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461. Trastuzumab Deruxtecan in Residual HER2-Positive Early Breast Cancer.
作者: Sibylle Loibl.;Yeon Hee Park.;Zhiming Shao.;Chiun-Sheng Huang.;Carlos Barrios.;Jame Abraham.;Aleix Prat.;Naoki Niikura.;Seock-Ah Im.;Wei Li.;Huiping Li.;Yongsheng Wang.;Herui Yao.;Sung-Bae Kim.;Cui-Zhi Geng.;Wuilbert Rodriguez Pantigoso.;Francisco Javier Ramírez Godinez.;Chuangui Song.;Yuan Ching Chang.;Augusto Antoniazzi.;Shin-Cheh Chen.;Zhigao Li.;Zbigniew Nowecki.;Joline Lim.;Elton Mathias.;Yuta Sato.;Wenjing Lu.;Hanan Abdel-Monem.;Michael Untch.;Charles E Geyer.; .
来源: N Engl J Med. 2026年394卷9期845-857页
Patients with human epidermal growth factor receptor 2 (HER2)-positive early breast cancer and residual disease after neoadjuvant therapy are at high risk for recurrence.
462. Ketamine or Etomidate for Tracheal Intubation of Critically Ill Adults.
作者: Jonathan D Casey.;Kevin P Seitz.;Brian E Driver.;Kevin W Gibbs.;Adit A Ginde.;Stacy A Trent.;Derek W Russell.;Amelia L Muhs.;Matthew E Prekker.;John P Gaillard.;Daniel Resnick-Ault.;L Jane Stewart.;Micah R Whitson.;Stephanie C DeMasi.;Aaron E Robinson.;Jessica A Palakshappa.;Neil R Aggarwal.;Jason C Brainard.;David J Douin.;Tanya K Marvi.;Benjamin K Scott.;Sarah M Alber.;Carolynn Lyle.;Sheetal Gandotra.;Graham W Van Schaik.;Aaron J Lacy.;Kristen C Sherlin.;Heidi L Erickson.;J Maycee Cain.;Brianne Redman.;Logan L Beach.;Barbara Gould.;Jasmine McIntosh.;Ariel A Lewis.;Bradley D Lloyd.;Tiffany L Israel.;Brant Imhoff.;Li Wang.;Alexandra B Spicer.;Matthew M Churpek.;Todd W Rice.;Wesley H Self.;Jin H Han.;Matthew W Semler.; .
来源: N Engl J Med. 2025年
For critically ill adults undergoing tracheal intubation, observational studies suggest that the use of etomidate to induce anesthesia may increase the risk of death. Whether the use of ketamine rather than etomidate decreases the risk of death is uncertain.
463. Universal Base-Edited CAR7 T Cells for T-Cell Acute Lymphoblastic Leukemia.
作者: Robert Chiesa.;Christos Georgiadis.;Hebatalla Rashed.;Roland Preece.;Prudence Hardefeldt.;Jan Chu.;Jemma Selvage.;Avijeet Mishra.;Batoul Ahmed.;Stuart Adams.;Rebecca Thomas.;Kimberly Gilmour.;Annie Etuk.;Deborah Yallop.;David O'Connor.;Waseem Qasim.; .
来源: N Engl J Med. 2026年394卷2期152-165页
CD7 is an attractive target for chimeric antigen receptor (CAR) T-cell therapy in relapsed or refractory T-cell acute lymphoblastic leukemia (ALL). Supportive results of first-in-human studies of base-edited anti-CD7 CAR (BE-CAR7) T cells with triple C→T deamination-mediated knockouts of TCRαβ, CD52, and CD7 have been reported previously.
464. Teclistamab plus Daratumumab in Relapsed or Refractory Multiple Myeloma.
作者: Luciano J Costa.;Nizar J Bahlis.;Aurore Perrot.;Ajay K Nooka.;Jin Lu.;Charlotte Pawlyn.;Roberto Mina.;Gaston Caeiro.;Alain Kentos.;Vania Hungria.;Donna Reece.;Ting Niu.;Anne K Mylin.;Charlotte T Hansen.;Raphael Teipel.;Britta Besemer.;Meletios A Dimopoulos.;Elena Zamagni.;Satoshi Yoshihara.;Kihyun Kim.;Chang Ki Min.;Paul Geerts.;Elena Van Leeuwen-Segarceanu.;Agata Tyczynska.;Juan Luis Reguera.;Magnus Johansson.;Markus Hansson.;Mehmet Turgut.;Mark Grey.;Surbhi Sidana.;Paula Rodriguez-Otero.;Joaquin Martinez-Lopez.;Hamza Hashmi.;Robin Carson.;Rachel Kobos.;Weili Sun.;Kristen Lantz.;Anne Seifert.;Deborah Briseno-Toomey.;Lisa O'Rourke.;Maria Rubin.;Diego Vieyra.;Lijuan Kang.;Maria Victoria Mateos.; .
来源: N Engl J Med. 2026年394卷8期739-752页
In a phase 1-2 trial, teclistamab, a bispecific antibody targeting CD3 on T-cell surfaces and B-cell maturation antigen on myeloma cells, showed durable responses in heavily pretreated patients with relapsed or refractory multiple myeloma. Daratumumab, a monoclonal antibody targeting CD38 protein, has shown survival benefit in patients with multiple myeloma.
465. Ianalumab plus Eltrombopag in Immune Thrombocytopenia.
作者: Adam Cuker.;Thomas Stauch.;Nichola Cooper.;Hanny Al-Samkari.;Marc Michel.;Waleed Ghanima.;Patrick Urban.;Justyna Fronczek.;Matthew Foster.;Marine Weill.;Lei Zhang.;Ming Hou.;Thomas Zander.;Azizan Sharif.;Jing Sun.;Uttam Kumar Nath.;Roger Schutgens.;Elena Rossi.;Lien Deleu.;Libor Červinek.;Jae-Ho Yoon.;Hung Chang.;Theera Ruchutrakool.;Masaki Iino.;Tatsunori Goto.;Francesco Zaja.; .
来源: N Engl J Med. 2025年
Current second-line treatments for immune thrombocytopenia (ITP) require long-term administration. Ianalumab, a monoclonal antibody targeting B cells, is being assessed as a short-course second-line therapy in ITP.
467. Fixed-Duration versus Continuous Treatment for Chronic Lymphocytic Leukemia.
作者: Othman Al-Sawaf.;Janina Stumpf.;Can Zhang.;Florian Simon.;Francesc Bosch.;Emadoldin Feyzi.;Paolo Ghia.;Michael Gregor.;Arnon P Kater.;Vesa Lindström.;Mattias Mattsson.;Carsten U Niemann.;Philipp B Staber.;Tamar Tadmor.;Patrick Thornton.;Clemens-Martin Wendtner.;Ann Janssens.;Thomas Noesslinger.;Jan-Paul Bohn.;Caspar da Cunha-Bang.;Christian B Poulsen.;Juha Ranti.;Thomas Illmer.;Bjoern Schoettker.;Sebastian Böttcher.;Tobias Gaska.;Elisabeth Vandenberghe.;Ruth Clifford.;Ohad Benjamini.;Anna Maria Frustaci.;Lydia Scarfò.;Paolo Sportoletti.;John Schreurs.;Mark-David Levin.;Hanneke van der Straaten.;Marjolein van der Klift.;Hoa Tran.;Javier de la Serna.;Javier Loscertales.;Oscar Lindblad.;Anna Bergendahl Sandstedt.;Jeroen Goede.;Michael Baumann.;Anna Maria Fink.;Kirsten Fischer.;Matthias Ritgen.;Karl-Anton Kreuzer.;Christof Schneider.;Eugen Tausch.;Stephan Stilgenbauer.;Sandra Robrecht.;Barbara Eichhorst.;Michael Hallek.; .
来源: N Engl J Med. 2026年394卷11期1084-1096页
Treatment of chronic lymphocytic leukemia (CLL) currently consists of two main approaches - continuous therapy with Bruton's tyrosine kinase inhibitors and fixed-duration regimens combining venetoclax with either CD20 antibodies or Bruton's tyrosine kinase inhibitors. Comparisons of these two therapeutic approaches are lacking.
469. Prime Editing for p47phox-Deficient Chronic Granulomatous Disease.
作者: Jennifer L Gori.;Elie Haddad.;Haydar Frangoul.;Donald B Kohn.;Emma C Morris.;Bradley N Martin.;Briana A Deary.;McKinley Nickerson.;Rebecca L Scholz.;Isabel Fernandez.;Karine Leveille.;Suk See De Ravin.;Elizabeth M Kang.;Marie Pierzynski.;Tyra Estwick.;Patricia Littel.;Douglas B Kuhns.;Debra A Long Priel.;Pierre Teira.;Stuart Turvey.;Jordyn Arnold.;Misty D Evans.;Meghann McManus.;Ben Carpenter.;David P Waterman.;Andrew V Anzalone.;Alexandria Petrusich.;Christa E Osuna.;Tiernan T O'Malley.;Jacob Stewart-Ornstein.;Jack M Heath.;Barrett J Nehilla.;Mohammed Asmal.;Harry L Malech.
来源: N Engl J Med. 2026年394卷12期1195-1203页
Chronic granulomatous disease (CGD) is a severe monogenic immunodeficiency caused by damaging variants in genes required for microbicidal NADPH oxidase activity. Autosomal recessive p47phox-deficient CGD (p47-CGD) is predominantly caused by a two-nucleotide deletion in exon 2 (delGT) of NCF1. We developed PM359, an autologous CD34+ hematopoietic stem-cell therapy in which prime editing is used to correct delGT. Two participants received PM359 after myeloid conditioning with busulfan: neutrophils and platelets engrafted promptly in both patients. Adverse events were consistent with myeloid conditioning with busulfan. NADPH oxidase activity was observed in neutrophils within 1 month and was maintained for 6 months and 4 months as of the last follow-up visit in Participants 1 and 2, respectively. These results support further investigation of prime editing of CD34+ cells to treat p47-CGD. (Funded by Prime Medicine; ClinicalTrials.gov number, NCT06559176.).
472. Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.
作者: Steven W Pipe.;Wolfgang Miesbach.;Michael Recht.;Frank W G Leebeek.;Nigel S Key.;Giancarlo Castaman.;Susan Lattimore.;Michiel Coppens.;Sandra Le Quellec.;Vaibhav Mahajan.;Sean Gill.;Douglass Drelich.;Paul E Monahan.; .
来源: N Engl J Med. 2026年394卷5期463-474页
Prophylactic treatment for hemophilia B necessitates lifelong, regular intravenous factor IX infusions. Gene therapy offers the possibility of a single-dose treatment that produces durable endogenous factor IX expression and disease control. Etranacogene dezaparvovec comprises an adeno-associated virus serotype 5 (AAV5) vector and the highly active Padua factor IX variant. The primary analysis of this study showed that etranacogene dezaparvovec reduced annualized bleeding rates and adverse events were mainly of low-grade severity. Final data from 5 years of follow-up are now available.
473. Dual Targeting of Extramedullary Myeloma with Talquetamab and Teclistamab.
作者: Shaji Kumar.;María-Victoria Mateos.;Jing Christine Ye.;Shebli Atrash.;Hila Magen.;Hang Quach.;Michael P Chu.;Suzanne Trudel.;Joshua Richter.;Paula Rodríguez-Otero.;Hun Chuah.;Moshe Gatt.;Eva Medvedova.;Shahzad Raza.;Dok Hyun Yoon.;Tadao Ishida.;Jeffrey V Matous.;Laura Rosiñol.;Koichi Onodera.;Emma Scott.;Christoph Heuck.;Jenny Zhang.;Todd Henninger.;Lisa O'Rourke.;Payal Thakkar.;Mariacristina Festa.;Lin Huang.;Jiangxiu Zhou.;Mikihiro Takamoto.;Lixia Pei.;Jiashen Lu.;Nicholas Au.;Maria Krevvata.;Saad Z Usmani.;Yael C Cohen.; .
来源: N Engl J Med. 2026年394卷1期51-61页
Patients with plasmacytomas that are noncontiguous with bone marrow (true extramedullary myeloma) are at high risk for disease progression or relapse. Phase 1 of the RedirecTT-1 study showed promising efficacy with dual-antigen targeting of myeloma with talquetamab (anti-G protein-coupled receptor family C group 5 member D) plus teclistamab (anti-B-cell maturation antigen) in patients with triple-class-exposed relapsed or refractory multiple myeloma, including those with true extramedullary myeloma.
475. A Threat to Evidence-Based Vaccine Policy and Public Health Security at the FDA.
作者: Robert M Califf.;Andrew C von Eschenbach.;Michael A Friedman.;Brett P Giroir.;Scott Gottlieb.;Margaret A Hamburg.;Jane E Henney.;David A Kessler.;Mark B McClellan.;Stephen M Ostroff.;Norman E Sharpless.;Janet Woodcock.
来源: N Engl J Med. 2026年394卷1期4-6页 476. Noninferiority of One HPV Vaccine Dose to Two Doses.
作者: Aimée R Kreimer.;Carolina Porras.;Danping Liu.;Allan Hildesheim.;Loretto J Carvajal.;Rebeca Ocampo.;Byron Romero.;Mitchell H Gail.;Bernal Cortes.;Monica S Sierra.;Karla Coronado.;Joshua Sampson.;Carolina Coto.;Casey L Dagnall.;Daniela Mora.;Troy J Kemp.;Michael Zuniga.;Ligia A Pinto.;Gloriana Barrientos.;John Schussler.;Yenory Estrada.;Cristian Montero.;Carlos Avila.;Dave Ruggieri.;Jean T Cyr.;Stephen Chanock.;Douglas R Lowy.;John T Schiller.;Rolando Herrero.
来源: N Engl J Med. 2025年393卷24期2421-2433页
Multidose human papillomavirus (HPV) vaccination is efficacious, yet the vaccine has been underused globally. Emerging data suggest that a single dose may provide protection. Whether a single dose of HPV vaccine would provide similar protection to two doses is uncertain.
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