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241. Atrasentan in Patients with IgA Nephropathy.
作者: Hiddo J L Heerspink.;Meg Jardine.;Donald E Kohan.;Richard A Lafayette.;Adeera Levin.;Adrian Liew.;Hong Zhang.;Amit Lodha.;Todd Gray.;Yi Wang.;Ronny Renfurm.;Jonathan Barratt.; .
来源: N Engl J Med. 2025年392卷6期544-554页
Patients with IgA nephropathy and severe proteinuria have a high lifetime risk of kidney failure. The efficacy and safety of the selective endothelin type A receptor antagonist atrasentan in reducing proteinuria in patients with IgA nephropathy are incompletely understood.
242. Long-Term Effects of Empagliflozin in Patients with Chronic Kidney Disease.
作者: .;William G Herrington.;Natalie Staplin.;Nikita Agrawal.;Christoph Wanner.;Jennifer B Green.;Sibylle J Hauske.;Jonathan R Emberson.;David Preiss.;Parminder Judge.;Doreen Zhu.;Rejive Dayanandan.;Ryoki Arimoto.;Kaitlin J Mayne.;Sarah Y A Ng.;Emily Sammons.;Michael Hill.;Will Stevens.;Karl Wallendszus.;Susanne Brenner.;Alfred K Cheung.;Zhi-Hong Liu.;Jing Li.;Lai Seong Hooi.;Wen Liu.;Takashi Kadowaki.;Masaomi Nangaku.;Adeer Levin.;David Z I Cherney.;Aldo P Maggioni.;Roberto Pontremoli.;Rajat Deo.;Shinya Goto.;Xavier Rossello.;Katherine R Tuttle.;Dominik Steubl.;Dan Massey.;Martina Brueckmann.;Martin J Landray.;Colin Baigent.;Richard Haynes.; .
来源: N Engl J Med. 2025年392卷8期777-787页
In the EMPA-KIDNEY trial, empagliflozin, a sodium-glucose cotransporter 2 (SGLT2) inhibitor, had positive cardiorenal effects in patients with chronic kidney disease who were at risk for disease progression. Post-trial follow-up was designed to assess how the effects of empagliflozin would evolve after the discontinuation of the trial drug.
243. Alternative Complement Pathway Inhibition with Iptacopan in IgA Nephropathy.
作者: Vlado Perkovic.;Jonathan Barratt.;Brad Rovin.;Naoki Kashihara.;Bart Maes.;Hong Zhang.;Hernán Trimarchi.;Dmitrij Kollins.;Olympia Papachristofi.;Severina Jacinto-Sanders.;Tobias Merkel.;Nicolas Guerard.;Ronny Renfurm.;Thomas Hach.;Dana V Rizk.; .
来源: N Engl J Med. 2025年392卷6期531-543页
The alternative complement pathway plays a key role in the pathogenesis of IgA nephropathy. Iptacopan specifically binds to factor B and inhibits the alternative pathway.
244. CRISPR-Based Therapy for Hereditary Angioedema.
作者: Danny M Cohn.;Padmalal Gurugama.;Markus Magerl.;Constance H Katelaris.;David Launay.;Laurence Bouillet.;Remy S Petersen.;Karen Lindsay.;Emel Aygören-Pürsün.;David Maag.;James S Butler.;Mrinal Y Shah.;Adele Golden.;Yuanxin Xu.;Ahmed M Abdelhady.;David Lebwohl.;Hilary J Longhurst.
来源: N Engl J Med. 2025年392卷5期458-467页
Hereditary angioedema is a rare genetic disease characterized by severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy that is based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (KLKB1). A single dose of NTLA-2002 may provide lifelong control of angioedema attacks.
245. Phase 3 Trial of Stereotactic Body Radiotherapy in Localized Prostate Cancer.
作者: Nicholas van As.;Clare Griffin.;Alison Tree.;Jaymini Patel.;Peter Ostler.;Hans van der Voet.;Andrew Loblaw.;William Chu.;Daniel Ford.;Shaun Tolan.;Suneil Jain.;Philip Camilleri.;Kiran Kancherla.;John Frew.;Andrew Chan.;Olivia Naismith.;John Armstrong.;John Staffurth.;Alexander Martin.;Ian Dayes.;Paula Wells.;Derek Price.;Emily Williamson.;Julia Pugh.;Georgina Manning.;Stephanie Brown.;Stephanie Burnett.;Emma Hall.
来源: N Engl J Med. 2024年391卷15期1413-1425页
Whether stereotactic body radiotherapy (SBRT) is noninferior to conventionally or moderately hypofractionated regimens with respect to biochemical or clinical failure in patients with localized prostate cancer is unclear.
246. Nivolumab+AVD in Advanced-Stage Classic Hodgkin's Lymphoma.
作者: Alex F Herrera.;Michael LeBlanc.;Sharon M Castellino.;Hongli Li.;Sarah C Rutherford.;Andrew M Evens.;Kelly Davison.;Angela Punnett.;Susan K Parsons.;Sairah Ahmed.;Carla Casulo.;Nancy L Bartlett.;Joseph M Tuscano.;Matthew G Mei.;Brian T Hess.;Ryan Jacobs.;Hayder Saeed.;Pallawi Torka.;Boyu Hu.;Craig Moskowitz.;Supreet Kaur.;Gaurav Goyal.;Christopher Forlenza.;Andrew Doan.;Adam Lamble.;Pankaj Kumar.;Saeeda Chowdhury.;Brett Brinker.;Namita Sharma.;Avina Singh.;Kristie A Blum.;Anamarija M Perry.;Alexandra E Kovach.;David Hodgson.;Louis S Constine.;Lale Kostakoglu Shields.;Anca Prica.;Hildy Dillon.;Richard F Little.;Margaret A Shipp.;Michael Crump.;Brad Kahl.;John P Leonard.;Sonali M Smith.;Joo Y Song.;Kara M Kelly.;Jonathan W Friedberg.
来源: N Engl J Med. 2024年391卷15期1379-1389页
Incorporating brentuximab vedotin into the treatment of advanced-stage classic Hodgkin's lymphoma improves outcomes in adult and pediatric patients. However, brentuximab vedotin increases the toxic effects of treatment in adults, more than half of pediatric patients who receive the drug undergo consolidative radiation, and relapse remains a challenge. Programmed death 1 blockade is effective in Hodgkin's lymphoma, including in preliminary studies involving previously untreated patients.
247. Radical Prostatectomy or Watchful Waiting in Early Prostate Cancer.
作者: Lars Holmberg.;Hans Garmo.;Swen-Olof Andersson.;Ove Andrén.;Eva Johansson.;Kimmo Taari.;Michael Häggman.;Stig Nordling.;Christer Busch.;Hans-Olov Adami.;Jan-Erik Johansson.;Anna Bill-Axelson.
来源: N Engl J Med. 2024年391卷14期1362-1364页 248. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
作者: Florian Eichler.;Christine N Duncan.;Patricia L Musolino.;Troy C Lund.;Ashish O Gupta.;Satiro De Oliveira.;Adrian J Thrasher.;Patrick Aubourg.;Jörn-Sven Kühl.;Daniel J Loes.;Hernan Amartino.;Nicholas Smith.;Juliana Folloni Fernandes.;Caroline Sevin.;Raman Sankar.;Shaun A Hussain.;Paul Gissen.;Jean-Hugues Dalle.;Uwe Platzbecker.;Gerald F Downey.;Elizabeth McNeil.;Laura Demopoulos.;Andrew C Dietz.;Himal L Thakar.;Paul J Orchard.;David A Williams.
来源: N Engl J Med. 2024年391卷14期1302-1312页
Cerebral adrenoleukodystrophy is a severe form of X-linked adrenoleukodystrophy characterized by white-matter disease, loss of neurologic function, and early death. Elivaldogene autotemcel (eli-cel) gene therapy, which consists of autologous CD34+ cells transduced with Lenti-D lentiviral vector containing ABCD1 complementary DNA, is being tested in persons with cerebral adrenoleukodystrophy.
249. Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
作者: Christine N Duncan.;Jacob R Bledsoe.;Bartosz Grzywacz.;Amy Beckman.;Melissa Bonner.;Florian S Eichler.;Jörn-Sven Kühl.;Marian H Harris.;Sarah Slauson.;Richard A Colvin.;Vinod K Prasad.;Gerald F Downey.;Francis J Pierciey.;Melissa A Kinney.;Marianna Foos.;Ankit Lodaya.;Nicole Floro.;Geoffrey Parsons.;Andrew C Dietz.;Ashish O Gupta.;Paul J Orchard.;Himal L Thakar.;David A Williams.
来源: N Engl J Med. 2024年391卷14期1287-1301页
Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenoleukodystrophy. However, the risk of oncogenesis with eli-cel is unclear.
250. Collagenase Injection versus Limited Fasciectomy for Dupuytren's Contracture.
作者: Joseph Dias.;Puvanendran Tharmanathan.;Catherine Arundel.;Charlie Welch.;Qi Wu.;Paul Leighton.;Maria Armaou.;Nick Johnson.;Sophie James.;John Cooke.;Lionel Bainbridge.;Michael Craigen.;David Warwick.;Samantha Brady.;Lydia G Flett.;Judy Jones.;Catherine N Knowlson.;Michelle Watson.;Ada Keding.;Catherine E Hewitt.;David Torgerson.
来源: N Engl J Med. 2024年391卷16期1499-1510页
Treatments for Dupuytren's contracture include limited fasciectomy and collagenase injection. Comparisons of the effectiveness of these treatments have been limited.
251. Fractional Doses of Pneumococcal Conjugate Vaccine - A Noninferiority Trial.
作者: Katherine E Gallagher.;Ruth Lucinde.;Christian Bottomley.;Mary Kaniu.;Badaud Suaad.;Mary Mutahi.;Laura Mwalekwa.;Sarah Ragab.;Louise Twi-Yeboah.;James A Berkley.;Mainga Hamaluba.;Angela Karani.;Jimmy Shangala.;Mark Otiende.;Elizabeth Gardiner.;Daisy Mugo.;Peter G Smith.;Collins Tabu.;Fred Were.;David Goldblatt.;J Anthony G Scott.
来源: N Engl J Med. 2024年391卷21期2003-2013页
Pneumococcal conjugate vaccines are an expensive component of the routine immunization schedule. Fractional-dose regimens may be one option to increase the sustainability of the vaccine program.
252. Phase 2 Trial of Anti-TL1A Monoclonal Antibody Tulisokibart for Ulcerative Colitis.
作者: Bruce E Sands.;Brian G Feagan.;Laurent Peyrin-Biroulet.;Silvio Danese.;David T Rubin.;Olivier Laurent.;Allison Luo.;Deanna D Nguyen.;Jiandong Lu.;Mark Yen.;Jaroslaw Leszczyszyn.;Radosław Kempiński.;Dermot P B McGovern.;Christopher Ma.;Timothy E Ritter.;Stephan Targan.; .
来源: N Engl J Med. 2024年391卷12期1119-1129页
Tulisokibart is a tumor necrosis factor-like cytokine 1A (TL1A) monoclonal antibody in development for the treatment of moderately to severely active ulcerative colitis. A genetic-based diagnostic test was designed to identify patients with an increased likelihood of response.
253. Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
作者: Adam Cuker.;Kaan Kavakli.;Laurent Frenzel.;Jiaan-Der Wang.;Jan Astermark.;Monica H Cerqueira.;Alfonso Iorio.;Olga Katsarou-Fasouli.;Robert Klamroth.;Amy D Shapiro.;Cédric Hermans.;Akira Ishiguro.;Andrew D Leavitt.;Johannes B Oldenburg.;Margareth C Ozelo.;Jerome Teitel.;Francesca Biondo.;Annie Fang.;Joanne Fuiman.;John McKay.;Pengling Sun.;John E J Rasko.;Jeremy Rupon.; .
来源: N Engl J Med. 2024年391卷12期1108-1118页
Fidanacogene elaparvovec, an adeno-associated virus (AAV) gene-therapy vector for hemophilia B containing a high-activity human factor IX variant (FIX-R338L/FIX-Padua), was associated with sustained factor IX activity in a phase 1-2a study.
254. Ziresovir in Hospitalized Infants with Respiratory Syncytial Virus Infection.
作者: Shunying Zhao.;Yunxiao Shang.;Yong Yin.;Yingxue Zou.;Yongsheng Xu.;Lili Zhong.;Hailin Zhang.;Hua Zhang.;Deyu Zhao.;Tong Shen.;Dongming Huang.;Qiang Chen.;Qiaozhi Yang.;Yungang Yang.;Xiaoyan Dong.;Ling Li.;Zhimin Chen.;Enmei Liu.;Li Deng.;Wenhui Jiang.;Huanji Cheng.;Guangmin Nong.;Xiufang Wang.;Yiping Chen.;Rong Ding.;Wei Zhou.;Yuejie Zheng.;Zhaobo Shen.;Xiaoxia Lu.;Chuangli Hao.;Xiaoping Zhu.;Tan Jia.;Yan Wu.;Gang Zou.;Ki Rito.;Jim Z Wu.;Hanmin Liu.;Xin Ni.; .
来源: N Engl J Med. 2024年391卷12期1096-1107页
Respiratory syncytial virus (RSV) is a leading cause of severe illness in infants, with no effective treatment. Results of a phase 2 trial suggested that ziresovir may have efficacy in the treatment of infants hospitalized with RSV infection.
255. Results after Four Years of Screening for Prostate Cancer with PSA and MRI.
作者: Jonas Hugosson.;Rebecka Arnsrud Godtman.;Jonas Wallstrom.;Ulrika Axcrona.;Anders Bergh.;Lars Egevad.;Kjell Geterud.;Ali Khatami.;Andreas Socratous.;Vasiliki Spyratou.;Linda Svensson.;Johan Stranne.;Marianne Månsson.;Mikael Hellstrom.
来源: N Engl J Med. 2024年391卷12期1083-1095页
Data on the efficacy and safety of screening for prostate cancer with magnetic resonance imaging (MRI) are needed from studies of follow-up screening.
256. Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia.
作者: Hanny Al-Samkari.;Raj S Kasthuri.;Vivek N Iyer.;Allyson M Pishko.;Jake E Decker.;Clifford R Weiss.;Kevin J Whitehead.;Miles B Conrad.;Marc S Zumberg.;Jenny Y Zhou.;Joseph Parambil.;Derek Marsh.;Marianne Clancy.;Lauren Bradley.;Lisa Wisniewski.;Benjamin A Carper.;Sonia M Thomas.;Keith R McCrae.
来源: N Engl J Med. 2024年391卷11期1015-1027页
Hereditary hemorrhagic telangiectasia (HHT) is characterized by extensive telangiectasias and arteriovenous malformations. The primary clinical manifestation is epistaxis that results in iron-deficiency anemia and reduced health-related quality of life.
257. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease.
作者: Daniel Wolff.;Corey Cutler.;Stephanie J Lee.;Iskra Pusic.;Henrique Bittencourt.;Jennifer White.;Mehdi Hamadani.;Sally Arai.;Amandeep Salhotra.;Jose A Perez-Simon.;Amin Alousi.;Hannah Choe.;Mi Kwon.;Arancha Bermúdez.;Inho Kim.;Gerard Socié.;Saurabh Chhabra.;Vedran Radojcic.;Timothy O'Toole.;Chuan Tian.;Peter Ordentlich.;Zachariah DeFilipp.;Carrie L Kitko.; .
来源: N Engl J Med. 2024年391卷11期1002-1014页
Colony-stimulating factor 1 receptor (CSF1R)-dependent monocytes and macrophages are key mediators of chronic graft-versus-host disease (GVHD), a major long-term complication of allogeneic hematopoietic stem-cell transplantation. The CSF1R-blocking antibody axatilimab has shown promising clinical activity in chronic GVHD.
258. Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors.
作者: Jennifer A Chan.;Susan Geyer.;Tyler Zemla.;Michael V Knopp.;Spencer Behr.;Sydney Pulsipher.;Fang-Shu Ou.;Amylou C Dueck.;Jared Acoba.;Ardaman Shergill.;Edward M Wolin.;Thorvardur R Halfdanarson.;Bhavana Konda.;Nikolaos A Trikalinos.;Bernard Tawfik.;Nitya Raj.;Shagufta Shaheen.;Namrata Vijayvergia.;Arvind Dasari.;Jonathan R Strosberg.;Elise C Kohn.;Matthew H Kulke.;Eileen M O'Reilly.;Jeffrey A Meyerhardt.
来源: N Engl J Med. 2025年392卷7期653-665页
Treatment options for patients with advanced neuroendocrine tumors are limited. The efficacy of cabozantinib in the treatment of previously treated, progressive extrapancreatic or pancreatic neuroendocrine tumors is unclear.
259. Perioperative Durvalumab with Neoadjuvant Chemotherapy in Operable Bladder Cancer.
作者: Thomas Powles.;James W F Catto.;Matthew D Galsky.;Hikmat Al-Ahmadie.;Joshua J Meeks.;Hiroyuki Nishiyama.;Toan Quang Vu.;Lorenzo Antonuzzo.;Pawel Wiechno.;Vagif Atduev.;Ariel G Kann.;Tae-Hwan Kim.;Cristina Suárez.;Chao-Hsiang Chang.;Florian Roghmann.;Mustafa Özgüroğlu.;Bernhard J Eigl.;Niara Oliveira.;Tomas Buchler.;Moran Gadot.;Yousef Zakharia.;Jon Armstrong.;Ashok Gupta.;Stephan Hois.;Michiel S van der Heijden.; .
来源: N Engl J Med. 2024年391卷19期1773-1786页
Neoadjuvant chemotherapy followed by radical cystectomy is the standard treatment for cisplatin-eligible patients with muscle-invasive bladder cancer. Adding perioperative immunotherapy may improve outcomes.
260. Ponsegromab for the Treatment of Cancer Cachexia.
作者: John D Groarke.;Jeffrey Crawford.;Susie M Collins.;Shannon Lubaczewski.;Eric J Roeland.;Tateaki Naito.;Andrew E Hendifar.;Marie Fallon.;Koichi Takayama.;Timothy Asmis.;Richard F Dunne.;Isik Karahanoglu.;Carrie A Northcott.;Magdalena A Harrington.;Michelle Rossulek.;Ruolun Qiu.;Aditi R Saxena.
来源: N Engl J Med. 2024年391卷24期2291-2303页
Cachexia is a common complication of cancer and is associated with an increased risk of death. The level of growth differentiation factor 15 (GDF-15), a circulating cytokine, is elevated in cancer cachexia. In a small, open-label, phase 1b study involving patients with cancer cachexia, ponsegromab, a humanized monoclonal antibody inhibiting GDF-15, was associated with improved weight, appetite, and physical activity, along with suppressed serum GDF-15 levels.
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