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1. 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).
2. 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.
3. 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.
4. 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.
5. 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).
6. 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.
7. 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).
8. 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.
9. 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.
10. Lorundrostat Efficacy and Safety in Patients with Uncontrolled Hypertension.
作者: Luke J Laffin.;Branko Kopjar.;Carrie Melgaard.;Kathy Wolski.;Jessica Ibbitson.;Shivani Bhikam.;Matthew R Weir.;Elizabeth O Ofili.;Reena Mehra.;James M Luther.;Debbie L Cohen.;Ashish Sarraju.;Michael J Wilkinson.;John M Flack.;David Rodman.;Steven E Nissen.; .
来源: N Engl J Med. 2025年392卷18期1813-1823页
Aldosterone dysregulation contributes to hypertension. Lorundrostat is an aldosterone synthase inhibitor, but data on its efficacy and safety in patients with hypertension are limited.
11. Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study.
作者: John E J Rasko.;Benjamin J Samelson-Jones.;Lindsey A George.;Adam Giermasz.;Jonathan M Ducore.;Jerome M Teitel.;Catherine E McGuinn.;Katherine A High.;Ype P de Jong.;Amit Chhabra.;Amanda O'Brien.;Lynne M Smith.;Ian Winburn.;Jeremy Rupon.
来源: N Engl J Med. 2025年392卷15期1508-1517页
Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX variant (FIX-R338L, or FIX-Padua) in a phase 1-2a study. The long-term safety and efficacy of this treatment are not known.
12. Tecovirimat for Clade I MPXV Infection in the Democratic Republic of Congo.
作者: .;Rosine Ali.;Jules Alonga.;Jean-Luc Biampata.;Michael Kombozi Basika.;Irina Maljkovic Berry.;Nella Bisento.;Emily Blum.;Tyler Bonnett.;Katherine Cone.;Ian Crozier.;Richard Davey.;Ali Dilu.;Lori E Dodd.;Iman Gulati.;Dennis Hruby.;Augustin Ibanda.;Francis Isse.;Sylva Sivasingana Kasareka.;Gaby Kayembe.;Richard Kojan.;Esaie Kindombe Luzolo.;H Clifford Lane.;Leader Lawanga.;Laurens Liesenborghs.;Claude Shosongo Lunghe.;Yves Lula.;Mariano Lusakibanza.;Gaston Tona Lutete.;Placide Mbala-Kingebeni.;Alejandra Miranda.;Daniel Mukadi-Bamuleka.;Gael Mukendi.;Patrick Mutombo Lupola.;Jean-Jacques Muyembe-Tamfum.;Robin Ndungunu.;Bruce Nganga.;Nsengi Ntamabyaliro.;Veronique Nussenblatt.;Imoite Omulepu.;John Omalokoho Onosomba.;Michael Proschan.;Kevin Rubenstein.;Inga Saknite.;Adam Schechner.;Kathryn Shaw-Saliba.;Billy Sivahera.;Mary Smolskis.;Amy Tillman.;Eric Tkaczyk.;Celestin Tshimanga.;Olivier Tshiani Mbaya.;Antoine Tshomba.;Freddy Yemba Unda Tshomba.;David Vallee.;Susan Vogel.;Shera Weyers.
来源: N Engl J Med. 2025年392卷15期1484-1496页
Tecovirimat is available for the treatment of mpox (formerly known as monkeypox) in Europe and the United States, on the basis of findings from efficacy studies in animals and safety evaluations in healthy humans. Evidence from randomized, controlled trials of safety and efficacy in patients with mpox is lacking.
13. Tolebrutinib in Nonrelapsing Secondary Progressive Multiple Sclerosis.
作者: Robert J Fox.;Amit Bar-Or.;Anthony Traboulsee.;Celia Oreja-Guevara.;Gavin Giovannoni.;Patrick Vermersch.;Sana Syed.;Ye Li.;Wendy S Vargas.;Timothy J Turner.;Erik Wallstroem.;Daniel S Reich.; .
来源: N Engl J Med. 2025年392卷19期1883-1892页
Throughout the course of multiple sclerosis, gradually progressive neurologic impairment can occur, which has been called disability accrual. Current disease-modifying therapies for multiple sclerosis have limited effects on disability accrual unrelated to relapses, which is thought to be partially caused by chronic, nonresolving neuroinflammation within the central nervous system. Tolebrutinib is an oral, brain-penetrant Bruton's tyrosine kinase inhibitor that targets myeloid cells (including microglia) and B cells in both the periphery and central nervous system. There are no approved treatments for nonrelapsing secondary progressive multiple sclerosis.
14. Tolebrutinib versus Teriflunomide in Relapsing Multiple Sclerosis.
作者: Jiwon Oh.;Douglas L Arnold.;Bruce A C Cree.;Carolina Ionete.;Ho Jin Kim.;Maria Pia Sormani.;Sana Syed.;Yixin Chen.;Christina R Maxwell.;Patrick Benoit.;Timothy J Turner.;Erik Wallstroem.;Heinz Wiendl.; .
来源: N Engl J Med. 2025年392卷19期1893-1904页
Tolebrutinib is an oral, brain-penetrant, and bioactive Bruton's tyrosine kinase inhibitor that modulates peripheral inflammation and persistent immune activation within the central nervous system, including disease-associated microglia and B cells. More data are needed on its efficacy and safety in treating relapsing multiple sclerosis.
15. Alteplase for Posterior Circulation Ischemic Stroke at 4.5 to 24 Hours.
作者: Shenqiang Yan.;Ying Zhou.;Maarten G Lansberg.;David S Liebeskind.;Changzheng Yuan.;Han Yu.;Fujian Chen.;Hongfang Chen.;Bing Zhang.;Lingqun Mao.;Xiaoling Zhang.;Xiaona Wang.;Xuting Zhang.;Yi Chen.;Huan Zhou.;Wansi Zhong.;Yaode He.;Kun Chen.;Jianbing Wang.;Hui Chen.;Yuhui Huang.;Bruce C V Campbell.;Min Lou.; .
来源: N Engl J Med. 2025年392卷13期1288-1296页
The effects and risks of the use of intravenous thrombolysis between 4.5 and 24 hours after the onset of a posterior circulation ischemic stroke are not well studied.
16. Pulsed Field or Cryoballoon Ablation for Paroxysmal Atrial Fibrillation.
作者: Tobias Reichlin.;Thomas Kueffer.;Patrick Badertscher.;Peter Jüni.;Sven Knecht.;Gregor Thalmann.;Nikola Kozhuharov.;Philipp Krisai.;Corinne Jufer.;Jens Maurhofer.;Dik Heg.;Tiago V Pereira.;Felix Mahfoud.;Helge Servatius.;Hildegard Tanner.;Michael Kühne.;Laurent Roten.;Christian Sticherling.; .
来源: N Engl J Med. 2025年392卷15期1497-1507页
Pulmonary-vein isolation is an effective treatment for paroxysmal atrial fibrillation. Pulsed field ablation (PFA) is a nonthermal ablation method with few adverse effects beyond the myocardium. Data are lacking on outcomes after PFA as compared with cryoballoon ablation as assessed with continuous rhythm monitoring.
17. Lepodisiran - A Long-Duration Small Interfering RNA Targeting Lipoprotein(a).
作者: Steven E Nissen.;Wei Ni.;Xi Shen.;Qiuqing Wang.;Ann Marie Navar.;Stephen J Nicholls.;Kathy Wolski.;Laura Michael.;Axel Haupt.;John H Krege.; .
来源: N Engl J Med. 2025年392卷17期1673-1683页
Elevated lipoprotein(a) concentrations are associated with atherosclerotic cardiovascular disease. The safety and efficacy of lepodisiran, an extended-duration, small interfering RNA targeting hepatic synthesis of lipoprotein(a), are unknown.
18. Dapagliflozin in Patients Undergoing Transcatheter Aortic-Valve Implantation.
作者: Sergio Raposeiras-Roubin.;Ignacio J Amat-Santos.;Xavier Rossello.;Rocío González Ferreiro.;Inmaculada González Bermúdez.;Diego Lopez Otero.;Luis Nombela-Franco.;Livia Gheorghe.;Jose L Diez.;Carlos Baladrón Zorita.;José A Baz.;Antonio J Muñoz García.;Victoria Vilalta.;Soledad Ojeda-Pineda.;José M de la Torre Hernández.;Juan G Cordoba Soriano.;Ander Regueiro.;Pascual Bordes Siscar.;Jorge Salgado Fernández.;Bruno Garcia Del Blanco.;Roberto Martín-Reyes.;Rafael Romaguera.;César Moris.;Sergio García Blas.;Juan A Franco-Peláez.;Ignacio Cruz-González.;Dabit Arzamendi.;Nieves Romero Rodríguez.;Felipe Díez-Del Hoyo.;Santiago Camacho Freire.;Francisco Bosa Ojeda.;Juan C Astorga Burgo.;Eduardo Molina Navarro.;Juan Caballero Borrego.;Valeriano Ruiz Quevedo.;Ángel Sánchez-Recalde.;Vicente Peral Disdier.;Eduardo Alegría-Barrero.;Javier Torres-Llergo.;Gisela Feltes.;José A Fernández Díaz.;Carlos Cuellas.;Gustavo Jiménez Britez.;Juan Sánchez-Rubio Lezcano.;Cristina Barreiro-Pardal.;Iván Núñez-Gil.;Emad Abu-Assi.;Andrés Iñiguez-Romo.;Valentín Fuster.;Borja Ibáñez.; .
来源: N Engl J Med. 2025年392卷14期1396-1405页
Sodium-glucose cotransporter 2 (SGLT2) inhibitors reduce the risk of heart-failure admission among high-risk patients. However, most patients with valvular heart disease, including those undergoing transcatheter aortic-valve implantation (TAVI), have been excluded from randomized trials.
19. Extended Reduced-Dose Apixaban for Cancer-Associated Venous Thromboembolism.
作者: Isabelle Mahé.;Marc Carrier.;Didier Mayeur.;Jean Chidiac.;Eric Vicaut.;Nicolas Falvo.;Olivier Sanchez.;Claire Grange.;Manuel Monreal.;Juan J López-Núñez.;Remedios Otero-Candelera.;Grégoire Le Gal.;Erik Yeo.;Marc Righini.;Helia Robert-Ebadi.;Menno V Huisman.;Frederikus A Klok.;Peter Westerweel.;Giancarlo Agnelli.;Cecilia Becattini.;Aristotelis Bamias.;Kostas Syrigos.;Sebastian Szmit.;Adam Torbicki.;Peter Verhamme.;Anthony Maraveyas.;Alexander T Cohen.;Cihan Ay.;Céline Chapelle.;Guy Meyer.;Francis Couturaud.;Patrick Mismetti.;Philippe Girard.;Laurent Bertoletti.;Silvy Laporte.; .
来源: N Engl J Med. 2025年392卷14期1363-1373页
In patients with active cancer and venous thromboembolism, whether extended treatment with a reduced dose of an oral anticoagulant is effective in preventing recurrent thromboembolic events and decreasing bleeding is unclear.
20. Tezepelumab in Adults with Severe Chronic Rhinosinusitis with Nasal Polyps.
作者: Brian J Lipworth.;Joseph K Han.;Martin Desrosiers.;Claire Hopkins.;Stella E Lee.;Joaquim Mullol.;Oliver Pfaar.;Ting Li.;Claudia Chen.;Gun Almqvist.;Mary Kay Margolis.;Julie McLaren.;Shankar Jagadeesh.;Jamie MacKay.;Ayman Megally.;Åsa Hellqvist.;Vaishali S Mankad.;Lila Bahadori.;Sandhia S Ponnarambil.; .
来源: N Engl J Med. 2025年392卷12期1178-1188页
Treatment with tezepelumab has been effective for sinonasal symptoms in patients with severe, uncontrolled asthma and a history of chronic rhinosinusitis with nasal polyps, but its efficacy and safety in adults with severe, uncontrolled chronic rhinosinusitis with nasal polyps is unknown.
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