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3284. Severe Immune-Related Enteritis after In Utero Exposure to Pembrolizumab.
作者: Manuel A Baarslag.;Joosje H Heimovaara.;Jessica S W Borgers.;Koen J van Aerde.;Hans J P M Koenen.;Ruben L Smeets.;Pauline L M Buitelaar.;Dick Pluim.;Shoko Vos.;Stefanie S V Henriet.;Jan Willem B de Groot.;Martine van Grotel.;Hilde Rosing.;Jos H Beijnen.;Alwin D R Huitema.;John B A G Haanen.;Frédéric Amant.;Nicole Gierenz.
来源: N Engl J Med. 2023年389卷19期1790-1796页
Immune checkpoint blockade has become standard treatment for many types of cancer. Such therapy is indicated most often in patients with advanced or metastatic disease but has been increasingly used as adjuvant therapy in those with early-stage disease. Adverse events include immune-related organ inflammation resembling autoimmune diseases. We describe a case of severe immune-related gastroenterocolitis in a 4-month-old infant who presented with intractable diarrhea and failure to thrive after in utero exposure to pembrolizumab. Known causes of the symptoms were ruled out, and the diagnosis of pembrolizumab-induced immune-related gastroenterocolitis was supported by the results of histopathological assays, immunophenotyping, and analysis of the level of antibodies against programmed cell death protein 1 (PD-1). The infant's condition was successfully treated with prednisolone and infliximab.
3285. Trial of Botulinum Toxin for Isolated or Essential Head Tremor.
作者: Ana Marques.;Bruno Pereira.;Marion Simonetta-Moreau.;Giovanni Castelnovo.;Marie De Verdal.;Frédérique Fluchère.;Chloé Laurencin.;Bertrand Degos.;Mélissa Tir.;Alexandre Kreisler.;Geneviève Blanchet-Fourcade.;Dominique Guehl.;Olivier Colin.;Aurelia Poujois.;Sophie Sangla.;Laurent Tatu.;Philippe Derost.;Dominique Gayraud.;Christine Tranchant.;David Amarantini.;David Devos.;Olivier Rascol.;Jean-Christophe Corvol.;Franck Durif.;Isabelle Rieu.; .
来源: N Engl J Med. 2023年389卷19期1753-1765页
Local injections of botulinum toxin type A have been used to treat essential head tremor but have not been extensively studied in randomized trials.
3286. Actionable Genotypes and Their Association with Life Span in Iceland.
作者: Brynjar O Jensson.;Gudny A Arnadottir.;Hildigunnur Katrinardottir.;Run Fridriksdottir.;Hannes Helgason.;Asmundur Oddsson.;Gardar Sveinbjornsson.;Hannes P Eggertsson.;Gisli H Halldorsson.;Bjarni A Atlason.;Hakon Jonsson.;Gudjon R Oskarsson.;Arni Sturluson.;Sigurjon A Gudjonsson.;Gudmundur A Thorisson.;Florian Zink.;Kristjan H S Moore.;Gunnar Palsson.;Asgeir Sigurdsson.;Adalbjorg Jonasdottir.;Aslaug Jonasdottir.;Magnus K Magnusson.;Anna Helgadottir.;Valgerdur Steinthorsdottir.;Julius Gudmundsson.;Simon N Stacey.;Rafn Hilmarsson.;Isleifur Olafsson.;Oskar T Johannsson.;David O Arnar.;Jona Saemundsdottir.;Olafur T Magnusson.;Gisli Masson.;Bjarni V Halldorsson.;Agnar Helgason.;Hreinn Stefansson.;Ingileif Jonsdottir.;Hilma Holm.;Thorunn Rafnar.;Unnur Thorsteinsdottir.;Daniel F Gudbjartsson.;Kari Stefansson.;Patrick Sulem.
来源: N Engl J Med. 2023年389卷19期1741-1752页
In 2021, the American College of Medical Genetics and Genomics (ACMG) recommended reporting actionable genotypes in 73 genes associated with diseases for which preventive or therapeutic measures are available. Evaluations of the association of actionable genotypes in these genes with life span are currently lacking.
3288. Osimertinib with or without Chemotherapy in EGFR-Mutated Advanced NSCLC.
作者: David Planchard.;Pasi A Jänne.;Ying Cheng.;James C-H Yang.;Noriko Yanagitani.;Sang-We Kim.;Shunichi Sugawara.;Yan Yu.;Yun Fan.;Sarayut L Geater.;Konstantin Laktionov.;Chee K Lee.;Natalia Valdiviezo.;Samreen Ahmed.;Jean-Marc Maurel.;Igor Andrasina.;Jonathan Goldman.;Dana Ghiorghiu.;Yuri Rukazenkov.;Alex Todd.;Kunihiko Kobayashi.; .
来源: N Engl J Med. 2023年389卷21期1935-1948页
Osimertinib is a third-generation epidermal growth factor receptor-tyrosine kinase inhibitor (EGFR-TKI) that is selective for EGFR-TKI-sensitizing and EGFR T790M resistance mutations. Evidence suggests that the addition of chemotherapy may extend the benefits of EGFR-TKI therapy.
3293. Sparsentan versus Irbesartan in Focal Segmental Glomerulosclerosis.
作者: Michelle N Rheault.;Charles E Alpers.;Jonathan Barratt.;Stewart Bieler.;Pietro Canetta.;Dong-Wan Chae.;Gaia Coppock.;Ulysses Diva.;Loreto Gesualdo.;Hiddo J L Heerspink.;Jula K Inrig.;Gianna M Kirsztajn.;Donald Kohan.;Radko Komers.;Laura A Kooienga.;Kenneth Lieberman.;Alex Mercer.;Irene L Noronha.;Vlado Perkovic.;Jai Radhakrishnan.;William Rote.;Brad Rovin.;Vladimir Tesar.;Hernán Trimarchi.;James Tumlin.;Muh Geot Wong.;Howard Trachtman.; .
来源: N Engl J Med. 2023年389卷26期2436-2445页
An unmet need exists for focal segmental glomerulosclerosis (FSGS) treatment. In an 8-week, phase 2 trial, sparsentan, a dual endothelin-angiotensin receptor antagonist, reduced proteinuria in patients with FSGS. The efficacy and safety of longer-term treatment with sparsentan for FSGS are unknown.
3294. A Phase 2 Trial of Sibeprenlimab in Patients with IgA Nephropathy.
作者: Mohit Mathur.;Jonathan Barratt.;Bobby Chacko.;Tak Mao Chan.;Laura Kooienga.;Kook-Hwan Oh.;Manisha Sahay.;Yusuke Suzuki.;Muh Geot Wong.;Jill Yarbrough.;Jing Xia.;Brian J G Pereira.; .
来源: N Engl J Med. 2024年390卷1期20-31页
A proliferation-inducing ligand (APRIL) is implicated in the pathogenesis of IgA nephropathy. Sibeprenlimab is a humanized IgG2 monoclonal antibody that binds to and neutralizes APRIL.
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