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321. Belantamab Mafodotin, Bortezomib, and Dexamethasone for Multiple Myeloma.
作者: Vania Hungria.;Pawel Robak.;Marek Hus.;Vera Zherebtsova.;Christopher Ward.;P Joy Ho.;Ana Carolina Ribas de Almeida.;Roman Hajek.;Kihyun Kim.;Sebastian Grosicki.;Hanlon Sia.;Adam Bryant.;Marcelo Pitombeira de Lacerda.;Gracia Aparecida Martinez.;Anna Maria Sureda Balarí.;Irwindeep Sandhu.;Claudio Cerchione.;Peter Ganly.;Meletios Dimopoulos.;Chengcheng Fu.;Mamta Garg.;Al-Ola Abdallah.;Albert Oriol.;Moshe E Gatt.;Michele Cavo.;Robert Rifkin.;Tomoaki Fujisaki.;Michał Mielnik.;Nick Pirooz.;Astrid McKeown.;Simon McNamara.;Xiangdong Zhou.;Maureen Nichols.;Eric Lewis.;Rachel Rogers.;Hena Baig.;Lydia Eccersley.;Sumita Roy-Ghanta.;Joanna Opalinska.;María-Victoria Mateos.; .
来源: N Engl J Med. 2024年391卷5期393-407页
Belantamab mafodotin had single-agent activity in patients with relapsed or refractory multiple myeloma, a finding that supports further evaluation of the agent in combination with standard-care therapies.
322. Asciminib in Newly Diagnosed Chronic Myeloid Leukemia.
作者: Andreas Hochhaus.;Jianxiang Wang.;Dong-Wook Kim.;Dennis Dong Hwan Kim.;Jiri Mayer.;Yeow-Tee Goh.;Philipp le Coutre.;Naoto Takahashi.;Inho Kim.;Gabriel Etienne.;David Andorsky.;Ghayas C Issa.;Richard A Larson.;Felice Bombaci.;Shruti Kapoor.;Tracey McCulloch.;Kamel Malek.;Lillian Yau.;Sophie Ifrah.;Matthias Hoch.;Jorge E Cortes.;Timothy P Hughes.; .
来源: N Engl J Med. 2024年391卷10期885-898页
Patients with newly diagnosed chronic myeloid leukemia (CML) need long-term therapy with high efficacy and safety. Asciminib, a BCR::ABL1 inhibitor specifically targeting the ABL myristoyl pocket, may offer better efficacy and safety and fewer side effects than currently available frontline ATP-competitive tyrosine kinase inhibitors (TKIs).
323. Oral Sebetralstat for On-Demand Treatment of Hereditary Angioedema Attacks.
作者: Marc A Riedl.;Henriette Farkas.;Emel Aygören-Pürsün.;Fotis Psarros.;Daniel F Soteres.;Maria Staevska.;Mauro Cancian.;David Hagin.;Daisuke Honda.;Isaac Melamed.;Sinisa Savic.;Marcin Stobiecki.;Paula J Busse.;Eunice Dias de Castro.;Nancy Agmon-Levin.;Richard Gower.;Aharon Kessel.;Marcin Kurowski.;Ramon Lleonart.;Vesna Grivcheva Panovska.;H James Wedner.;Paul K Audhya.;James Hao.;Matthew Iverson.;Michael D Smith.;Christopher M Yea.;William R Lumry.;Andrea Zanichelli.;Jonathan A Bernstein.;Marcus Maurer.;Danny M Cohn.; .
来源: N Engl J Med. 2024年391卷1期32-43页
Approved on-demand treatments for hereditary angioedema attacks need to be administered parenterally, a route of administration that is associated with delays in treatment or withholding of therapy.
324. Efficacy and Safety of Donidalorsen for Hereditary Angioedema.
作者: Marc A Riedl.;Raffi Tachdjian.;William R Lumry.;Timothy Craig.;Gül Karakaya.;Asli Gelincik.;Marcin Stobiecki.;Joshua S Jacobs.;Nihal M Gokmen.;Avner Reshef.;Mark M Gompels.;Michael E Manning.;Laura Bordone.;Kenneth B Newman.;Sabrina Treadwell.;Sophie Wang.;Aaron Yarlas.;Danny M Cohn.; .
来源: N Engl J Med. 2024年391卷1期21-31页
Hereditary angioedema is a rare disorder characterized by episodic, potentially life-threatening swelling caused by kallikrein-kinin dysregulation. Long-term prophylaxis can stabilize this system. Donidalorsen, an antisense oligonucleotide, specifically reduces prekallikrein expression.
325. Zodasiran, an RNAi Therapeutic Targeting ANGPTL3, for Mixed Hyperlipidemia.
作者: Robert S Rosenson.;Daniel Gaudet.;Robert A Hegele.;Christie M Ballantyne.;Stephen J Nicholls.;Kathryn J Lucas.;Javier San Martin.;Rong Zhou.;Ma'an Muhsin.;Ting Chang.;Jennifer Hellawell.;Gerald F Watts.; .
来源: N Engl J Med. 2024年391卷10期913-925页
Angiopoietin-like 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases and hepatic uptake of triglyceride-rich lipoprotein remnants. ANGPTL3 loss-of-function carriers have lower levels of triglycerides, low-density lipoprotein (LDL) cholesterol, high-density lipoprotein (HDL) cholesterol, and non-HDL cholesterol and a lower risk of atherosclerotic cardiovascular disease than noncarriers. Zodasiran is an RNA interference (RNAi) therapy targeting expression of ANGPTL3 in the liver.
326. Plozasiran, an RNA Interference Agent Targeting APOC3, for Mixed Hyperlipidemia.
作者: Christie M Ballantyne.;Szilard Vasas.;Masoud Azizad.;Peter Clifton.;Robert S Rosenson.;Ting Chang.;Stacey Melquist.;Rong Zhou.;Ma'an Mushin.;Nicholas J Leeper.;Jennifer Hellawell.;Daniel Gaudet.
来源: N Engl J Med. 2024年391卷10期899-912页
Persons with mixed hyperlipidemia are at risk for atherosclerotic cardiovascular disease due to an elevated non-high-density lipoprotein (HDL) cholesterol level, which is driven by remnant cholesterol in triglyceride-rich lipoproteins. The metabolism and clearance of triglyceride-rich lipoproteins are down-regulated through apolipoprotein C3 (APOC3)-mediated inhibition of lipoprotein lipase.
327. A Randomized Phase 2 Trial of Felzartamab in Antibody-Mediated Rejection.
作者: Katharina A Mayer.;Eva Schrezenmeier.;Matthias Diebold.;Philip F Halloran.;Martina Schatzl.;Sabine Schranz.;Susanne Haindl.;Silke Kasbohm.;Alexander Kainz.;Farsad Eskandary.;Konstantin Doberer.;Uptal D Patel.;Jaideep S Dudani.;Heinz Regele.;Nicolas Kozakowski.;Johannes Kläger.;Rainer Boxhammer.;Kerstin Amann.;Elisabeth Puchhammer-Stöckl.;Hannes Vietzen.;Julia Beck.;Ekkehard Schütz.;Aylin Akifova.;Christa Firbas.;Houston N Gilbert.;Bilgin Osmanodja.;Fabian Halleck.;Bernd Jilma.;Klemens Budde.;Georg A Böhmig.
来源: N Engl J Med. 2024年391卷2期122-132页
Antibody-mediated rejection is a leading cause of kidney-transplant failure. The targeting of CD38 to inhibit graft injury caused by alloantibodies and natural killer (NK) cells may be a therapeutic option.
328. Effects of Semaglutide on Chronic Kidney Disease in Patients with Type 2 Diabetes.
作者: Vlado Perkovic.;Katherine R Tuttle.;Peter Rossing.;Kenneth W Mahaffey.;Johannes F E Mann.;George Bakris.;Florian M M Baeres.;Thomas Idorn.;Heidrun Bosch-Traberg.;Nanna Leonora Lausvig.;Richard Pratley.; .
来源: N Engl J Med. 2024年391卷2期109-121页
Patients with type 2 diabetes and chronic kidney disease are at high risk for kidney failure, cardiovascular events, and death. Whether treatment with semaglutide would mitigate these risks is unknown.
329. Dupilumab for COPD with Blood Eosinophil Evidence of Type 2 Inflammation.
作者: Surya P Bhatt.;Klaus F Rabe.;Nicola A Hanania.;Claus F Vogelmeier.;Mona Bafadhel.;Stephanie A Christenson.;Alberto Papi.;Dave Singh.;Elizabeth Laws.;Naimish Patel.;George D Yancopoulos.;Bolanle Akinlade.;Jennifer Maloney.;Xin Lu.;Deborah Bauer.;Ashish Bansal.;Raolat M Abdulai.;Lacey B Robinson.; .
来源: N Engl J Med. 2024年390卷24期2274-2283页
Dupilumab, a fully human monoclonal antibody that blocks the shared receptor component for interleukin-4 and interleukin-13, key and central drivers of type 2 inflammation, has shown efficacy and safety in a phase 3 trial involving patients with chronic obstructive pulmonary disease (COPD) and type 2 inflammation and an elevated risk of exacerbation. Whether the findings would be confirmed in a second phase 3 trial was unclear.
330. Early Diagnosis and Treatment of COPD and Asthma - A Randomized, Controlled Trial.
作者: Shawn D Aaron.;Katherine L Vandemheen.;G Alex Whitmore.;Celine Bergeron.;Louis-Philippe Boulet.;Andréanne Côté.;R Andrew McIvor.;Erika Penz.;Stephen K Field.;Catherine Lemière.;Irvin Mayers.;Mohit Bhutani.;Tanweer Azher.;M Diane Lougheed.;Samir Gupta.;Nicole Ezer.;Christopher J Licskai.;Paul Hernandez.;Martha Ainslie.;Gonzalo G Alvarez.;Sunita Mulpuru.; .
来源: N Engl J Med. 2024年390卷22期2061-2073页
Many persons with chronic obstructive pulmonary disease (COPD) or asthma have not received a diagnosis, so their respiratory symptoms remain largely untreated.
331. A Modular Communicative Leadless Pacing-Defibrillator System.
作者: Reinoud E Knops.;Michael S Lloyd.;Paul R Roberts.;David J Wright.;Lucas V A Boersma.;Rahul Doshi.;Paul A Friedman.;Petr Neuzil.;Carina Blomström-Lundqvist.;Maria Grazia Bongiorni.;Martin C Burke.;Daniel Gras.;Steven P Kutalek.;Anish K Amin.;Eugene Y Fu.;Laurence M Epstein.;Jose Maria Tolosana.;Thomas D Callahan.;Johan D Aasbo.;Ralph Augostini.;Harish Manyam.;Devi G Nair.;Blandine Mondésert.;Wilber W Su.;Chris Pepper.;Marc A Miller.;Jon Grammes.;Karim Saleh.;Christelle Marquie.;Faisal M Merchant.;Yong-Mei Cha.;Colin Cunnington.;David S Frankel.;Julie West.;Elizabeth Matznick.;Bryan Swackhamer.;Amy J Brisben.;Jonathan Weinstock.;Kenneth M Stein.;Vivek Y Reddy.;Lluis Mont.; .
来源: N Engl J Med. 2024年391卷15期1402-1412页
The subcutaneous implantable cardioverter-defibrillator (ICD) is associated with fewer lead-related complications than a transvenous ICD; however, the subcutaneous ICD cannot provide bradycardia and antitachycardia pacing. Whether a modular pacing-defibrillator system comprising a leadless pacemaker in wireless communication with a subcutaneous ICD to provide antitachycardia and bradycardia pacing is safe remains unknown.
332. Intensive Ambulance-Delivered Blood-Pressure Reduction in Hyperacute Stroke.
作者: Gang Li.;Yapeng Lin.;Jie Yang.;Craig S Anderson.;Chen Chen.;Feifeng Liu.;Laurent Billot.;Qiang Li.;Xiaoying Chen.;Xiaoqiu Liu.;Xinwen Ren.;Chunfang Zhang.;Ping Xu.;Lijun Wu.;Feng Wang.;Daijun Qiu.;Mei Jiang.;Yiqian Peng.;Chaohui Li.;Yiyang Huang.;Xiaohui Zhao.;Jiye Liang.;Yao Wang.;Xiangjun Wu.;Xiaoyun Xu.;Guofang Chen.;Dongya Huang.;Yue Zhang.;Lian Zuo.;Guozhao Ma.;Yumei Yang.;Junjie Hao.;Xiahong Xu.;Xinli Xiong.;Yueyu Tang.;Yijia Guo.;Jianping Yu.;Shuping Li.;Song He.;Fengkai Mao.;Quandan Tan.;Song Tan.;Nengwei Yu.;Ruxiang Xu.;Mingwei Sun.;Binghu Li.;Jiang Guo.;Leibo Liu.;Hueiming Liu.;Menglu Ouyang.;Lei Si.;Hisatomi Arima.;Philip M Bath.;Gary A Ford.;Thompson Robinson.;Else Charlotte Sandset.;Jeffrey L Saver.;Nikola Sprigg.;H Bart van der Worp.;Lili Song.; .; .
来源: N Engl J Med. 2024年390卷20期1862-1872页
Treatment of acute stroke, before a distinction can be made between ischemic and hemorrhagic types, is challenging. Whether very early blood-pressure control in the ambulance improves outcomes among patients with undifferentiated acute stroke is uncertain.
333. Perioperative Nivolumab in Resectable Lung Cancer.
作者: Tina Cascone.;Mark M Awad.;Jonathan D Spicer.;Jie He.;Shun Lu.;Boris Sepesi.;Fumihiro Tanaka.;Janis M Taube.;Robin Cornelissen.;Libor Havel.;Nina Karaseva.;Jaroslaw Kuzdzal.;Lubos B Petruzelka.;Lin Wu.;Jean-Louis Pujol.;Hiroyuki Ito.;Tudor-Eliade Ciuleanu.;Ludmila de Oliveira Muniz Koch.;Annelies Janssens.;Aurelia Alexandru.;Sabine Bohnet.;Fedor V Moiseyenko.;Yang Gao.;Yasutaka Watanabe.;Cinthya Coronado Erdmann.;Padma Sathyanarayana.;Stephanie Meadows-Shropshire.;Steven I Blum.;Mariano Provencio Pulla.; .
来源: N Engl J Med. 2024年390卷19期1756-1769页
Standard treatment with neoadjuvant nivolumab plus chemotherapy significantly improves outcomes in patients with resectable non-small-cell lung cancer (NSCLC). Perioperative treatment (i.e., neoadjuvant therapy followed by surgery and adjuvant therapy) with nivolumab may further improve clinical outcomes.
334. Andexanet for Factor Xa Inhibitor-Associated Acute Intracerebral Hemorrhage.
作者: Stuart J Connolly.;Mukul Sharma.;Alexander T Cohen.;Andrew M Demchuk.;Anna Członkowska.;Arne G Lindgren.;Carlos A Molina.;Daniel Bereczki.;Danilo Toni.;David J Seiffge.;David Tanne.;Else Charlotte Sandset.;Georgios Tsivgoulis.;Hanne Christensen.;Jan Beyer-Westendorf.;Jonathan M Coutinho.;Mark Crowther.;Peter Verhamme.;Pierre Amarenco.;Risto O Roine.;Robert Mikulik.;Robin Lemmens.;Roland Veltkamp.;Saskia Middeldorp.;Thompson G Robinson.;Truman John Milling.;Vitor Tedim-Cruz.;Wilfried Lang.;Anders Himmelmann.;Per Ladenvall.;Mikael Knutsson.;Ella Ekholm.;Andrew Law.;Amanda Taylor.;Tetyana Karyakina.;Lizhen Xu.;Kate Tsiplova.;Sven Poli.;Bernd Kallmünzer.;Christoph Gumbinger.;Ashkan Shoamanesh.; .
来源: N Engl J Med. 2024年390卷19期1745-1755页
Patients with acute intracerebral hemorrhage who are receiving factor Xa inhibitors have a risk of hematoma expansion. The effect of andexanet alfa, an agent that reverses the effects of factor Xa inhibitors, on hematoma volume expansion has not been well studied.
335. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy.
作者: Martin S Maron.;Ahmad Masri.;Michael E Nassif.;Roberto Barriales-Villa.;Michael Arad.;Nuno Cardim.;Lubna Choudhury.;Brian Claggett.;Caroline J Coats.;Hans-Dirk Düngen.;Pablo Garcia-Pavia.;Albert A Hagège.;James L Januzzi.;Matthew M Y Lee.;Gregory D Lewis.;Chang-Sheng Ma.;Michelle Michels.;Iacopo Olivotto.;Artur Oreziak.;Anjali T Owens.;John A Spertus.;Scott D Solomon.;Jacob Tfelt-Hansen.;Marion van Sinttruije.;Josef Veselka.;Hugh Watkins.;Daniel L Jacoby.;Stephen B Heitner.;Stuart Kupfer.;Fady I Malik.;Lisa Meng.;Amy Wohltman.;Theodore P Abraham.; .
来源: N Engl J Med. 2024年390卷20期1849-1861页
One of the major determinants of exercise intolerance and limiting symptoms among patients with obstructive hypertrophic cardiomyopathy (HCM) is an elevated intracardiac pressure resulting from left ventricular outflow tract obstruction. Aficamten is an oral selective cardiac myosin inhibitor that reduces left ventricular outflow tract gradients by mitigating cardiac hypercontractility.
336. Trial of Thrombectomy for Stroke with a Large Infarct of Unrestricted Size.
作者: Vincent Costalat.;Tudor G Jovin.;J F Albucher.;Christophe Cognard.;Hilde Henon.;Nasreddine Nouri.;Benjamin Gory.;Sebastien Richard.;Gaultier Marnat.;Igor Sibon.;Federico Di Maria.;Mariam Annan.;Grégoire Boulouis.;Pere Cardona.;Michael Obadia.;Michel Piotin.;Romain Bourcier.;Benoit Guillon.;Sophie Godard.;Anne Pasco-Papon.;Omer F Eker.;Tae-Hee Cho.;Guillaume Turc.;Olivier Naggara.;Stéphane Velasco.;Matthias Lamy.;Frédéric Clarençon.;Sonia Alamowitch.;Arturo Renu.;Laurent Suissa.;Hervé Brunel.;Jean-Christophe Gentric.;Serge Timsit.;Chantal Lamy.;Cyril Chivot.;Francisco Macian-Montoro.;Charbel Mounayer.;Ozlem Ozkul-Wermester.;Chrysanthi Papagiannaki.;Valérie Wolff.;Raoul Pop.;Anna Ferrier.;Emmanuel Chabert.;Frédéric Ricolfi.;Yannick Béjot.;Elena Lopez-Cancio.;Pedro Vega.;Laurent Spelle.;Christian Denier.;Mònica Millán.;Juan F Arenillas.;Mikael Mazighi.;Emmanuel Houdart.;Maria Del Mar Freijo.;Alain Duhamel.;Nerses Sanossian.;David S Liebeskind.;Julien Labreuche.;Bertrand Lapergue.;Caroline Arquizan.; .
来源: N Engl J Med. 2024年390卷18期1677-1689页
The use of thrombectomy in patients with acute stroke and a large infarct of unrestricted size has not been well studied.
337. Gene Editing for CEP290-Associated Retinal Degeneration.
作者: Eric A Pierce.;Tomas S Aleman.;Kanishka T Jayasundera.;Bright S Ashimatey.;Keunpyo Kim.;Alia Rashid.;Michael C Jaskolka.;Rene L Myers.;Byron L Lam.;Steven T Bailey.;Jason I Comander.;Andreas K Lauer.;Albert M Maguire.;Mark E Pennesi.
来源: N Engl J Med. 2024年390卷21期1972-1984页
CEP290-associated inherited retinal degeneration causes severe early-onset vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) gene-editing complex designed to treat inherited retinal degeneration caused by a specific damaging variant in intron 26 of CEP290 (IVS26 variant).
338. Video versus Direct Laryngoscopy for Urgent Intubation of Newborn Infants.
作者: Lucy E Geraghty.;Emma A Dunne.;Caitríona M Ní Chathasaigh.;Akke Vellinga.;Niamh C Adams.;Eoin M O'Currain.;Lisa K McCarthy.;Colm P F O'Donnell.
来源: N Engl J Med. 2024年390卷20期1885-1894页
Repeated attempts at endotracheal intubation are associated with increased adverse events in neonates. When clinicians view the airway directly with a laryngoscope, fewer than half of first attempts are successful. The use of a video laryngoscope, which has a camera at the tip of the blade that displays a view of the airway on a screen, has been associated with a greater percentage of successful intubations on the first attempt than the use of direct laryngoscopy in adults and children. The effect of video laryngoscopy among neonates is uncertain.
339. Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.
作者: Marie Scully.;Ana Antun.;Spero R Cataland.;Paul Coppo.;Claire Dossier.;Nathalie Biebuyck.;Wolf-Achim Hassenpflug.;Karim Kentouche.;Paul Knöbl.;Johanna A Kremer Hovinga.;M Fernanda López-Fernández.;Masanori Matsumoto.;Thomas L Ortel.;Jerzy Windyga.;Indranil Bhattacharya.;Michael Cronin.;Hong Li.;Björn Mellgård.;Munjal Patel.;Parth Patwari.;Shan Xiao.;Pinghai Zhang.;Linda T Wang.; .
来源: N Engl J Med. 2024年390卷17期1584-1596页
Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known.
340. Subcutaneous Administration of a Monoclonal Antibody to Prevent Malaria.
作者: Kassoum Kayentao.;Aissata Ongoiba.;Anne C Preston.;Sara A Healy.;Zonghui Hu.;Jeff Skinner.;Safiatou Doumbo.;Jing Wang.;Hamidou Cisse.;Didier Doumtabe.;Abdrahamane Traore.;Hamadi Traore.;Adama Djiguiba.;Shanping Li.;Mary E Peterson.;Shinyi Telscher.;Azza H Idris.;William C Adams.;Adrian B McDermott.;Sandeep Narpala.;Bob C Lin.;Leonid Serebryannyy.;Somia P Hickman.;Andrew J McDougal.;Sandra Vazquez.;Matthew Reiber.;Judy A Stein.;Jason G Gall.;Kevin Carlton.;Philipp Schwabl.;Siriman Traore.;Mamadou Keita.;Amatigué Zéguimé.;Adama Ouattara.;M'Bouye Doucoure.;Amagana Dolo.;Sean C Murphy.;Daniel E Neafsey.;Silvia Portugal.;Abdoulaye Djimdé.;Boubacar Traore.;Robert A Seder.;Peter D Crompton.; .
来源: N Engl J Med. 2024年390卷17期1549-1559页
Subcutaneous administration of the monoclonal antibody L9LS protected adults against controlled Plasmodium falciparum infection in a phase 1 trial. Whether a monoclonal antibody administered subcutaneously can protect children from P. falciparum infection in a region where this organism is endemic is unclear.
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