalence of people meeting our case definition of young COPD was 4.5%. Compared with nonobstructed participants, the adjusted hazard ratio (an adjusted hazard ratio greater than unity indicates more incident cases) for participants with young COPD for death before 75 years of age was 1.43 (95% confidence interval [CI], 1.19 to 1.73; P<0.001); for incident hospitalization or death due to chronic lower respiratory disease, the adjusted hazard ratio was 2.56 (95% CI, 2.05 to 3.20); for coronary heart disease, the adjusted hazard ratio was 1.12 (95% CI, 0.85 to 1.47); and for heart failure, the adjusted hazard ratio was 1.72 (95%CI, 1.26 to 2.35). The hazards of the clinical outcomes in participants with simple obstruction (spirometric obstruction without symptoms and <10 pack-years; prevalence, 2.4%) were similar to those of nonobstructed participants.</p><p><strong>Conclusions: </strong>Young COPD was present in 4.5% of adults under 50 years of age in the cohorts examined. The diagnosis was associated with premature mortality as well as respiratory and heart-failure events. (Funded by the National Heart, Lung, and Blood Institute and others.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDoa2400424"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-01Epub Date: 2025-07-22DOI: 10.1056/EVIDctw2400393
Amy LaLonde, Steven E Nissen
AbstractStatistical testing of more than one hypothesis has the potential to increase the risk of wrongly concluding that the result for a given end point is statistically significant (false discovery). This review is designed to acquaint nonstatisticians with traditional approaches for controlling type I error and with the seemingly complex procedure known as graphical testing.
{"title":"Multiplicity Control in Clinical Trials.","authors":"Amy LaLonde, Steven E Nissen","doi":"10.1056/EVIDctw2400393","DOIUrl":"https://doi.org/10.1056/EVIDctw2400393","url":null,"abstract":"<p><p>AbstractStatistical testing of more than one hypothesis has the potential to increase the risk of wrongly concluding that the result for a given end point is statistically significant (false discovery). This review is designed to acquaint nonstatisticians with traditional approaches for controlling type I error and with the seemingly complex procedure known as graphical testing.</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDctw2400393"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-07-01Epub Date: 2025-06-24DOI: 10.1056/EVIDe2500070
Alyssa R Letourneau, Lindsey R Baden
{"title":"Managing Individual and Community Risk - Broad-Spectrum Antimicrobial Use.","authors":"Alyssa R Letourneau, Lindsey R Baden","doi":"10.1056/EVIDe2500070","DOIUrl":"https://doi.org/10.1056/EVIDe2500070","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 7","pages":"EVIDe2500070"},"PeriodicalIF":0.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478078","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-07-01Epub Date: 2025-06-24DOI: 10.1056/EVIDoa2400443
Didier Debieuvre, Lionel Falchero, Olivier Molinier, Sébastien Couraud, Alexis Cortot, Nicolas Meyer, Bernard Asselain, Etienne Auvray, Dorine Templement-Grangerat, Acya Bizieux, Jean Tredaniel, Sophie Schneider, Jean-Bernard Auliac, Olivier Bylicki, Alexia Letierce, Hugues Morel
Background: Lung cancer is the leading cause of cancer-related death worldwide. The aim of the KBP-2020 study was to describe survival among patients diagnosed with lung adenocarcinoma in France in 2000, 2010, and 2020, outside academic medical centers.
Methods: We collected prospective data from all patients diagnosed with lung cancer in nonacademic public hospitals in France in 2020. We compared these data with those from similar studies performed in 2000 and 2010 to map the evolution of survival.
Results: The KBP-2020 cohort comprised 5015 patients with lung adenocarcinoma. The 3-year overall survival (OS) rate was 38.6%, ranging from 21.3% among patients with metastatic disease at diagnosis to 84.0% for those with stage I disease at diagnosis. The median OS in the overall population more than doubled in 20 years, from 8.5 months in 2000 to 20.7 months in 2020. Female sex, higher performance status, and earlier disease stage were associated with an increased 3-year OS. Patients with metastatic lung adenocarcinoma with EGFR, ALK, or ROS1 molecular alterations who were treated with targeted therapy had a higher 3-year OS rate than such patients without these alterations - 36.0% versus 18.5%. Among those patients with metastatic disease without the above-noted molecular alterations, the 3-year OS rate was 36.2% with first-line immunotherapy versus 14.3% without immunotherapy, and median OS was 21.0 months versus 4.2 months.
Conclusions: Improvements in the OS of patients with lung adenocarcinoma were seen over 20 years in this setting of nonacademic public hospitals in France. Targeted therapy and immunotherapy were associated with longer OS among patients with metastatic disease. (Le Nouveau Souffle and others; trial registration number, NCT04402099.).
背景:肺癌是世界范围内癌症相关死亡的主要原因。KBP-2020研究的目的是描述2000年、2010年和2020年法国非学术医疗中心诊断为肺腺癌患者的生存率。方法:我们收集了2020年在法国非学术性公立医院诊断为肺癌的所有患者的前瞻性数据。我们将这些数据与2000年和2010年进行的类似研究的数据进行了比较,以绘制生存进化图。结果:KBP-2020队列包括5015例肺腺癌患者。3年总生存率(OS)为38.6%,从诊断时转移性疾病患者的21.3%到诊断时I期疾病患者的84.0%。总体人口的OS中位数在20年内翻了一倍多,从2000年的8.5个月增加到2020年的20.7个月。女性、较高的工作状态和较早的疾病阶段与3年总生存期增加有关。伴有EGFR、ALK或ROS1分子改变的转移性肺腺癌患者接受靶向治疗的3年OS率高于未发生这些改变的患者,分别为36.0%和18.5%。在没有上述分子改变的转移性疾病患者中,接受一线免疫治疗的3年OS率为36.2%,未接受免疫治疗的为14.3%,中位OS为21.0个月,而未接受免疫治疗的为4.2个月。结论:20年来,在法国非学术性公立医院中,肺腺癌患者的OS有所改善。靶向治疗和免疫治疗与转移性疾病患者更长的生存期相关。(Le Nouveau Souffle等;试验注册号:NCT04402099)。
{"title":"Survival of Patients with Lung Adenocarcinoma Diagnosed in 2000, 2010, and 2020.","authors":"Didier Debieuvre, Lionel Falchero, Olivier Molinier, Sébastien Couraud, Alexis Cortot, Nicolas Meyer, Bernard Asselain, Etienne Auvray, Dorine Templement-Grangerat, Acya Bizieux, Jean Tredaniel, Sophie Schneider, Jean-Bernard Auliac, Olivier Bylicki, Alexia Letierce, Hugues Morel","doi":"10.1056/EVIDoa2400443","DOIUrl":"10.1056/EVIDoa2400443","url":null,"abstract":"<p><strong>Background: </strong>Lung cancer is the leading cause of cancer-related death worldwide. The aim of the KBP-2020 study was to describe survival among patients diagnosed with lung adenocarcinoma in France in 2000, 2010, and 2020, outside academic medical centers.</p><p><strong>Methods: </strong>We collected prospective data from all patients diagnosed with lung cancer in nonacademic public hospitals in France in 2020. We compared these data with those from similar studies performed in 2000 and 2010 to map the evolution of survival.</p><p><strong>Results: </strong>The KBP-2020 cohort comprised 5015 patients with lung adenocarcinoma. The 3-year overall survival (OS) rate was 38.6%, ranging from 21.3% among patients with metastatic disease at diagnosis to 84.0% for those with stage I disease at diagnosis. The median OS in the overall population more than doubled in 20 years, from 8.5 months in 2000 to 20.7 months in 2020. Female sex, higher performance status, and earlier disease stage were associated with an increased 3-year OS. Patients with metastatic lung adenocarcinoma with <i>EGFR</i>, <i>ALK</i>, or <i>ROS1</i> molecular alterations who were treated with targeted therapy had a higher 3-year OS rate than such patients without these alterations - 36.0% versus 18.5%. Among those patients with metastatic disease without the above-noted molecular alterations, the 3-year OS rate was 36.2% with first-line immunotherapy versus 14.3% without immunotherapy, and median OS was 21.0 months versus 4.2 months.</p><p><strong>Conclusions: </strong>Improvements in the OS of patients with lung adenocarcinoma were seen over 20 years in this setting of nonacademic public hospitals in France. Targeted therapy and immunotherapy were associated with longer OS among patients with metastatic disease. (Le Nouveau Souffle and others; trial registration number, NCT04402099.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 7","pages":"EVIDoa2400443"},"PeriodicalIF":0.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478082","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-07-01Epub Date: 2025-06-24DOI: 10.1056/EVIDe2500128
Felix Bongomin, Davidson H Hamer
{"title":"Blocking Human-to-Mosquito Transmission of <i>Plasmodium falciparum</i> - A New Path to Malaria Elimination?","authors":"Felix Bongomin, Davidson H Hamer","doi":"10.1056/EVIDe2500128","DOIUrl":"https://doi.org/10.1056/EVIDe2500128","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 7","pages":"EVIDe2500128"},"PeriodicalIF":0.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478151","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-07-01Epub Date: 2025-06-24DOI: 10.1056/EVIDpp2500164
Nancy Figueroa
{"title":"What Do You Mean, I Have Heart Failure?","authors":"Nancy Figueroa","doi":"10.1056/EVIDpp2500164","DOIUrl":"https://doi.org/10.1056/EVIDpp2500164","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 7","pages":"EVIDpp2500164"},"PeriodicalIF":0.0,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478083","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}