NEJM evidence最新文献

{"title":"Biology-Directed Therapy for Myeloid/Lymphoid Neoplasms with <i>FGFR1</i> Rearrangements.","authors":"Vivian G Oehler, Roland B Walter","doi":"10.1056/EVIDe2500196","DOIUrl":"https://doi.org/10.1056/EVIDe2500196","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 9","pages":"EVIDe2500196"},"PeriodicalIF":0.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144981727","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}

{"title":"Copper IUDs - One Size Does Not Fit All.","authors":"Nancy Z Fang, Jeanelle Sheeder","doi":"10.1056/EVIDe2500114","DOIUrl":"https://doi.org/10.1056/EVIDe2500114","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDe2500114"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692656","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}

{"title":"Seize the Day - Early Detection of COPD.","authors":"Mustafa Abdo, Klaus F Rabe","doi":"10.1056/EVIDe2500125","DOIUrl":"https://doi.org/10.1056/EVIDe2500125","url":null,"abstract":"","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDe2500125"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692660","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}

{"title":"An 80-Year-Old Man with Left Groin Pain.","authors":"Bryce La Course, Zach Rubnitz, Lacey Woods, Heather Balch","doi":"10.1056/EVIDmr2400412","DOIUrl":"https://doi.org/10.1056/EVIDmr2400412","url":null,"abstract":"<p><p>AbstractMorning Report is a time-honored tradition where physicians-in-training present cases to their colleagues and clinical experts to collaboratively examine an interesting patient presentation. The Morning Report section seeks to carry on this tradition by presenting a patient's chief concern and story, inviting the reader to develop a differential diagnosis and discover the diagnosis alongside the authors of the case. This report examines the story of an 80-year-old man with progressively worsening left groin pain following a fall 1 week prior to presentation. Using questions, physical examination, and diagnostic testing, an illness script for the presentation emerges; the differential is refined until a final diagnosis is made.</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDmr2400412"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692652","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}

{"title":"Contraceptive Efficacy and Comparative Side Effects of a Mini Copper Intrauterine Device.","authors":"Courtney A Schreiber, Kavita Nanda, David Hubacher, David K Turok, Jeffrey T Jensen, Mitchell D Creinin, Katharine O White, Ila Dayananda, Stephanie B Teal, Pai-Lien Chen, Beatrice A Chen, Alisa B Goldberg, Jennifer L Kerns, Clint Dart, Anita L Nelson, Michael A Thomas, David F Archer, Jill E Brown, Paula M Castaño, Anne E Burke, Bliss Kaneshiro, Diana L Blithe","doi":"10.1056/EVIDoa2400480","DOIUrl":"10.1056/EVIDoa2400480","url":null,"abstract":"<p><strong>Background: </strong>Intrauterine devices (IUDs) are highly effective, yet acceptability and continuation differ by user and device characteristics. We evaluated the efficacy of the NTCu380 Mini, a 380-mm² copper IUD available in Europe and Canada, and compared safety and continuation with the TCu380A IUD, the 380-mm² copper IUD available in the United States.</p><p><strong>Methods: </strong>We randomly assigned participants between 16 and 40 years of age in a 4:1 ratio to the NTCu380 Mini or TCu380A and assessed over 37 months. The primary outcome was NTCu380 Mini efficacy measured by the Pearl Index (defined as the number of pregnancies per 100 woman-years) in participants 35 years of age and under. Additional outcomes included NTCu380 Mini time to pregnancy by survival analysis, and comparative adverse events (AEs) and continuation rates of NTCu380 Mini versus TCu380A.</p><p><strong>Results: </strong>We randomly assigned 887 participants to NTCu380 Mini (744 [83.9%] nulliparous) and 218 to TCu380A (183 [83.9%] nulliparous), of whom 875 (98.6%) and 213 (97.7%), respectively, had successful placement. The NTCu380 Mini IUD 3-year cumulative Pearl Index was 1.86 (95% confidence interval [CI], 1.20 to 2.74) pregnancies per 100 women-years; the cumulative rate of pregnancy through year 3 was 4.8% (95% CI, 2.8 to 6.9%). Serious AEs occurred in 31 (3.5%) NTCu380 Mini and 4 (1.9%) TCu380A users (P=0.28). Discontinuation occurred in 449 (51.3%) NTCu380 Mini and 122 (57.3%) TCu380A users (P=0.07). AEs led to discontinuation less frequently among NTCu380 Mini (152 [20.8%]) than TCu380A users (57 [33.2%]) (P=0.001), particularly bleeding or pelvic pain AEs (103 [14.5%] vs. 46 [27.3%], respectively; P<0.001).</p><p><strong>Conclusions: </strong>In this predominantly nulliparous population, the NTCu380 Mini IUD had an approximate 5% pregnancy rate over 3 years, with fewer side effects leading to discontinuation than observed among TCu380A users. (Funded by the Bill and Melinda Gates Foundation and others; ClinicalTrials.gov number, NCT03124160.) The findings and conclusions in this article are those of the authors and do not necessarily reflect the views of the Planned Parenthood Federation of America, Inc.</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDoa2400480"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692655","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}

{"title":"Cell-Based Ciliary Neurotrophic Factor Therapy for Macular Telangiectasia Type 2.","authors":"Emily Y Chew, Mark Gillies, Glenn J Jaffe, Alain Gaudric, Cathy Egan, Ian Constable, Traci Clemons, Thomas Aaberg, Debora C Manning, Thomas C Hohman, Alan Bird, Martin Friedlander","doi":"10.1056/EVIDoa2400481","DOIUrl":"10.1056/EVIDoa2400481","url":null,"abstract":"<p><strong>Background: </strong>Revakinagene taroretcel (NT-501) is an encapsulated cell therapy producing ciliary neurotrophic factor that slowed retinal degeneration in patients with macular telangiectasia type 2 (MacTel) in phase 2 trials.</p><p><strong>Methods: </strong>In NTMT-03-A and NTMT-03-B - identically designed phase 3, multicenter, randomized sham-controlled trials - we evaluated efficacy and safety of NT-501 in MacTel. The primary end point was rate of change in ellipsoid zone area (EZA) (photoreceptor) loss over 24 months (mm²/24 months). Secondary outcomes included changes in retinal sensitivity, reading speed, and National Eye Institute Visual Function Questionnaire 25 (NEI VFQ-25) scores (range, 0 to 100; higher scores indicate better function). Safety end points included the proportion of participants experiencing one or more treatment-emergent serious adverse event(s) and loss of 15 or more letters in best-corrected visual acuity (BCVA). Delayed dark adaptation and miosis were among the monitored adverse events.</p><p><strong>Results: </strong>In NTMT-03-A, adjusted rates of change of EZA loss were 0.075 mm²/24 months (95% confidence interval [CI], 0.051 to 0.099) and 0.166 mm²/24 months (95% CI, 0.141 to 0.191) in the NT-501 (n=58) and sham (n=57) groups, respectively, with a difference of -0.091 mm²/24 months (95% CI, -0.125 to -0.056; P<0.001) between groups. In NTMT-03-B, rates of EZA loss were 0.111 mm²/24 months (95% CI, 0.084 to 0.139) and 0.160 mm²/24 months (95% CI, 0.131 to 0.189) in the NT-501 (n=59) and sham (n=54) groups, respectively, with a difference of -0.049 mm²/24 months (95% CI, -0.089 to -0.008; P=0.02). Retinal sensitivity and reading-speed changes between groups were inconsistent in the trials. NEI VFQ-25 scores, BCVA loss, and treatment-emergent serious adverse events did not differ between treatment groups. Miosis was experienced by 17% and 14% of participants receiving NT-501 in NTMT-03-A and NTMT-03-B, respectively, and by none of the participants in sham groups. Delayed dark adaptation was experienced by 17% and 24% of participants receiving NT-501 in NTMT-03-A and NTMT-03-B, respectively, by none in the NTMT-03-A sham group, and by 2% in the NTMT-03-B sham group.</p><p><strong>Conclusions: </strong>NT-501 for MacTel resulted in statistically significantly reduced EZA loss compared with sham procedures. (Funded by Neurotech Pharmaceuticals; ClinicalTrials.gov numbers, NCT03316300 and NCT03319849.).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":"4 8","pages":"EVIDoa2400481"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144692653","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}

{"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}

{"title":"Semaglutide in Adults with Type 1 Diabetes and Obesity.","authors":"Viral N Shah, Halis K Akturk, Davida Kruger, Andrew Ahmann, Anuj Bhargava, Giorgos Bakoyannis, Laura Pyle, Janet K Snell-Bergeon","doi":"10.1056/EVIDoa2500173","DOIUrl":"10.1056/EVIDoa2500173","url":null,"abstract":"<p><strong>Background: </strong>Once-weekly semaglutide is approved for the management of type 2 diabetes and obesity. The efficacy and safety of semaglutide in adults with type 1 diabetes are not established.</p><p><strong>Methods: </strong>In this 26-week, double-blind trial, we randomly assigned 72 adults with type 1 diabetes using an automated insulin delivery (AID) system and with a body mass index of 30 or higher in a 1:1 ratio to receive once-weekly semaglutide up to 1 mg or placebo. The primary composite end point consisted of achieving all of the following elements: continuous glucose monitoring (CGM)-based time between 70 and 180 mg/dl of greater than 70% and time below 70 mg/dl of less than 4%; and weight reduction of at least 5%.</p><p><strong>Results: </strong>A significantly greater percentage of patients in the semaglutide group than in the placebo group achieved the primary composite outcome (36% vs. 0%; between-group difference, 36 percentage points; 95% confidence interval [CI], 20.6 to 52.2; P<0.001). The difference in the least-squares mean change from baseline to week 26 for the semaglutide versus placebo group for glycated hemoglobin was -0.3 percentage points (95% CI, -0.6 to -0.05), for percentage of time with CGM glucose levels between 70 and 180 mg/dl it was 8.8 percentage points (95% CI, 3.9 to 13.7), and for body weight it was -8.8 kg (95% CI, -10.6 to -7.0). There were two severe hypoglycemia events in each group, and no diabetic ketoacidosis was reported.</p><p><strong>Conclusions: </strong>In adults with type 1 diabetes and obesity, semaglutide treatment, compared with AID use alone, significantly improved achievement of a composite of time in range of greater than 70%, with time below range of less than 4%, and a 5% body weight reduction. (Funded by Breakthrough T1D [Type 1 Diabetes]; ADJUST-T1D trial ; Clinicaltrials.gov number, NCT05537233).</p>","PeriodicalId":74256,"journal":{"name":"NEJM evidence","volume":" ","pages":"EVIDoa2500173"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478148","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}

{"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}

{"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}