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1. Low-Dose Aspirin for PI3K-Altered Localized Colorectal Cancer.
作者: Anna Martling.;Ida Hed Myrberg.;Mef Nilbert.;Henrik Grönberg.;Fredrik Granath.;Martin Eklund.;Tom Öresland.;Lene H Iversen.;Carola Haapamäki.;Martin Janson.;Karin Westberg.;Josefin Segelman.;Urban Ersson.;Mattias Prytz.;Eva Angenete.;Rebecka Bergström.;Markus Mayrhofer.;Bengt Glimelius.;Johan Lindberg.; .
来源: N Engl J Med. 2025年393卷11期1051-1064页
Aspirin reduces the incidence of colorectal adenoma and colorectal cancer among high-risk persons. Observational studies suggest that aspirin may also improve disease-free survival after diagnosis, particularly among patients with tumors harboring somatic PIK3CA mutations. However, data from randomized trials are lacking.
2. Oral Semaglutide at a Dose of 25 mg in Adults with Overweight or Obesity.
作者: Sean Wharton.;Ildiko Lingvay.;Pawel Bogdanski.;Ruben Duque do Vale.;Stephan Jacob.;Tobias Karlsson.;Chaithra Shaji.;Domenica Rubino.;W Timothy Garvey.; .
来源: N Engl J Med. 2025年393卷11期1077-1087页
Oral semaglutide at a dose of 25 mg may provide an alternative treatment option to injectable semaglutide (2.4 mg) and higher-dose oral semaglutide (50 mg) for persons with overweight or obesity.
3. One Dose versus Three Doses of Benzathine Penicillin G in Early Syphilis.
作者: Edward W Hook.;Jodie A Dionne.;Kimberly Workowski.;Candice J McNeil.;Stephanie N Taylor.;Teresa A Batteiger.;Julia C Dombrowski.;Kenneth H Mayer.;Arlene C Seña.;Matthew M Hamill.;Harold C Wiesenfeld.;Chunming Zhu.;Charlotte Perlowski.;Jorge E Mejia-Galvis.;Lori M Newman.
来源: N Engl J Med. 2025年393卷9期869-878页
Controversy persists regarding the appropriate duration of therapy with benzathine penicillin G in persons with early (i.e., primary, secondary, or early latent) syphilis (Treponema pallidum infection).
4. Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy.
作者: Milind Y Desai.;Anjali T Owens.;Theodore Abraham.;Iacopo Olivotto.;Pablo Garcia-Pavia.;Renato D Lopes.;Perry Elliott.;Fabio Fernandes.;Nicolas Verheyen.;Lars Maier.;Benjamin Meder.;Olga Azevedo.;Hiroaki Kitaoka.;Kathy Wolski.;Qiuqing Wang.;Wael Jaber.;Lisa Mitchell.;Jonathan Myers.;Thomas Rano.;Zhiqun Gong.;Yue Zhong.;Suzanne Carter-Bonanza.;Victoria Florea.;Ron Aronson.;Steven E Nissen.; .
来源: N Engl J Med. 2025年393卷10期961-972页
Mavacamten is approved to treat adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM). However, its effects in nonobstructive HCM remain uncertain.
5. Aficamten or Metoprolol Monotherapy for Obstructive Hypertrophic Cardiomyopathy.
作者: Pablo Garcia-Pavia.;Martin S Maron.;Ahmad Masri.;Bela Merkely.;Michael E Nassif.;Maria Luisa Peña-Peña.;Roberto Barriales-Villa.;Ozlem Bilen.;Melissa Burroughs.;Brian Claggett.;Juan Pablo Costabel.;Edileide de Barros Correia.;Anne M Dybro.;Perry Elliott.;Sheila M Hegde.;Neal K Lakdawala.;Gregory D Lewis.;Amy Mann.;Zi Michael Miao.;Ajith Nair.;Steen H Poulsen.;Patricia Reant.;P Christian Schulze.;Scott D Solomon.;Andrew Wang.;Regina Sohn.;Indrias Berhane.;Stephen B Heitner.;Daniel L Jacoby.;Stuart Kupfer.;Fady I Malik.;Amy Wohltman.;Michael A Fifer.; .
来源: N Engl J Med. 2025年393卷10期949-960页
Beta-blockers have been the initial treatment for symptomatic obstructive hypertrophic cardiomyopathy (HCM) despite limited evidence of their efficacy. Aficamten is a cardiac myosin inhibitor that reduces left ventricular outflow tract gradients, improves exercise capacity, and decreases HCM symptoms when added to standard medications. Whether aficamten as monotherapy provides greater clinical benefit than beta-blockers as monotherapy remains unknown.
6. Multidomain Rehabilitation for Older Patients with Myocardial Infarction.
作者: Elisabetta Tonet.;Andrea Raisi.;Silvia Zagnoni.;Giorgio Chiaranda.;Giovanni Pasanisi.;Daniela Aschieri.;Paola Emanuela D'Intino.;Rita Pavasini.;Paolo Cimaglia.;Roberta Campana.;Francesco Vitali.;Tommaso Piva.;Gianni Casella.;Serena Caglioni.;Valentina Zerbini.;Giulia Bugani.;Marta Cocco.;Erica Menegatti.;Martina De Raffele.;Simona Mandini.;Donato Martella.;Nicola Pesenti.;Gianni Mazzoni.;Simone Biscaglia.;Stefano Volpato.;Giovanni Grazzi.;Gianluca Campo.; .
来源: N Engl J Med. 2025年393卷10期973-982页
The benefit of rehabilitation interventions in patients who are 65 years of age or older with myocardial infarction and impaired physical performance remains unclear.
7. Phase 3 Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis.
作者: Bruce C Trapnell.;Yoshikazu Inoue.;Francesco Bonella.;Tisha Wang.;Cormac McCarthy.;Toru Arai.;Keiichi Akasaka.;Francesca Mariani.;Nesrin Mogulkoc.;Jin Woo Song.;Tomohisa Baba.;Stephane Jouneau.;Tadahisa Numakura.;Nesrin Öcal.;Florin Mihaltan.;Ali Ataya.;Elisabeth Bendstrup.;Ilaria Campo.;Brenna Carey.;Ross Arena.;Brian Robinson.;Rosanna Fleming.;Yasmine Wasfi.;Raymond Pratt.; .
来源: N Engl J Med. 2025年393卷8期764-773页
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare disease characterized by progressive surfactant accumulation and hypoxemia caused by autoantibodies against granulocyte-macrophage colony-stimulating factor (GM-CSF), which alveolar macrophages require to clear surfactant. Molgramostim is a formulation of inhaled recombinant human GM-CSF, but its efficacy and safety in patients with aPAP have not been studied sufficiently.
8. Risdiplam in Presymptomatic Spinal Muscular Atrophy.
作者: Richard S Finkel.;Laurent Servais.;Dmitry Vlodavets.;Edmar Zanoteli.;Maria Mazurkiewicz-Bełdzińska.;Yuh-Jyh Jong.;Aledie Navas-Nazario.;Mohammad Al-Muhaizea.;Alexandra P Q C Araujo.;Leslie Nelson.;Yi Wang.;Birgit Jaber.;Ksenija Gorni.;Heidemarie Kletzl.;Laura Palfreeman.;Michael Rabbia.;Dave Summers.;Eleni Gaki.;Kathryn R Wagner.;Paulo Fontoura.;Michelle A Farrar.;Enrico Bertini.; .
来源: N Engl J Med. 2025年393卷7期671-682页
Risdiplam, an oral pre-messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic disease are unclear.
9. Ciprofloxacin versus Aminoglycoside-Ciprofloxacin for Bubonic Plague.
作者: Rindra Vatosoa Randremanana.;Mihaja Raberahona.;Josephine Bourner.;Minoarisoa Rajerison.;Tansy Edwards.;Ravaka Randriamparany.;Tsinjo Fehizoro Razafindratsinana.;Lisy Hanitra Razananaivo.;Gabriella Zadonirina.;Théodora Mayouya-Gamana.;Alex Paddy Abdel Salam.;Reziky Tiandraza Mangahasimbola.;Voahangy Andrianaivoarimanana.;Elise Pesonel.;Rivonirina Andry Rakotoarivelo.;Mamy Jean de Dieu Randria.;Peter Horby.;Piero Olliaro.; .
来源: N Engl J Med. 2025年393卷6期544-555页
Plague is a high-consequence infectious disease with epidemic potential. Current treatment guidelines are based on weak evidence.
10. Ivermectin to Control Malaria - A Cluster-Randomized Trial.
作者: Carlos Chaccour.;Marta Maia.;Mercy Kariuki.;Paula Ruiz-Castillo.;Caroline Wanjiku.;Lydia Kasiwa.;Aurelia Brazeal.;Aina Casellas.;Mwanajuma Ngama.;Truphena Onyango.;Eldo Elobolobo.;Karisa Kazungu.;Mary Mael.;Winnie Wangari.;Khadija Nuru.;Rachel Otuko.;Almudena Sanz.;Isaac Ringera.;Allan Matano.;Starford Mitora.;Marta Ribes.;Joe Brew.;Nika Gorski.;Patricia Nicolas.;Sara Stanulovic.;Isaiah Omondi.;Joanna Furnival-Adams.;Laura Túnez.;Jamal Mbarak.;Vegovito Vegove.;Esther Yaa.;Shadrack Mramba.;Yegon Kibet.;Naomi Nyambura.;Charles Rotich.;Scholastica Wanjiru.;Musa Vura.;Faith Wanjiku.;Leslie Sam.;Lisa Collins.;Kang Xia.;Felix Hammann.;Francisco Saúte.;Matthew Rudd.;Cassidy Rist.;Caroline Jones.;Joseph Mwangangi.;N Regina Rabinovich.
来源: N Engl J Med. 2025年393卷4期362-375页
Malaria control and elimination is threatened by the spread of insecticide resistance and behavioral adaptation of vectors. Whether mass administration of ivermectin, a broad-spectrum antiparasitic drug that also kills mosquitoes feeding on treated persons, can reduce malaria transmission is unclear.
11. Multidose Ondansetron after Emergency Visits in Children with Gastroenteritis.
作者: Stephen B Freedman.;Sarah Williamson-Urquhart.;Amy C Plint.;Andrew Dixon.;Darcy Beer.;Gary Joubert.;Petros Pechlivanoglou.;Yaron Finkelstein.;Anna Heath.;Jasper Zhongyuan Zhang.;Angela Wallace.;Martin Offringa.;Terry P Klassen.; .
来源: N Engl J Med. 2025年393卷3期255-266页
Ondansetron improves outcomes when administered in emergency departments to children with acute gastroenteritis-associated vomiting. It is commonly prescribed at discharge to reduce symptoms, but evidence to support this practice is limited.
12. AAV9-Mediated Gene Therapy for Infantile-Onset Pompe's Disease.
作者: Xiuwei Ma.;Lu Zhuang.;Wenhao Ma.;Jun Li.;Yingying Mao.;Jianhua Wang.;Xiaodong Wang.;Juan Xu.;Shuangqing Yu.;Ruijie Gu.;Yongxia Wang.;Zhongqiu Li.;Xinyang Jiang.;Sheng Zhang.;Fang He.;Xiao Yang.;Lina Zhu.;Shan Zhang.;Yanping Zhang.;Ting Li.;Qiuping Li.;Zhijie Wu.;Cengceng Zhang.;Yiying Zhang.;Xiaoyan Dong.;Hui Xiong.;Xiaobing Wu.;Zhichun Feng.
来源: N Engl J Med. 2025年392卷24期2438-2446页
Four patients with infantile-onset Pompe's disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid α-glucosidase (GAA) (dose, 1.2 × 1014 vector genomes per kilogram of body weight). One patient was withdrawn from the study and subsequently died. The other patients had improvement in cardiac outcomes and motor function over a 52-week observation period. Anti-GAA antibodies were not detected in any of the patients during the observation period. Respiratory tract infections were the most common adverse events. (Funded by the National Natural Science Foundation of China and National High Level Hospital Clinical Research Funding; Chinese Clinical Trial Registry number, ChiCTR2200063229.).
13. Once-Monthly Maridebart Cafraglutide for the Treatment of Obesity - A Phase 2 Trial.
作者: Ania M Jastreboff.;Donna H Ryan.;Harold E Bays.;Peter R Ebeling.;Mia G Mackowski.;Nisha Philipose.;Leorah Ross.;Yimeng Liu.;Cassandra E Burns.;Siddique A Abbasi.;Nicola Pannacciulli.; .
来源: N Engl J Med. 2025年393卷9期843-857页
Maridebart cafraglutide (known as MariTide) is a long-acting peptide-antibody conjugate that combines glucagon-like peptide-1 receptor agonism and glucose-dependent insulinotropic polypeptide receptor antagonism and that is intended for the treatment of obesity.
14. Weekly Fixed-Dose Insulin Efsitora in Type 2 Diabetes without Previous Insulin Therapy.
作者: Julio Rosenstock.;Timothy Bailey.;Lisa Connery.;Eden Miller.;Cyrus Desouza.;Qianqian Wang.;Jennifer Leohr.;Alastair Knights.;Molly C Carr.;Christopher J Child.; .
来源: N Engl J Med. 2025年393卷4期325-335页
In previous treat-to-target trials, adjustments to the dose of basal insulin have been made at least weekly, according to fasting blood glucose levels. A fixed-dose regimen of insulin efsitora alfa (efsitora), a once-weekly basal insulin, may provide a benefit in adults with type 2 diabetes who have not received previous insulin therapy.
15. Orforglipron, an Oral Small-Molecule GLP-1 Receptor Agonist, in Early Type 2 Diabetes.
作者: Julio Rosenstock.;Stanley Hsia.;Luis Nevarez Ruiz.;Sarah Eyde.;David Cox.;Wen-Shuo Wu.;Rong Liu.;Jianghao Li.;Laura Fernández Landó.;Max Denning.;Lisa Ludwig.;Yanyun Chen.; .
来源: N Engl J Med. 2025年393卷11期1065-1076页
Orforglipron is a small-molecule, nonpeptide glucagon-like peptide-1 (GLP-1) receptor agonist in clinical development for type 2 diabetes and weight management. Additional data on the efficacy and safety of orforglipron are needed.
16. Coadministered Cagrilintide and Semaglutide in Adults with Overweight or Obesity.
作者: W Timothy Garvey.;Matthias Blüher.;Cynthia Karenina Osorto Contreras.;Melanie J Davies.;Eva Winning Lehmann.;Kirsi H Pietiläinen.;Domenica Rubino.;Paolo Sbraccia.;Thomas Wadden.;Niels Zeuthen.;John P H Wilding.; .
来源: N Engl J Med. 2025年393卷7期635-647页
Semaglutide at a dose of 2.4 mg has established weight-loss and cardiovascular benefits, and cagrilintide at a dose of 2.4 mg has shown promising results in early-phase trials; the efficacy of the combination (known as CagriSema) on weight loss in persons with either overweight and coexisting conditions or obesity is unknown.
17. Cagrilintide-Semaglutide in Adults with Overweight or Obesity and Type 2 Diabetes.
作者: Melanie J Davies.;Harpreet S Bajaj.;Christa Broholm.;Astrid Eliasen.;W Timothy Garvey.;Carel W le Roux.;Ildiko Lingvay.;Christian Bøge Lyndgaard.;Julio Rosenstock.;Sue D Pedersen.; .
来源: N Engl J Med. 2025年393卷7期648-659页
Cagrilintide and semaglutide have each been shown to induce weight loss as monotherapies. Data are needed on the coadministration of cagrilintide and semaglutide (called CagriSema) for weight management in adults with type 2 diabetes, including those in a subgroup who are undergoing continuous glucose monitoring.
18. Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes.
作者: Trevor W Reichman.;James F Markmann.;Jon Odorico.;Piotr Witkowski.;John J Fung.;Martin Wijkstrom.;Fouad Kandeel.;Eelco J P de Koning.;Anne L Peters.;Chantal Mathieu.;Leslie S Kean.;Bote G Bruinsma.;Chenkun Wang.;Molly Mascia.;Bastiano Sanna.;Gautham Marigowda.;Felicia Pagliuca.;Doug Melton.;Camillo Ricordi.;Michael R Rickels.; .
来源: N Engl J Med. 2025年393卷9期858-868页
Zimislecel is an allogeneic stem cell-derived islet-cell therapy. Data on the safety and efficacy of zimislecel in persons with type 1 diabetes are needed.
19. Neoadjuvant and Adjuvant Pembrolizumab in Locally Advanced Head and Neck Cancer.
作者: Ravindra Uppaluri.;Robert I Haddad.;Yungan Tao.;Christophe Le Tourneau.;Nancy Y Lee.;William Westra.;Rebecca Chernock.;Makoto Tahara.;Kevin J Harrington.;Arkadiy L Klochikhin.;Irene Braña.;Gustavo Vasconcelos Alves.;Brett G M Hughes.;Marc Oliva.;Iane Pinto Figueiredo Lima.;Tsutomu Ueda.;Tomasz Rutkowski.;Ursula Schroeder.;Paul-Stefan Mauz.;Thorsten Fuereder.;Simon Laban.;Nobuhiko Oridate.;Aron Popovtzer.;Nicolas Mach.;Yevhen Korobko.;Diogo Alpuim Costa.;Anupama Hooda-Nehra.;Cristina P Rodriguez.;R Bryan Bell.;Cole Manschot.;Kimberly Benjamin.;Burak Gumuscu.;Douglas Adkins.; .
来源: N Engl J Med. 2025年393卷1期37-50页
The benefit of the addition of perioperative pembrolizumab to standard care with surgery and adjuvant therapy for patients with locally advanced head and neck squamous-cell carcinoma (HNSCC) is unclear.
20. Bevacizumab and Erlotinib in Hereditary and Sporadic Papillary Kidney Cancer.
作者: Ramaprasad Srinivasan.;Sandeep Gurram.;Eric A Singer.;Abhinav Sidana.;Munjid Al Harthy.;Mark W Ball.;Julia C Friend.;Lisa Mac.;Erin Purcell.;Cathy D Vocke.;Christopher J Ricketts.;Heidi H Kong.;Edward W Cowen.;Ashkan A Malayeri.;Joanna H Shih.;Maria J Merino.;W Marston Linehan.
来源: N Engl J Med. 2025年392卷23期2346-2356页
Hereditary leiomyomatosis and renal-cell cancer (HLRCC) is an inherited disorder characterized by germline pathogenic variants in the gene encoding fumarate hydratase and an increased risk of papillary renal-cell carcinoma. No effective therapy is known for patients with advanced HLRCC-associated papillary renal-cell carcinoma, and most patients die from progressive disease.
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