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126. Intravenous Tenecteplase before Thrombectomy in Stroke.
作者: Zhongming Qiu.;Fengli Li.;Hongfei Sang.;Guangxiong Yuan.;Dongjing Xie.;Kai Zhou.;Maohua Li.;Zhaoyou Meng.;Zhenyu Kong.;Zhongfan Ruan.;Chaoqun Li.;Guangui Yang.;Junxiong Wu.;Chen Long.;Bo Yang.;Hai Hu.;Yanling Li.;Jun Luo.;Zhonghua Shi.;Xianjun Huang.;Shunfu Jiang.;Tingyu Yi.;Guoyong Zeng.;Jincheng Liu.;Xiaojun Luo.;Shudong Liu.;Mingze Chang.;Youlin Wu.;Yufeng Tang.;Zhenxuan Tian.;Zhizhong Yan.;Haojin Zhao.;Yuqi Peng.;Hongguo Dai.;Peiyang Zhou.;Huagang Li.;Wenhua Liu.;Dengwen Song.;Bo Lei.;Zhongbin Xia.;Xiaolin Tan.;Maojun Zhao.;Xinggang Feng.;Lingyu Cai.;Qi Li.;Yuelu Wu.;Bingwu Jiang.;Yan Tian.;Linyu Li.;Ling Jiang.;Xingmin Long.;Feng You.;Jian Tao.;Jin Zhou.;Derong Wu.;Chong Zheng.;Congguo Yin.;Duolao Wang.;Mengjie Lu.;Gregory W Albers.;Raul G Nogueira.;Bruce C V Campbell.;Thanh N Nguyen.;Jeffrey L Saver.;Wenjie Zi.;Qingwu Yang.; .
来源: N Engl J Med. 2025年
The safety and efficacy of treatment with intravenous tenecteplase before endovascular thrombectomy in patients with acute ischemic stroke due to large-vessel occlusion remain uncertain.
128. As-Needed Albuterol-Budesonide in Mild Asthma.
作者: Craig LaForce.;Frank Albers.;Anna Danilewicz.;Allison Jeynes-Ellis.;Monica Kraft.;Reynold A Panettieri.;Robert Rees.;Samuel Bardsley.;Lynn Dunsire.;Tim Harrison.;Olami Sobande.;Raulin Surujbally.;Frank Trudo.;Christy Cappelletti.;Alberto Papi.;Richard Beasley.;Bradley E Chipps.;Elliot Israel.;Hitesh Pandya.;Martin Clancy.;Leonard B Bacharier.; .
来源: N Engl J Med. 2025年
As-needed use of albuterol-budesonide has been shown to result in a significantly lower risk of severe asthma exacerbation than as-needed use of albuterol alone among patients with moderate-to-severe asthma. Data on albuterol-budesonide in mild asthma are needed.
129. 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.
131. 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.
134. 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.
135. First-Line Treatment of Pulmonary Sarcoidosis with Prednisone or Methotrexate.
作者: Vivienne Kahlmann.;Montse Janssen Bonás.;Catharina C Moor.;Jan C Grutters.;Rémy L M Mostard.;Henricus N A J van Rijswijk.;Jan van der Maten.;Emiel R Marges.;Linda A A Moonen.;Maria J Overbeek.;Bart Koopman.;Daan W Loth.;Esther J Nossent.;Michiel Wagenaar.;Henk Kramer.;Pascal L M L Wielders.;Peter I Bonta.;Stefan Walen.;Brigitte A H A Bogaarts.;Réne Kerstens.;Mayka Overgaauw.;Marcel Veltkamp.;Marlies S Wijsenbeek.
来源: N Engl J Med. 2025年
Prednisone is currently recommended as the first-line treatment for pulmonary sarcoidosis but is associated with many side effects. Methotrexate, which is recommended as a second-line treatment, appears to have fewer side effects than prednisone but a slower onset of action. Data are needed on the efficacy and side-effect profile of methotrexate as compared with prednisone as first-line treatment for pulmonary sarcoidosis.
136. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
作者: Kiran Musunuru.;Sarah A Grandinette.;Xiao Wang.;Taylor R Hudson.;Kevin Briseno.;Anne Marie Berry.;Julia L Hacker.;Alvin Hsu.;Rachel A Silverstein.;Logan T Hille.;Aysel N Ogul.;Nancy A Robinson-Garvin.;Juliana C Small.;Sarah McCague.;Samantha M Burke.;Christina M Wright.;Sarah Bick.;Venkata Indurthi.;Shweta Sharma.;Michael Jepperson.;Christopher A Vakulskas.;Michael Collingwood.;Katie Keogh.;Ashley Jacobi.;Morgan Sturgeon.;Christian Brommel.;Ellen Schmaljohn.;Gavin Kurgan.;Thomas Osborne.;He Zhang.;Kyle Kinney.;Garrett Rettig.;Christopher J Barbosa.;Sean C Semple.;Ying K Tam.;Cathleen Lutz.;Lindsey A George.;Benjamin P Kleinstiver.;David R Liu.;Kim Ng.;Sadik H Kassim.;Petros Giannikopoulos.;Mohamad-Gabriel Alameh.;Fyodor D Urnov.;Rebecca C Ahrens-Nicklas.
来源: N Engl J Med. 2025年392卷22期2235-2243页
Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality in early infancy, we immediately began to develop a customized lipid nanoparticle-delivered base-editing therapy. After regulatory approval had been obtained for the therapy, the patient received two infusions at approximately 7 and 8 months of age. In the 7 weeks after the initial infusion, the patient was able to receive an increased amount of dietary protein and a reduced dose of a nitrogen-scavenger medication to half the starting dose, without unacceptable adverse events and despite viral illnesses. No serious adverse events occurred. Longer follow-up is warranted to assess safety and efficacy. (Funded by the National Institutes of Health and others.).
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