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61. Belzutifan for Advanced Pheochromocytoma or Paraganglioma.
作者: Camilo Jimenez.;Mikkel Andreassen.;Alice Durand.;Sophie Moog.;Andrew Hendifar.;Staffan Welin.;Francesca Spada.;Rohini Sharma.;Edward Wolin.;Joseph Ruether.;Rocio Garcia-Carbonero.;Martin Fassnacht.;Jaume Capdevila.;Jaydira Del Rivero.;Othon Iliopoulos.;Olivier Huillard.;Raymond Jang.;Knut Mai.;Elena Artamonova.;Andreas Hallqvist.;Tobias Else.;Amos Odeleye-Ajakaye.;Alexander Gozman.;Girish S Naik.;Alfredo Berruti.; .
来源: N Engl J Med. 2025年393卷20期2012-2022页
Pheochromocytoma and paraganglioma are neoplasms originating in the adrenal medulla and extraadrenal paraganglia, respectively. Most cases of metastatic pheochromocytoma and paraganglioma are driven by dysregulation of the hypoxia-inducible factor 2α (HIF-2α) pathway. Belzutifan is a HIF-2α inhibitor that may provide antitumor activity in patients with advanced pheochromocytoma or paraganglioma.
62. Disitamab Vedotin plus Toripalimab in HER2-Expressing Advanced Urothelial Cancer.
作者: Xinan Sheng.;Gongqian Zeng.;Cuijian Zhang.;Qingyun Zhang.;Jiasheng Bian.;Haitao Niu.;Jun Li.;Yanxia Shi.;Kai Yao.;Bin Hu.;Ziling Liu.;Hong Liao.;Zhixian Yu.;Baiye Jin.;Peng Zhao.;Tiejun Yang.;Xianling Liu.;Yang Qin.;Xueyi Xue.;Xin Gou.;Jian Huang.;Jiang Gu.;Xiaolong Qi.;Lu Zhang.;Guoxian Ma.;Beisong Liu.;Jianmin Fang.;Shusuan Jiang.;Zhisong He.;Aiping Zhou.;Jun Guo.; .
来源: N Engl J Med. 2025年393卷23期2324-2337页
Human epidermal growth factor receptor 2 (HER2)-directed antibody-drug conjugate monotherapy has shown preliminary clinical efficacy in patients with chemotherapy-refractory HER2-positive locally advanced or metastatic urothelial cancer. Previous data showed promising antitumor activity and safety of HER2-specific disitamab vedotin as monotherapy and when combined with programmed cell death protein 1 (PD-1)-directed immunotherapy in this cancer.
63. ctDNA-Guided Adjuvant Atezolizumab in Muscle-Invasive Bladder Cancer.
作者: Thomas Powles.;Ariel G Kann.;Daniel Castellano.;Marine Gross-Goupil.;Hiroyuki Nishiyama.;Sergio Bracarda.;Jørgen Bjerggaard Jensen.;Lydia Makaroff.;Shusuan Jiang.;Ja Hyeon Ku.;Se Hoon Park.;Oscar Reig Torras.;Dingwei Ye.;Marco Maruzzo.;Andrea Necchi.;Rafael Morales-Barrera.;Emilio Francesco Giunta.;Jae Lyun Lee.;Giampaolo Tortora.;Yüksel Ürün.;Lukasz Dolowy.;Dilek Erdem.;Alvaro Pinto.;Fabricio Grando.;Wei Zou.;Zoe June Assaf.;Jacqueline Vuky.;Viraj Degaonkar.;Elizabeth E Steinberg.;Joaquim Bellmunt.;Jürgen E Gschwend.; .
来源: N Engl J Med. 2025年393卷24期2395-2408页
Patients with muscle-invasive bladder cancer have varied outcomes after cystectomy. Circulating tumor DNA (ctDNA)-based detection of molecular residual disease may identify patients at high risk for recurrence after cystectomy who can benefit from adjuvant immunotherapy, thus sparing patients at lower risk from unnecessary treatment burden.
64. Subretinal Photovoltaic Implant to Restore Vision in Geographic Atrophy Due to AMD.
作者: Frank G Holz.;Yannick Le Mer.;Mahiul M K Muqit.;Lars-Olof Hattenbach.;Andrea Cusumano.;Salvatore Grisanti.;Laurent Kodjikian.;Marco Andrea Pileri.;Frederic Matonti.;Eric Souied.;Boris V Stanzel.;Peter Szurman.;Michel Weber.;Karl Ulrich Bartz-Schmidt.;Nicole Eter.;Marie Noelle Delyfer.;Jean François Girmens.;Koen A van Overdam.;Armin Wolf.;Ralf Hornig.;Martina Corazzol.;Frank Brodie.;Lisa Olmos de Koo.;Daniel Palanker.;José-Alain Sahel.
来源: N Engl J Med. 2026年394卷3期232-242页
Geographic atrophy due to age-related macular degeneration (AMD) is the leading cause of irreversible blindness and affects more than 5 million persons worldwide. No therapies to restore vision in such persons currently exist. The photovoltaic retina implant microarray (PRIMA) system combines a subretinal photovoltaic implant and glasses that project near-infrared light to the implant in order to restore sight to areas of central retinal atrophy.
65. Improved Survival with Enzalutamide in Biochemically Recurrent Prostate Cancer.
作者: Neal D Shore.;Murilo de Almeida Luz.;Ugo De Giorgi.;Martin Gleave.;Geoffrey T Gotto.;Christopher M Pieczonka.;Gabriel P Haas.;Choung-Soo Kim.;Miguel Ramirez-Backhaus.;Antti Rannikko.;Matko Kalac.;Swetha Sridharan.;Matt Rosales.;Yiyun Tang.;Ronald F Tutrone.;Balaji Venugopal.;Arnauld Villers.;Henry H Woo.;Fong Wang.;Stephen J Freedland.
来源: N Engl J Med. 2026年394卷6期563-575页
In the phase 3 EMBARK trial, enzalutamide plus leuprolide and enzalutamide monotherapy were associated with longer metastasis-free survival than leuprolide alone among patients with biochemically recurrent prostate cancer. The final analysis of overall survival has not been reported.
66. Nivolumab for Resected Stage III or IV Melanoma at 9 Years.
作者: Paolo A Ascierto.;Michele Del Vecchio.;Barbara Merelli.;Helen Gogas.;Ana M Arance.;Stéphane Dalle.;Charles Lance Cowey.;Michael Schenker.;Caroline Gaudy-Marqueste.;Jacopo Pigozzo.;Iván Márquez-Rodas.;Marcus O Butler.;Anna Maria Di Giacomo.;Oleg Gligich.;Luis De La Cruz-Merino.;Petr Arenberger.;Victoria Atkinson.;Paul Nathan.;Andrew Hill.;Michael Millward.;Leslie A Fecher.;Nikhil I Khushalani.;Paola Queirolo.;Raheela Soomro.;Dhanrajsinh Rathod.;Margarita Askelson.;Melanie Pe Benito.;Devanand Joseph.;James Larkin.
来源: N Engl J Med. 2026年394卷4期333-342页
In the CheckMate 238 trial, patients with resected stage IIIB-C or stage IV melanoma who were treated with nivolumab had longer recurrence-free survival than those who received ipilimumab. Data were needed on longer-term survival.
67. Survival with Osimertinib plus Chemotherapy in EGFR-Mutated Advanced NSCLC.
作者: Pasi A Jänne.;David Planchard.;Kunihiko Kobayashi.;James Chih-Hsin Yang.;Ying Liu.;Natalia Valdiviezo.;Tae Min Kim.;Liyan Jiang.;Hiroshi Kagamu.;Noriko Yanagitani.;Jialei Wang.;Bivas Biswas.;Artem Poltoratskiy.;Yeni Neron.;Carlos Rojas.;Leona Koubkova.;Carles Escriu.;Doreen A Ezeife.;Helen Mann.;Elena Armenteros-Monterroso.;Yuri Rukazenkov.;Chee Khoon Lee.; .
来源: N Engl J Med. 2026年394卷1期27-38页
The primary analysis of this trial showed that first-line treatment with osimertinib plus chemotherapy with a platinum-based agent and pemetrexed led to significantly longer progression-free survival than osimertinib monotherapy among patients with epidermal growth factor receptor (EGFR)-mutated advanced non-small-cell lung cancer (NSCLC). Results from the planned final analysis of overall survival are needed.
68. Sevabertinib in Advanced HER2-Mutant Non-Small-Cell Lung Cancer.
作者: Xiuning Le.;Tae Min Kim.;Herbert H Loong.;Arsela Prelaj.;Boon Cher Goh.;Lin Li.;Yong Fang.;Shun Lu.;Xiaorong Dong.;Lin Wu.;Yuki Shinno.;Gennaro Daniele.;Tsung-Ying Yang.;Hye Ryun Kim.;Gerrina Ruiter.;Jun Zhao.;Silvia Novello.;Liyun Miao.;Pasi A Jänne.;Koichi Goto.;Dominik Rüttinger.;Tine Descamps.;Jan Christoph Brase.;Weichao Bao.;Rui Li.;Nicoletta Brega.;Paolo Grassi.;Nicolas Girard.;Daniel Shao-Weng Tan.; .
来源: N Engl J Med. 2025年393卷18期1819-1832页
HER2 gene mutations occur in 2 to 4% of patients with non-small-cell lung cancer (NSCLC). Sevabertinib is an oral, reversible tyrosine kinase inhibitor that has shown anti-HER2 activity in preclinical models.
69. Mass Administration of Azithromycin to Infants in Mali to Reduce Mortality.
作者: Fadima Cheick Haidara.;Laura Adubra.;Mahamadou Abdou.;Dagmar Alber.;Ulla Ashorn.;Yin Bun Cheung.;Elaine Cloutman-Green.;Mamadou Diallo.;Camilla Ducker.;Yue-Mei Fan.;Gwydion Gruffudd.;Lotta Hallamaa.;Tiia Haapaniemi.;Rikhard Ihamuotila.;Jane Juma.;Nigel Klein.;Juho Luoma.;Owen Martell.;Akshaya Murugesan.;Collins Okello.;Oumar Samaké.;Cheick Amadou Tidiane Traore.;Taru Vehmasto.;Kaisa Ylikruuvi.;Samba Sow.;Per Ashorn.
来源: N Engl J Med. 2025年393卷15期1498-1508页
Mass administration of azithromycin to children 1 to 59 months of age has been shown to reduce mortality among infants and children in this age group in some areas of sub-Saharan Africa. The largest effects have appeared to be among infants younger than 12 months of age, within 3 months after treatment; this observation motivated the design of the current trial.
70. A Phase 3 Trial of Telitacicept for Systemic Lupus Erythematosus.
作者: Ronald F van Vollenhoven.;Li Wang.;Joan T Merrill.;Yi Liu.;Chunde Bao.;Fen Li.;Jiankang Hu.;Chenghui Huang.;Jianhong Zhao.;Cibo Huang.;Hanyou Mo.;Wei Wei.;Fu'ai Lu.;Jingyang Li.;Dongbao Zhao.;Wenxiang Wang.;Lin Li.;Qing Zuraw.;Xiaofei Wang.;Xuebin Wang.;Jianmin Fang.;Fengchun Zhang.; .
来源: N Engl J Med. 2025年393卷15期1475-1485页
Telitacicept, a new dual inhibitor of the cytokines B-lymphocyte stimulator (BLyS) and APRIL (a proliferation-inducing ligand), showed efficacy in adults with active systemic lupus erythematosus (SLE) in a phase 2b trial when added to standard therapy.
71. Sentinel-Lymph-Node Biopsy Alone or with Lymphadenectomy in Cervical Cancer.
作者: Hua Tu.;He Huang.;Yanfang Li.;Xiaojun Chen.;Chunyan Wang.;Min Zheng.;Yanna Zhang.;Weidong Zhao.;Yanling Feng.;Ting Wan.;Yongwen Huang.;Aijun Yu.;Weiguo Lu.;Jing Xiao.;Weiwei Shan.;Ping Zhang.;Changkun Zhu.;Danbo Wang.;Hu Zhou.;Jibin Li.;Beihua Kong.;Weiwei Feng.;Xipeng Wang.;Rongzhen Luo.;Xin Huang.;Jundong Li.;Zejian Lin.;Shuzhong Yao.;Jihong Liu.; .
来源: N Engl J Med. 2025年393卷15期1463-1474页
Limited data are available on survival outcomes after sentinel-lymph-node biopsy alone as compared with lymphadenectomy in cervical cancer.
72. DB-OTO Gene Therapy for Inherited Deafness.
作者: Vassili Valayannopoulos.;Manohar Bance.;Daniela S Carvalho.;John H Greinwald.;Steven A Harvey.;Akira Ishiyama.;Evie C Landry.;Hubert Löwenheim.;Lawrence R Lustig.;Manuel Manrique.;Robert Nash.;Rubén Polo.;Cedric V Pritchett.;Jay T Rubinstein.;A Eliot Shearer.;Ignacio Del Castillo.;Jeffery J Anderson.;C Eduardo Corrales.;Tera M Quigley.;William J Riggs.;Peter Weber.;Gary Wilson.;Susan C Irvin.;Hazem E Hassan.;Yanping Chen.;Rong Liu.;Meghan C Drummond.;Leah R Sabin.;Bret J Musser.;George D Yancopoulos.;Christos A Kyratsous.;Gary A Herman.;Aris Baras.;Jonathon P Whitton.; .
来源: N Engl J Med. 2026年394卷11期1074-1083页
Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; children typically receive cochlear implants. DB-OTO is a dual adeno-associated virus 1 gene therapy that delivers human OTOF complementary DNA (encoding otoferlin) regulated by a hair cell-specific promoter.
73. Randomized Trial of Targeted Indoor Spraying to Prevent Aedes-Borne Diseases.
作者: Natalie E Dean.;Amy M Crisp.;Azael Che-Mendoza.;Oscar D Kirstein.;Gloria A Barrera-Fuentes.;James T Earnest.;Henry N Puerta-Guardo.;Matthew H Collins.;Norma Pavia-Ruz.;Guadalupe Ayora-Talavera.;Gabriela González-Olvera.;Anuar Medina-Barreiro.;Wilbert Bibiano-Marín.;Shirin Jabbarzadeh.;M Elizabeth Halloran.;Ira M Longini.;Audrey Lenhart.;Lance A Waller.;Fabian Correa-Morales.;Jorge Palacio-Vargas.;Hector Gomez-Dantes.;Pablo Manrique-Saide.;Gonzalo M Vazquez-Prokopec.
来源: N Engl J Med. 2025年393卷14期1387-1398页
Targeted indoor residual spraying focuses insecticide applications on common resting surfaces of Aedes aegypti mosquitoes (an arboviral disease vector) in houses, such as exposed lower sections of walls and under furniture.
74. Sotatercept for Pulmonary Arterial Hypertension within the First Year after Diagnosis.
作者: Vallerie V McLaughlin.;Marius M Hoeper.;David B Badesch.;H Ardeschir Ghofrani.;J Simon R Gibbs.;Mardi Gomberg-Maitland.;Ioana R Preston.;Rogerio Souza.;Aaron B Waxman.;Grzegorz Kopeć.;Gisela Meyer.;Karen M Olsson.;Wei Fu.;Yaru Shi.;Barry Miller.;Samuel S Kim.;Harald S Mackenzie.;Michela Brambatti.;Mahesh J Patel.;Joerg Koglin.;Alexandra G Cornell.;Marc Humbert.; .
来源: N Engl J Med. 2025年393卷16期1599-1611页
Sotatercept, an activin-signaling inhibitor, reduces morbidity and mortality among patients with long-standing pulmonary arterial hypertension. Its effects in patients with pulmonary arterial hypertension within the first year after diagnosis are unclear.
75. Hypertonic Saline or Carbocisteine in Bronchiectasis.
作者: Judy M Bradley.;Brenda O'Neill.;Daniel F McAuley.;James D Chalmers.;Anthony De Soyza.;Adam T Hill.;Mary Carroll.;Michael R Loebinger.;Jamie Duckers.;Mike Clarke.;Rebecca H McLeese.;Kathryn Ferguson.;Andrew Jackson.;Christina Campbell.;Clíona McDowell.;Ashley Agus.;John Norrie.;Fiona Copeland.;Damian G Downey.;Rory Convery.;Martin Kelly.;William Flight.;Nick P Talbot.;John R Hurst.;John Steer.;Muhammad Anwar.;Mitra Shahidi.;Timothy Gatheral.;Mohamed Etumi.;Anita L Sullivan.;Andreea Alina Ionescu.;Veeresh Patil.;Milan Bhattacharya.;Steven Caskey.;Denise Cosgrove.;Conor Hagan.;Amelia Shoemark.;Terence McManus.;Gareth Davies.;J Stuart Elborn.; .
来源: N Engl J Med. 2025年393卷16期1565-1577页
Bronchiectasis guidelines are inconsistent with regard to the effectiveness of mucoactive agents, and their use varies geographically. Large trials are needed to assess safety and effectiveness.
76. Nexiguran Ziclumeran Gene Editing in Hereditary ATTR with Polyneuropathy.
作者: Julian D Gillmore.;Ed Gane.;Jörg Täubel.;Björn Pilebro.;Andoni Echaniz-Laguna.;Justin Kao.;William Litchy.;Safi Shahda.;Alexandra Haagensen.;Liron Walsh.;Derek Smith.;Jessica Kachadourian.;Jonathan H Ward.;David Lebwohl.;Peijuan Zhu.;Yuanxin Xu.;Adia Leung.;Alison Sonderfan.;David E Gutstein.;Garen Manvelian.;David Adams.
来源: N Engl J Med. 2025年393卷14期1375-1386页
Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare, multisystem, progressive, debilitating, and fatal disease characterized by tissue deposition of misfolded transthyretin (TTR) in peripheral nerves. Nexiguran ziclumeran (nex-z) is an investigational in vivo therapy based on CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and associated Cas9 endonuclease) that is designed to reduce serum TTR levels through selective inactivation of TTR in the liver.
77. Permethrin-Treated Baby Wraps for the Prevention of Malaria.
作者: Ross M Boyce.;Bonnie E Shook-Sa.;Ronnie Ndizeye.;Emmanuel Baguma.;Dana Giandomenico.;Caitlin A Cassidy.;Solomon Eshun.;Mark J Siedner.;Sarah G Staedke.;Moses Ntaro.;Jonathan J Juliano.;Raquel Reyes.;Edgar M Mulogo.
来源: N Engl J Med. 2025年393卷14期1399-1408页
Malaria remains a major cause of childhood death in sub-Saharan Africa. We leveraged the traditional practice of mothers carrying children on their backs in cloth wraps to assess whether treating the wraps with an insect repellent might provide a layer of protection against malaria.
78. Clesrovimab for Prevention of RSV Disease in Healthy Infants.
作者: Heather J Zar.;Eric A F Simões.;Shabir A Madhi.;Octavio Ramilo.;Shelly D Senders.;Julie S Shepard.;Kamolwish Laoprasopwattana.;Jorge Piedrahita.;Jose M Novoa.;Sergio L Vargas.;Marc Dionne.;Teresa Jackowska.;Enmei Liu.;Yasunori Ishihara.;Kazushige Ikeda.;Ying Zhang.;Radha A Railkar.;Jeannine Lutkiewicz.;Kalpit A Vora.;Xiaowei Zang.;Brian M Maas.;Andrew W Lee.;Andrea Guerra.;Anushua Sinha.; .
来源: N Engl J Med. 2025年393卷13期1292-1303页
Clesrovimab is a long-acting investigational monoclonal antibody against site IV of the respiratory syncytial virus (RSV) fusion protein. Data regarding the safety and efficacy of clesrovimab in healthy infants are needed.
79. 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.
80. A Randomized Trial of Shunting for Idiopathic Normal-Pressure Hydrocephalus.
作者: Mark G Luciano.;Michael A Williams.;Mark G Hamilton.;Heather L Katzen.;Nickolas A Dasher.;Abhay Moghekar.;Jun Hua.;Jan Malm.;Anders Eklund.;Naomi Alpert Abel.;Ahmed M Raslan.;Benjamin D Elder.;Jesse J Savage.;Daniel L Barrow.;Kiarash Shahlaie.;Hailey Jensen.;Thomas J Zwimpfer.;Jessica Wollett.;Daniel F Hanley.;Richard Holubkov.; .
来源: N Engl J Med. 2025年393卷22期2198-2209页
Idiopathic normal-pressure hydrocephalus is a neurologic disorder characterized by impaired gait, balance, cognition, and bladder control in older adults. The disorder is treated with shunt surgery, but the effectiveness of shunting is unclear.
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