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1. 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.
2. 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.
3. 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.
4. 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.
5. 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.
6. 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.).
7. 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.
8. 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.
9. 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.
10. 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.
11. 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.
12. Graft-versus-Host Disease Prophylaxis with Cyclophosphamide and Cyclosporin.
作者: David J Curtis.;Sushrut S Patil.;John Reynolds.;Duncan Purtill.;Clinton Lewis.;David S Ritchie.;David J Gottlieb.;David T Yeung.;Eric Wong.;Siok-Keen Tey.;Travis Perera.;John Moore.;Rachel M Koldej.;Richard De Abreu Lourenco.;John Stubbs.;C Orla Morrissey.;Nadia Munsef.;Andrea Arenas.;Geoffrey R Hill.; .
来源: N Engl J Med. 2025年393卷3期243-254页
Allogeneic peripheral-blood stem-cell transplantation (SCT) from a matched related donor after myeloablative conditioning is the preferred curative treatment for patients with high-risk blood cancers. The combination of a calcineurin inhibitor and an antimetabolite remains standard care for graft-versus-host disease (GVHD) prophylaxis in these patients. Data from two randomized trials have suggested that post-transplantation cyclophosphamide can reduce the risk of GVHD after SCT from a matched donor when it is added to or replaces the antimetabolite. However, the effects of post-transplantation cyclophosphamide specifically after SCT from a matched related donor remain uncertain, and effects in the context of myeloablative conditioning are unclear.
13. A Crossover Trial of Hospital-Wide Lactated Ringer's Solution versus Normal Saline.
作者: Lauralyn McIntyre.;Dean Fergusson.;Tracy McArdle.;Shane English.;Deborah J Cook.;Alison E Fox-Robichaud.;Claudio Martin.;John Marshall.;Michael Pugliese.;Kusum Menon.;Kednapa Thavorn.;Ian D Graham.;Steven Hawken.;Akshai Iyengar.;Kwadwo Kyeremanteng.;Raphael Saginur.;Andrew J E Seely.;Ian G Stiell.;Daniel Bainbridge.;Charles Weijer.;Monica Taljaard.; .
来源: N Engl J Med. 2025年393卷7期660-670页
Whether lactated Ringer's solution is clinically superior to normal saline for routine intravenous administration of fluids is uncertain.
14. A Randomized Trial of Acute Normovolemic Hemodilution in Cardiac Surgery.
作者: Fabrizio Monaco.;Chong Lei.;Matteo Aldo Bonizzoni.;Sergey Efremov.;Federica Morselli.;Fabio Guarracino.;Giuseppe Giardina.;Cristina Arangino.;Domenico Pontillo.;Michelangelo Vitiello.;Alessandro Belletti.;Valentina Ajello.;Margherita Licheri.;Caetano Nigro Neto.;Gaia Barucco.;Nazar A Bukamal.;Carolina Faustini.;Lorenzo Filippo Mantovani.;Alessandro Oriani.;Cristina Santonocito.;Marta Mucchetti.;Francesco Federici.;Chiara Gerli.;Sabrina Porta.;Anna Mara Scandroglio.;Hui Zhang.;Marina Pieri.;Roman Osinsky.;Stefano Lazzari.;Elizaveta Leonova.;Maria Grazia Calabrò.;Daniele Amitrano.;Stefano Turi.;Paolo Prati.;Stefano Fresilli.;Filippo D'Amico.;Jacopo D'Andria Ursoleo.;Rosa Labanca.;Marilena Marmiere.;Alessandro Pruna.;Tommaso Scquizzato.;Kaan Kırali.;Giacomo Monti.;Maria José Carvalho Carmona.;Kenichi Tanaka.;Valery Likhvantsev.;Lian Kah Ti.;Tiziana Bove.;Gianluca Paternoster.;Karen Singh.;Mustafa Emre Gürcü.;Vladimir Lomivorotov.;Giovanni Landoni.;Rinaldo Bellomo.;Alberto Zangrillo.; .
来源: N Engl J Med. 2025年393卷5期450-460页
Patients undergoing cardiac surgery often receive red-cell transfusions, along with the associated risks and costs. Early intraoperative normovolemic hemodilution (i.e., acute normovolemic hemodilution [ANH]) is a blood-conservation technique that entails autologous blood collection before initiation of cardiopulmonary bypass and reinfusion of the collected blood after bypass weaning. More data are needed on whether ANH reduces the number of patients receiving allogeneic red-cell transfusion.
15. 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.
16. Finerenone with Empagliflozin in Chronic Kidney Disease and Type 2 Diabetes.
作者: Rajiv Agarwal.;Jennifer B Green.;Hiddo J L Heerspink.;Johannes F E Mann.;Janet B McGill.;Amy K Mottl.;Julio Rosenstock.;Peter Rossing.;Muthiah Vaduganathan.;Meike Brinker.;Robert Edfors.;Na Li.;Markus F Scheerer.;Charlie Scott.;Masaomi Nangaku.; .
来源: N Engl J Med. 2025年393卷6期533-543页
Limited evidence exists to support the simultaneous initiation of sodium-glucose cotransporter-2 inhibitors and finerenone, a nonsteroidal mineralocorticoid receptor antagonist, in persons with chronic kidney disease and type 2 diabetes.
17. 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.
18. 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.
19. Measurable Residual Disease-Guided Therapy in Newly Diagnosed Myeloma.
作者: Aurore Perrot.;Jérôme Lambert.;Cyrille Hulin.;Andrea Pieragostini.;Lionel Karlin.;Bertrand Arnulf.;Philippe Rey.;Laurent Garderet.;Margaret Macro.;Martine Escoffre-Barbe.;Julie Gay.;Thomas Chalopin.;Romain Gounot.;Jean-Marc Schiano.;Mohamad Mohty.;Xavier Leleu.;Salomon Manier.;Clara Mariette.;Carine Chaleteix.;Thorsten Braun.;Bernard De Prijck.;Hervé Avet-Loiseau.;Jean-Yves Mary.;Jill Corre.;Philippe Moreau.;Cyrille Touzeau.; .
来源: N Engl J Med. 2025年393卷5期425-437页
Measurable residual disease (MRD) is a major prognostic factor in newly diagnosed multiple myeloma. An assessment of an MRD-guided consolidation strategy in patients who are eligible for autologous stem-cell transplantation (ASCT) may be useful.
20. Tarlatamab in Small-Cell Lung Cancer after Platinum-Based Chemotherapy.
作者: Giannis Mountzios.;Longhua Sun.;Byoung Chul Cho.;Umut Demirci.;Sofia Baka.;Mahmut Gümüş.;Antonio Lugini.;Bo Zhu.;Yan Yu.;Ippokratis Korantzis.;Ji-Youn Han.;Tudor-Eliade Ciuleanu.;Myung-Ju Ahn.;Pedro Rocha.;Julien Mazières.;Sally C M Lau.;Martin Schuler.;Fiona Blackhall.;Tatsuya Yoshida.;Taofeek K Owonikoko.;Luis Paz-Ares.;Tony Jiang.;Ali Hamidi.;Diana Gauto.;Gonzalo Recondo.;Charles M Rudin.; .
来源: N Engl J Med. 2025年393卷4期349-361页
Tarlatamab, a bispecific delta-like ligand 3-directed T-cell engager immunotherapy, received accelerated approval for the treatment of patients with previously treated small-cell lung cancer. Whether tarlatamab is more effective than chemotherapy in the treatment of patients whose small-cell lung cancer has progressed during or after initial platinum-based chemotherapy is not known.
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