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1. 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.
2. Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
作者: Ulrike M Reiss.;Andrew M Davidoff.;Edward G D Tuddenham.;Pratima Chowdary.;Jenny McIntosh.;Vincent Muczynski.;Malo Journou.;Giulia Simini.;Lydia Ireland.;Saira Mohamed.;Anne Riddell.;Arnulfo J Pie.;Andrew Hall.;Alberto Quaglia.;Sarah Mangles.;Johnny Mahlangu.;Kristina Haley.;Michael Recht.;Yu-Min Shen.;Kathleen G Halka.;Gail Fortner.;Christopher L Morton.;Zhengming Gu.;Randall T Hayden.;Ellis J Neufeld.;Victoria I Okhomina.;Guolian Kang.;Amit C Nathwani.
来源: N Engl J Med. 2025年392卷22期2226-2234页
Adeno-associated virus (AAV)-mediated gene therapy has emerged as a promising treatment for hemophilia B. Data on safety and durability from 13 years of follow-up in a cohort of patients who had been successfully treated with scAAV2/8-LP1-hFIXco gene therapy are now available.
3. A Trial of Trimethoprim-Sulfamethoxazole in Pregnancy to Improve Birth Outcomes.
作者: Bernard Chasekwa.;Fortunate Munhanzi.;Lenin Madhuyu.;Gabriel Mbewe.;Vincent Mabika.;Dzivaidzo Chidhanguro.;Tendai Kofi.;Jonthan Munengiwa.;Hilda Mapfumo.;Mercy Musapa.;Sipho Shumba.;Elizabeth Hungwe.;Mary Nhokwara.;Nester Bushe.;Rudo Kufa.;Phatisiwe Mazula.;Muchaneta Chikombingo.;Alice Tengende.;Admire Zanga.;Asaph Ziruma.;Tsitsi Bere.;Success Munyengwa.;Charity Mudimbu.;Zvikomborero Murwira.;Shepherd Mudzingwa.;Eddington Mpofu.;Batsirai Mutasa.;Virginia Sauramba.;Elisha Masakadze.;Thompson Runodamoto.;Courage Chiorera.;Alfred Mushininga.;Claire D Bourke.;Ruairi C Robertson.;Jeniffer Perussolo.;Nikos Donos.;Chandiwana Nyachowe.;Mary Muchekeza.;Jefrey Chikunya.;Melanie Smuk.;Kuda Mutasa.;Naume V Tavengwa.;Lisa L Langhaug.;Robert Ntozini.;Stephen P Munjanja.;Andrew J Prendergast.
来源: N Engl J Med. 2025年392卷21期2125-2134页
Maternal infections underlie several adverse birth outcomes. Whether trimethoprim-sulfamethoxazole prophylaxis during pregnancy will improve birth outcomes is unknown.
4. Omitting Regional Nodal Irradiation after Response to Neoadjuvant Chemotherapy.
作者: Eleftherios P Mamounas.;Hanna Bandos.;Julia R White.;Thomas B Julian.;Atif J Khan.;Simona F Shaitelman.;Mylin A Torres.;Frank A Vicini.;Patricia A Ganz.;Susan A McCloskey.;Peter C Lucas.;Nilendu Gupta.;X Allen Li.;Beryl McCormick.;Benjamin Smith.;Rahul D Tendulkar.;Vivek S Kavadi.;Koji Matsumoto.;Samantha Andrews Seaward.;William J Irvin.;Jolinta Y Lin.;Robert W Mutter.;Thierry M Muanza.;Jannifer Stromberg.;Reshma Jagsi.;Anna C Weiss.;Walter J Curran.;Norman Wolmark.
来源: N Engl J Med. 2025年392卷21期2113-2124页
The benefit of regional nodal irradiation in the treatment of breast cancer is well established for patients with pathologically positive axillary nodes, but whether it is also beneficial for patients whose nodes become pathologically tumor free (ypN0) after neoadjuvant chemotherapy remains unclear.
5. Once-Weekly Mazdutide in Chinese Adults with Obesity or Overweight.
作者: Linong Ji.;Hongwei Jiang.;Yan Bi.;Hua Li.;Junhang Tian.;Dexue Liu.;Yuzhu Zhao.;Wei Qiu.;Chongbing Huang.;Lei Chen.;Shao Zhong.;Jie Han.;Yawei Zhang.;Qiufang Lian.;Ping Yang.;Lingchun Lv.;Jieyu Gu.;Zihan Liu.;Huan Deng.;Yanqi Wang.;Li Li.;Lijuan Pei.;Lei Qian.; .
来源: N Engl J Med. 2025年392卷22期2215-2225页
Evidence suggests that incretin-based dual agonist pharmacotherapy is helpful in persons with obesity. Mazdutide, a glucagon-like peptide-1 and glucagon receptor dual agonist, may have efficacy in persons with overweight or obesity.
6. Nerandomilast in Patients with Progressive Pulmonary Fibrosis.
作者: Toby M Maher.;Shervin Assassi.;Arata Azuma.;Vincent Cottin.;Anna-Maria Hoffmann-Vold.;Michael Kreuter.;Justin M Oldham.;Luca Richeldi.;Claudia Valenzuela.;Marlies S Wijsenbeek.;Emmanuelle Clerisme-Beaty.;Carl Coeck.;Hui Gu.;Ivana Ritter.;Arno Schlosser.;Susanne Stowasser.;Florian Voss.;Gerrit Weimann.;Donald F Zoz.;Fernando J Martinez.; .
来源: N Engl J Med. 2025年392卷22期2203-2214页
Nerandomilast (BI 1015550) is an orally administered preferential inhibitor of phosphodiesterase 4B with antifibrotic and immunomodulatory properties. Nerandomilast has been shown to slow the progression of idiopathic pulmonary fibrosis, but an assessment of its effects in other types of progressive pulmonary fibrosis is needed.
7. Nerandomilast in Patients with Idiopathic Pulmonary Fibrosis.
作者: Luca Richeldi.;Arata Azuma.;Vincent Cottin.;Michael Kreuter.;Toby M Maher.;Fernando J Martinez.;Justin M Oldham.;Claudia Valenzuela.;Emmanuelle Clerisme-Beaty.;Maud Gordat.;Daniel Wachtlin.;Yi Liu.;Christina Schlecker.;Susanne Stowasser.;Donald F Zoz.;Marlies S Wijsenbeek.; .
来源: N Engl J Med. 2025年392卷22期2193-2202页
Nerandomilast (BI 1015550) is an orally administered preferential inhibitor of phosphodiesterase 4B with antifibrotic and immunomodulatory effects. In a phase 2 trial involving patients with idiopathic pulmonary fibrosis, treatment with nerandomilast stabilized lung function over a period of 12 weeks.
8. Navigational Bronchoscopy or Transthoracic Needle Biopsy for Lung Nodules.
作者: Robert J Lentz.;Katherine Frederick-Dyer.;Virginia B Planz.;Tatsuki Koyama.;Matthew C Aboudara.;Sameer K Avasarala.;Jonathan D Casey.;George Z Cheng.;Pierre-François D'Haese.;Jennifer D Duke.;Eric L Grogan.;Todd C Hoopman.;Joyce Johnson.;James M Katsis.;Jonathan S Kurman.;See-Wei Low.;Kamran Mahmood.;Otis B Rickman.;Lance Roller.;Cristina Salmon.;Samira Shojaee.;Briana Swanner.;Momen M Wahidi.;Charla Walston.;Gerard A Silvestri.;Lonny Yarmus.;Najib M Rahman.;Fabien Maldonado.; .
来源: N Engl J Med. 2025年392卷21期2100-2112页
Each year, millions of pulmonary nodules are identified incidentally or through lung cancer screening, and many involve biopsy to distinguish cancer from benign processes. Both navigational bronchoscopy and computed tomography-guided transthoracic needle biopsy are commonly used in patients undergoing biopsies of peripheral pulmonary nodules, but the relative diagnostic accuracy of these two approaches is unclear.
9. Second-Line Antiretroviral Therapy for Children Living with HIV in Africa.
作者: Victor Musiime.;Mutsa Bwakura-Dangarembizi.;Alexander J Szubert.;Vivian Mumbiro.;Hilda A Mujuru.;Cissy M Kityo.;Abbas Lugemwa.;Katja Doerholt.;Chishala Chabala.;Shafic Makumbi.;Veronica Mulenga.;Helen McIlleron.;David Burger.;Eva Natukunda.;Clare Shakeshaft.;Kyomuhendo Jovia Linda.;Kusum Nathoo.;Lara Monkiewicz.;Ibrahim Yawe.;Monica Kapasa.;Mary Nyathi.;Joyce Lungu.;Bwendo Nduna.;Wedu Ndebele.;Annabelle South.;Mwate Mwamabazi.;Godfrey Musoro.;Anna Griffiths.;Khozya Zyambo.;Rashidah Nazzinda.;Kevin Zimba.;Yingying Zhang.;Simon Walker.;Anna Turkova.;A Sarah Walker.;Alasdair Bamford.;Diana M Gibb.; .
来源: N Engl J Med. 2025年392卷19期1917-1932页
Children living with human immunodeficiency virus (HIV) have limited options for second-line antiretroviral therapy (ART).
10. Oveporexton, an Oral Orexin Receptor 2-Selective Agonist, in Narcolepsy Type 1.
作者: Yves Dauvilliers.;Giuseppe Plazzi.;Emmanuel Mignot.;Gert Jan Lammers.;Rafael Del Río Villegas.;Ramin Khatami.;Mitsutaka Taniguchi.;Anson Abraham.;Yaming Hang.;Harisha Kadali.;Marta Lamberton.;Sarah Sheikh.;Ellie Stukalin.;Rachel Neuwirth.;Todd J Swick.;Shinichiro Tanaka.;Christian von Hehn.;Philipp von Rosenstiel.;Hao Wang.;Alice Cai.;Melissa Naylor.;Tina Olsson.
来源: N Engl J Med. 2025年392卷19期1905-1916页
Narcolepsy type 1 is a disorder of hypersomnolence caused by a loss of orexin neurons, which results in low orexin levels in the brain.
11. Enhanced CAR T-Cell Therapy for Lymphoma after Previous Failure.
作者: Jakub Svoboda.;Daniel J Landsburg.;James Gerson.;Sunita D Nasta.;Stefan K Barta.;Elise A Chong.;Michael Cook.;Noelle V Frey.;Joanne Shea.;Amanda Cervini.;Amy Marshall.;Megan Four.;Megan M Davis.;Julie K Jadlowsky.;Anne Chew.;Edward Pequignot.;Vanessa Gonzalez.;Julia Han Noll.;Luca Paruzzo.;Juliana Rojas-Levine.;Gabriela Plesa.;John Scholler.;Donald L Siegel.;Bruce L Levine.;David L Porter.;Saba Ghassemi.;Marco Ruella.;Andrew Rech.;Rachel M Leskowitz.;Joseph A Fraietta.;Wei-Ting Hwang.;Elizabeth Hexner.;Stephen J Schuster.;Carl H June.
来源: N Engl J Med. 2025年392卷18期1824-1835页
Chimeric antigen receptor (CAR) T cells targeting CD19 have transformed the treatment of B-cell cancers, but many patients do not have long-term remission. We designed an anti-CD19 enhanced (armored) CAR T-cell product (huCART19-IL18) that secretes interleukin-18 to enhance antitumor activity.
12. BCG Revaccination for the Prevention of Mycobacterium tuberculosis Infection.
作者: Alexander C Schmidt.;Lee Fairlie.;Elizabeth Hellström.;Angelique Luabeya Kany Kany.;Keren Middelkoop.;Kogieleum Naidoo.;Gonasagrie Nair.;Anele Gela.;Elisa Nemes.;Thomas J Scriba.;Amy Cinar.;Nicole Frahm.;Robin Mogg.;David Kaufman.;Michael W Dunne.;Mark Hatherill.; .
来源: N Engl J Med. 2025年392卷18期1789-1800页
In a previous phase 2 trial, bacille Calmette-Guérin (BCG) revaccination was not shown to provide protection from primary Mycobacterium tuberculosis infection but prevented sustained M. tuberculosis infection, defined by an initial conversion on a QuantiFERON-TB (QFT) test (an interferon-γ release assay) from negative to positive, followed by two additional positive QFT tests at 3 and 6 months after the initial conversion (a secondary end point). A vaccine efficacy of 45% (95% confidence interval [CI], 6 to 68) was observed.
13. Mepolizumab to Prevent Exacerbations of COPD with an Eosinophilic Phenotype.
作者: Frank C Sciurba.;Gerard J Criner.;Stephanie A Christenson.;Fernando J Martinez.;Alberto Papi.;Nicolas Roche.;Jean Bourbeau.;Stephanie Korn.;Mona Bafadhel.;MeiLan K Han.;Stefanie Kolterer.;Karen Miller.;Dalal Mouneimne.;Joanne Fletcher.;Bhabita Mayer.;Jeff Min.;Ian D Pavord.; .
来源: N Engl J Med. 2025年392卷17期1710-1720页
Mepolizumab is a humanized monoclonal antibody that targets interleukin-5, a cytokine that plays a central role in eosinophilic inflammation, which is present in 20 to 40% of patients with chronic obstructive pulmonary disease (COPD).
14. Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
作者: Claire Booth.;Julián Sevilla.;Elena Almarza.;Caroline Y Kuo.;Josune Zubicaray.;Dayna Terrazas.;Gráinne O'Toole.;Maria Chitty-Lopez.;Grace Choi.;Eileen Nicoletti.;Janel Long-Boyle.;Augustine Fernandes.;Kritika Chetty.;Satiro De Oliveira.;Crystal Banuelos.;Jinhua Xu-Bayford.;Antonella Lucía Bastone.;Philipp John-Neek.;Connie Jackson.;Theodore B Moore.;Kimberly Gilmour.;Axel Schambach.;Michael Rothe.;Sanchali Kasbekar.;Gayatri R Rao.;Kinnari Patel.;Gaurav Shah.;Adrian J Thrasher.;Juan A Bueren.;Jonathan D Schwartz.;Donald B Kohn.
来源: N Engl J Med. 2025年392卷17期1698-1709页
The β2 common integrin subunit CD18 is essential for leukocyte-endothelial adhesion and extravasation to inflamed or infected tissue. Damaging variants in ITGB2, which encodes CD18, cause leukocyte adhesion deficiency type I (LAD-I), an inborn error of immunity that leads to frequent life-threatening infections and a high risk of death among affected children. Allogeneic hematopoietic stem-cell transplantation (HSCT) represents a curative treatment but is limited by donor availability, a high incidence of graft-versus-host disease, and graft failure.
15. Phase 3 Trial of Semaglutide in Metabolic Dysfunction-Associated Steatohepatitis.
作者: Arun J Sanyal.;Philip N Newsome.;Iris Kliers.;Laura Harms Østergaard.;Michelle T Long.;Mette Skalshøi Kjær.;Anna M G Cali.;Elisabetta Bugianesi.;Mary E Rinella.;Michael Roden.;Vlad Ratziu.; .
来源: N Engl J Med. 2025年392卷21期2089-2099页
Semaglutide, a glucagon-like peptide-1 receptor agonist, is a candidate for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
16. Zongertinib in Previously Treated HER2-Mutant Non-Small-Cell Lung Cancer.
作者: John V Heymach.;Gerrina Ruiter.;Myung-Ju Ahn.;Nicolas Girard.;Egbert F Smit.;David Planchard.;Yi-Long Wu.;Byoung Chul Cho.;Noboru Yamamoto.;Joshua K Sabari.;Yanqiu Zhao.;Hai-Yan Tu.;Kiyotaka Yoh.;Ernest Nadal.;Behbood Sadrolhefazi.;Maren Rohrbacher.;Ute von Wangenheim.;Sabina Eigenbrod-Giese.;Jon Zugazagoitia.; .
来源: N Engl J Med. 2025年392卷23期2321-2333页
Innovative oral targeted therapies are warranted for patients with human epidermal growth factor receptor 2 (HER2)-mutant non-small-cell lung cancer (NSCLC). Zongertinib is an oral, irreversible, HER2-selective tyrosine kinase inhibitor that has been shown to have efficacy in persons with advanced or metastatic solid tumors with HER2 alterations in a phase 1 study.
17. Nonoperative Management of Mismatch Repair-Deficient Tumors.
作者: Andrea Cercek.;Michael B Foote.;Benoit Rousseau.;J Joshua Smith.;Jinru Shia.;Jenna Sinopoli.;Jill Weiss.;Melissa Lumish.;Lindsay Temple.;Miteshkumar Patel.;Callahan Wilde.;Leonard B Saltz.;Guillem Argiles.;Zsofia Stadler.;Oliver Artz.;Steven Maron.;Geoffrey Ku.;Ping Gu.;Yelena Y Janjigian.;Daniela Molena.;Gopa Iyer.;Jonathan Coleman.;Wassim Abida.;Seth Cohen.;Kevin Soares.;Mark Schattner.;Vivian E Strong.;Rona Yaeger.;Philip Paty.;Marina Shcherba.;Ryan Sugarman.;Paul B Romesser.;Alice Zervoudakis.;Avni Desai.;Neil H Segal.;Imane El Dika.;Maria Widmar.;Iris Wei.;Emmanouil Pappou.;Gerard Fumo.;Santiago Aparo.;Mithat Gonen.;Marc Gollub.;Vetri S Jayaprakasam.;Tae-Hyung Kim.;Julio Garcia Aguilar.;Martin Weiser.;Luis A Diaz.
来源: N Engl J Med. 2025年392卷23期2297-2308页
Among patients with mismatch repair-deficient (dMMR), locally advanced rectal cancer, neoadjuvant checkpoint blockade eliminated the need for surgery in a high proportion of patients. Whether this approach can be extended to all early-stage dMMR solid tumors, regardless of tumor site, is unknown.
18. Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
作者: Francesca Fumagalli.;Valeria Calbi.;Vera Gallo.;Alberto Andrea Zambon.;Salvatore Recupero.;Francesca Ciotti.;Marina Sarzana.;Maddalena Fraschini.;Stefano Scarparo.;Fabiola De Mattia.;Simona Miglietta.;Clelia Pierini.;Matias Soncini.;Francesco Morena.;Eugenio Montini.;Federica Barzaghi.;Giulia Consiglieri.;Francesca Ferrua.;Maddalena Migliavacca.;Francesca Tucci.;Elena Sophia Fratini.;Alessia Ippolito.;Paolo Silvani.;Maria Rosa Calvi.;Alessandra Clerici.;Ambra Corti.;Marcella Facchini.;Sara Locatelli.;Mara Sangalli.;Stefano Zancan.;Federica Miotto.;Maria Grazia Natali Sora.;Cristina Baldoli.;Sabata Martino.;Angélica Córdoba-Claros.;Sean L Moro.;Nicholas D Gollop.;Jeff Abate.;Muska N Yarzi.;Philippa Nutkins.;Andrew Shenker.;Mattia Calissano.;Jean Brooks.;Alan Richardson.;Laura Campbell.;Massimo Filippi.;Luigi Naldini.;Maria Pia Cicalese.;Fabio Ciceri.;Maria Ester Bernardo.;Alessandro Aiuti.
来源: N Engl J Med. 2025年392卷16期1609-1620页
Metachromatic leukodystrophy (MLD) is an ultrarare, severe lysosomal storage disorder caused by a deficiency of arylsulfatase A (ARSA).
19. Efficacy of Baloxavir Treatment in Preventing Transmission of Influenza.
作者: Arnold S Monto.;Klaus Kuhlbusch.;Corrado Bernasconi.;Bin Cao.;Herman Avner Cohen.;Emily Graham.;Aeron C Hurt.;Laurie Katugampola.;Takashi Kamezawa.;Adam S Lauring.;Barry McLean.;Takahiro Takazono.;Andreas Widmer.;Steffen Wildum.;Benjamin J Cowling.
来源: N Engl J Med. 2025年392卷16期1582-1593页
Baloxavir marboxil (baloxavir) rapidly reduces influenza virus shedding, which suggests that it may reduce transmission. Studies of treatment with neuraminidase inhibitors have not shown sufficient evidence that they prevent transmission to contacts.
20. Phase 3 Trial of the DPP-1 Inhibitor Brensocatib in Bronchiectasis.
作者: James D Chalmers.;Pierre-Régis Burgel.;Charles L Daley.;Anthony De Soyza.;Charles S Haworth.;David Mauger.;Michael R Loebinger.;Pamela J McShane.;Felix C Ringshausen.;Francesco Blasi.;Michal Shteinberg.;Kevin Mange.;Ariel Teper.;Carlos Fernandez.;Migdalia Zambrano.;Chunpeng Fan.;Xiangmin Zhang.;Mark L Metersky.; .
来源: N Engl J Med. 2025年392卷16期1569-1581页
In bronchiectasis, neutrophilic inflammation is associated with an increased risk of exacerbations and disease progression. Brensocatib, an oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP-1), targets neutrophil serine proteases, key mediators of neutrophilic inflammation.
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