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501. 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.
502. 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. 2026年394卷15期1503-1513页
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.
504. 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.
506. 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.).
509. 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.
510. 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.
512. 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 513. 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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