Macrocytosis combined with monoclonal gammopathy identifies patients carrying VEXAS-linked UBA1 mutations [0.03%]
巨幼红细胞合并单克隆丙种球蛋白病可识别携带VEXAS相关UBA1基因突变的患者
Verena Petzer,Wolfram Mayr,Eva Spoegler et al.
Verena Petzer et al.
Rab GTPases drive ligand-independent NOTCH1 activation via altered endocytic trafficking in chronic lymphocytic leukemia [0.03%]
Rab GTP酶通过改变慢性淋巴细胞白血病中配体非依赖性NOTCH1活化的内吞运输路径来驱动其激活
Filomena De Falco,Beatrice Del Papa,Daniele Sorcini et al.
Filomena De Falco et al.
Clinically relevant NOTCH1 activation is frequently observed in chronic lymphocytic leukemia (CLL), even in the absence of gene mutations, raising questions about the underlying mechanisms. In CLL cells, NOTCH1 activation may occur through ...
Stephen P Hibbs
Stephen P Hibbs
Combined inhibition of de novo pyrimidine synthesis and ATR promotes ATF4-mediated cell death in TP53-deficient ALL [0.03%]
TP53缺陷型急性淋巴细胞白血病中联合抑制从头嘧啶合成和ATR促进ATF4介导的细胞死亡
Kari J T Grünewald,Marjolein M G Kes,Gawin R Stoll et al.
Kari J T Grünewald et al.
Mutations or deletions affecting the TP53 gene predict a dismal outcome in relapsed acute lymphoblastic leukemia (ALL). Loss of p53 function compromises the response to many anti-leukemic therapies, underscoring the need for agents that are...
Transplant versus no transplant outcome in adolescents and adults with sickle cell disease eligible for allogeneic hematopoietic stem cell transplantation [0.03%]
适行同种造血干细胞移植的镰状细胞病青少年及成人患者移植与不移植结局比较
Cécile Pivert,Loïc Vasseur,Matthieu Jestin et al.
Cécile Pivert et al.
Hematopoietic stem cell transplantation (HSCT) remains the only curative option for sickle cell disease (SCD), but its use in adolescents and adults is limited by toxicity concerns. We evaluated the risk-benefit profile of HSCT in this popu...
Preclinical evaluation of the ROR1-targeting antibody-drug conjugates zilovertamab vedotin and VLS-211 against B-cell ALL patient-derived xenografts [0.03%]
B细胞急性淋巴细胞白血病患者来源异种移植瘤中ROR1靶向抗体药物偶联物zilovertamab vedotin和VLS-211的药理学评估
Christopher M Smith,Ben Watts,Kathryn Evans et al.
Christopher M Smith et al.
Although survival rates for acute lymphoblastic leukemia (ALL) have improved dramatically over the past 60 years, outcomes vary across different molecular subtypes and risk categories, and a significant number of patients remain difficult t...
Hemophagocytic lymphohistiocytosis-like syndrome after CD19-directed CAR T-cells for B-cell lymphoma and B-cell acute lymphoblastic leukemia: A LYSA, SFCE, and GRAALL study from the DESCAR-T registry [0.03%]
抗CD19 CAR-T细胞治疗B细胞淋巴瘤和B细胞急性淋巴细胞白血病后类噬血细胞性淋巴组织细胞增多症样综合征：来自DESCAR-T登记的LYSA、SFCE和GRAALL研究结果
Nicolas Gower,Roch Houot,Cécile Pizot et al.
Nicolas Gower et al.
While cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) are well-recognized toxicities of CAR T-cell therapy, immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome ...
Etienne Rivière,Alexandre Guy,Olivier Mansier et al.
Etienne Rivière et al.
Peginterferon‑α is useful to treat myeloproliferative neoplasms (MPNs) but can precipitate broad autoimmunity. By promoting beta-cell, thyroid, and systemic immune injury, it can lead to Type 1 diabetes, autoimmune thyroiditis, systemic l...
Risk of thrombosis and bleeding following CAR T-cell therapy: Insights from the Dutch "Follow that CAR!" registry [0.03%]
来自荷兰"跟上CAR！"登记处的CAR-T细胞治疗后血栓形成和出血风险的见解
Amica K Ko,Pim G N J Mutsaers,Lukas M van Kleij et al.
Amica K Ko et al.
Chimeric antigen receptor (CAR) T-cell therapy has significantly improved outcomes in patients with relapsed/refractory aggressive large B-cell lymphoma (R/R LBCL). Although thrombosis and bleeding complications have been increasingly repor...
Switching between complement inhibitors in paroxysmal nocturnal hemoglobinuria: Analysis of strategy, efficacy, and safety [0.03%]
阵发性夜间血红蛋白尿患者补体抑制剂之间转换的策略、疗效和安全性分析
Morag Griffin,Bruno Fattizzo,Jong Wook Lee et al.
Morag Griffin et al.
Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease. In 2026, approved complement inhibitors (CIs) for PNH include three C5 inhibitors (C5i) and three proximal inhibitors (PIs). Clinical trials for the approved PI pegcetaco...