{"title":"Popeye Sign in Transthyretin Amyloidosis. Reply.","authors":"Satoshi Kurisu, Hitoshi Fujiwara","doi":"10.1056/NEJMc2514537","DOIUrl":"https://doi.org/10.1056/NEJMc2514537","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"394 3","pages":"311"},"PeriodicalIF":78.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145985879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Restoring Vision for Patients with AMD and Geographic Atrophy.","authors":"Jacque Duncan","doi":"10.1056/nejme2514592","DOIUrl":"https://doi.org/10.1056/nejme2514592","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"219 1","pages":"298-301"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971900","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Steven E Schutzer,Anna-Marie Wellins,Raymond J Dattwyler
{"title":"Borrelia burgdorferi Infection and Erythema Migrans.","authors":"Steven E Schutzer,Anna-Marie Wellins,Raymond J Dattwyler","doi":"10.1056/nejmc2508805","DOIUrl":"https://doi.org/10.1056/nejmc2508805","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"39 1","pages":"302-303"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971903","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Advancing Diagnostic Excellence through Medical Education in Diagnostic Equity.","authors":"Arkene Levy, Jocelyn Mitchell-Williams","doi":"10.1056/NEJMc2516830","DOIUrl":"https://doi.org/10.1056/NEJMc2516830","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"394 3","pages":"308"},"PeriodicalIF":78.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145985621","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"2024-2025 Covid-19 Vaccine Outcomes in U.S. Veterans. Reply.","authors":"Miao Cai,Yan Xie,Ziyad Al-Aly","doi":"10.1056/nejmc2516766","DOIUrl":"https://doi.org/10.1056/nejmc2516766","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"177 1","pages":"307"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
BACKGROUNDIn patients with autoimmune hemolytic anemia (AIHA), the risk of relapse is high owing to persistent autoreactive B-cell activity. Multirefractory AIHA is a more advanced stage of disease that is defined by a lack of response to at least three lines of therapy. CD19-directed chimeric antigen receptor (CAR) T-cell therapy results in profound B-cell depletion and may be a useful approach to achieving drug-free remission in multirefractory AIHA.METHODSWe enrolled patients from a compassionate-use program and those from a phase 1 study who had primary multirefractory AIHA. Each patient received a single infusion of autologous CD19 CAR T cells. The primary objective was to assess the safety profile - the incidence, characteristics, and severity of adverse events, including cytokine-release syndrome and immune effector cell-associated neurotoxicity syndrome. Secondary objectives included efficacy and pharmacokinetic features. A complete response was defined by resolution of symptoms, an increased hemoglobin level, and normalization of hemolysis markers. B-cell reconstitution and the origin of relapse were analyzed with flow cytometry, single-cell RNA sequencing, and paired B-cell receptor sequencing.RESULTSCD19 CAR T cells were administered to 11 patients - 5 in the compassionate-use program and 6 in the phase 1 study. The median follow-up was 12.2 months (range, 7.3 to 21.9). All patients had a complete response; the median time to a complete response was 45 days (range, 21 to 153). The median duration of drug-free remission was 11.5 months (range, 6.8 to 21.0). Cytokine-release syndrome of grade 1 or 2 in severity occurred in 9 patients, and immune effector cell-associated neurotoxicity syndrome of grade 1 occurred in 1 patient. A total of 15 infections occurred among 7 patients, with no infections of grade 4 or higher. One patient had immune effector cell-associated hematotoxicity of grade 3. In multi-omics assessments of sequential samples, naive B cells were predominant in the reconstituted B-cell population in patients with drug-free remission, and crosstalk between HLA-DRB5+ B cells, CD4+ T cells, and B-cell maturation antigen-expressing long-lived plasma cells contributed to a relapse-specific B-cell niche.CONCLUSIONSCD19 CAR T-cell therapy had expected toxic effects and resulted in sustained remission in patients with multirefractory AIHA. (Funded by the National Key Research and Development Program of China and others; ClinicalTrials.gov number, NCT06231368.).
在自身免疫性溶血性贫血（AIHA）患者中，由于持续的自身反应性b细胞活性，复发的风险很高。多重难治性AIHA是一种更晚期的疾病，对至少三条治疗线缺乏反应。cd19靶向嵌合抗原受体（CAR） t细胞治疗导致b细胞严重耗竭，可能是实现多重难治性AIHA无药物缓解的有效方法。方法：我们招募了来自同情用药项目和来自一期研究的原发性多难治性AIHA患者。每位患者接受单次自体CD19 CAR - T细胞输注。主要目的是评估安全性——不良事件的发生率、特征和严重程度，包括细胞因子释放综合征和免疫效应细胞相关神经毒性综合征。次要目标包括疗效和药代动力学特征。完全缓解的定义是症状缓解、血红蛋白水平升高和溶血指标正常化。用流式细胞术、单细胞RNA测序和配对b细胞受体测序分析b细胞重构和复发的起源。结果11例患者使用了scd19 CAR - T细胞，其中5例在同情使用项目中，6例在i期研究中。中位随访时间为12.2个月（7.3 - 21.9个月）。所有患者均有完全缓解；达到完全缓解的中位时间为45天（范围21至153天）。无药物缓解的中位持续时间为11.5个月（范围为6.8至21.0个月）。9例患者出现1级或2级细胞因子释放综合征，1例患者出现1级免疫效应细胞相关神经毒性综合征。7例患者共发生15例感染，无4级及以上感染。1例患者有3级免疫效应细胞相关的血液毒性。在序列样本的多组学评估中，在无药物缓解患者的重组B细胞群中，原始B细胞占主导地位，HLA-DRB5+ B细胞、CD4+ T细胞和表达B细胞成熟抗原的长寿命浆细胞之间的串音有助于形成复发特异性B细胞生态位。结论：scd19 CAR - t细胞治疗具有预期的毒性作用，可使多重难治性AIHA患者持续缓解。（国家重点研发计划等资助；ClinicalTrials.gov编号：NCT06231368）。
{"title":"CD19 CAR T-Cell Therapy for Autoimmune Hemolytic Anemia.","authors":"Ruonan Li,Hong Pan,Lele Zhang,Jiaxiu Ma,Weiwang Li,Zhen Gao,Liwei Fang,Linzhu Tian,Yucan Shen,Fei Yang,Jingyu Zhao,Neng Nie,Jianping Li,Wenyan Wang,Xinan Pan,Yu Lian,Xingxin Li,Guangxin Peng,Liyun Li,Xiao Yu,Chun Xu,Yanjie Liu,Zhexiang Kuang,Jinbo Huang,Xin Zhao,Meili Ge,Lijun Liu,Shuo Chen,Yi Feng,Alex H Chang,Biping Deng,Min Dai,Lifang Huang,Lulu Lv,Yizhou Zheng,Yuechen Luo,Haiqing Xiong,Jun Shi","doi":"10.1056/nejmoa2509820","DOIUrl":"https://doi.org/10.1056/nejmoa2509820","url":null,"abstract":"BACKGROUNDIn patients with autoimmune hemolytic anemia (AIHA), the risk of relapse is high owing to persistent autoreactive B-cell activity. Multirefractory AIHA is a more advanced stage of disease that is defined by a lack of response to at least three lines of therapy. CD19-directed chimeric antigen receptor (CAR) T-cell therapy results in profound B-cell depletion and may be a useful approach to achieving drug-free remission in multirefractory AIHA.METHODSWe enrolled patients from a compassionate-use program and those from a phase 1 study who had primary multirefractory AIHA. Each patient received a single infusion of autologous CD19 CAR T cells. The primary objective was to assess the safety profile - the incidence, characteristics, and severity of adverse events, including cytokine-release syndrome and immune effector cell-associated neurotoxicity syndrome. Secondary objectives included efficacy and pharmacokinetic features. A complete response was defined by resolution of symptoms, an increased hemoglobin level, and normalization of hemolysis markers. B-cell reconstitution and the origin of relapse were analyzed with flow cytometry, single-cell RNA sequencing, and paired B-cell receptor sequencing.RESULTSCD19 CAR T cells were administered to 11 patients - 5 in the compassionate-use program and 6 in the phase 1 study. The median follow-up was 12.2 months (range, 7.3 to 21.9). All patients had a complete response; the median time to a complete response was 45 days (range, 21 to 153). The median duration of drug-free remission was 11.5 months (range, 6.8 to 21.0). Cytokine-release syndrome of grade 1 or 2 in severity occurred in 9 patients, and immune effector cell-associated neurotoxicity syndrome of grade 1 occurred in 1 patient. A total of 15 infections occurred among 7 patients, with no infections of grade 4 or higher. One patient had immune effector cell-associated hematotoxicity of grade 3. In multi-omics assessments of sequential samples, naive B cells were predominant in the reconstituted B-cell population in patients with drug-free remission, and crosstalk between HLA-DRB5+ B cells, CD4+ T cells, and B-cell maturation antigen-expressing long-lived plasma cells contributed to a relapse-specific B-cell niche.CONCLUSIONSCD19 CAR T-cell therapy had expected toxic effects and resulted in sustained remission in patients with multirefractory AIHA. (Funded by the National Key Research and Development Program of China and others; ClinicalTrials.gov number, NCT06231368.).","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"56 1","pages":"253-267"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971901","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"2024-2025 Covid-19 Vaccine Outcomes in U.S. Veterans. Reply.","authors":"Miao Cai, Yan Xie, Ziyad Al-Aly","doi":"10.1056/NEJMc2516766","DOIUrl":"https://doi.org/10.1056/NEJMc2516766","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"394 3","pages":"307"},"PeriodicalIF":78.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145985232","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Primary Palmoplantar Pustulosis.","authors":"Leila Shayegan, Ruth Ann Vleugels","doi":"10.1056/NEJMicm2505935","DOIUrl":"https://doi.org/10.1056/NEJMicm2505935","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"394 3","pages":"e5"},"PeriodicalIF":78.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145985890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Marlene L Durand,Mark J Siedner,John W Chen,Elizabeth J Rossin,David A Rosen,Erik H Klontz
{"title":"Case 2-2026: A 63-Year-Old Man with Pulmonary Nodules, Liver Mass, and Vision Loss.","authors":"Marlene L Durand,Mark J Siedner,John W Chen,Elizabeth J Rossin,David A Rosen,Erik H Klontz","doi":"10.1056/nejmcpc2402495","DOIUrl":"https://doi.org/10.1056/nejmcpc2402495","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"267 1","pages":"282-294"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971854","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Popeye Sign in Transthyretin Amyloidosis.","authors":"Raphael Boesche Guimarães,Felipe Milach","doi":"10.1056/nejmc2514537","DOIUrl":"https://doi.org/10.1056/nejmc2514537","url":null,"abstract":"","PeriodicalId":54725,"journal":{"name":"New England Journal of Medicine","volume":"261 1","pages":"310-311"},"PeriodicalIF":158.5,"publicationDate":"2026-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145971905","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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