[Rinsho ketsueki] The Japanese journal of clinical hematology最新文献_第2页

[Next-generation CAR gene-modified cell therapy: overcoming resistance and exploring novel applications]. [下一代CAR基因修饰细胞疗法：克服耐药性和探索新的应用]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.679

Reona Leo Sakemura

{"title":"[Next-generation CAR gene-modified cell therapy: overcoming resistance and exploring novel applications].","authors":"Reona Leo Sakemura","doi":"10.11406/rinketsu.66.679","DOIUrl":"https://doi.org/10.11406/rinketsu.66.679","url":null,"abstract":"In recent years, chimeric antigen receptor (CAR)-engineered cellular therapy has brought remarkable advancements in cancer immunotherapy and autoimmune disease treatment. CAR T-cell therapy has demonstrated high efficacy in multiple myeloma (MM), but its durability is limited due to immune suppression within the tumor microenvironment (TME). This study elucidates how cancer-associated fibroblasts (CAFs) impair BCMA CAR T-cell function, and describes development of dual-specific CAR T-cells targeting CAFs. The results showed that CAFs promoted CAR T-cell exhaustion via TGF-β, PD-L1, IL-10, and the FAS/FASL pathway. BCMA-FAP and BCMA-CS1 CAR T cells exhibited enhanced cytotoxicity against MM cells and CAFs, overcoming TME-mediated suppression. E-cadherin-targeting CAR MSCs (Ecad CAR-MSCs) to address graft-versus-host disease (GvHD) were also developed for this study. These CAR MSCs significantly reduced GvHD by selectively accumulating in the intestinal epithelium, suppressing T-cell activation via IL-10 and galectin-9 while promoting Treg induction. These findings suggest that CAF-targeting dual-specific CAR T cells enhance the efficacy of MM immunotherapy, while Ecad CAR-MSCs offer a novel approach to treating GvHD. These approaches hold promise for clinical translation to improve outcomes in cellular therapy.","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 7","pages":"679-686"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144796385","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

[Overview]. (概述)。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.580

Takahiro Suzuki

引用次数: 0

[New treatment strategies for paroxysmal nocturnal hemoglobinuria: drug selection in the era of novel complement inhibitors]. [阵发性夜间血红蛋白尿的新治疗策略：新型补体抑制剂时代的药物选择]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.756

Yasutaka Ueda

{"title":"[New treatment strategies for paroxysmal nocturnal hemoglobinuria: drug selection in the era of novel complement inhibitors].","authors":"Yasutaka Ueda","doi":"10.11406/rinketsu.66.756","DOIUrl":"10.11406/rinketsu.66.756","url":null,"abstract":"Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal disorder characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure. Eculizumab (Ecu), a C5 inhibitor, blocks intravascular hemolysis (IVH) and improves prognosis. Ravulizumab and crovalimab have longer half-lives, and reduce treatment burden. Crovalimab is effective in patients with C5 polymorphism resistant to Ecu. Despite C5 inhibition, approximately 70% of patients remain anemic due to bone marrow failure and extravascular hemolysis (EVH) from C3 deposition. Upstream complement inhibition can address both IVH and EVH. Pegcetacoplan (C3 inhibitor) improves anemia better than Ecu. Danicopan (factor D inhibitor) and ipracopan (factor B inhibitor) also improve anemia and fatigue. Proximal inhibitors offer better anemia control but pose a risk of breakthrough hemolysis (BTH), especially under complement-amplifying conditions. In addition, long-term real-world infection data remain necessary. While IVH control has improved PNH prognosis, future therapies should focus on patients with persistent anemia or insufficient improvement in quality of life, and should aim to enhance hemoglobin levels and overall well-being while managing BTH for optimal care.","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 8","pages":"756-765"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144994532","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

[Acquired pure red cell aplasia]. 获得性纯红细胞发育不全。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.926

Hideyuki Nakazawa

引用次数: 0

[Challenges in optimizing targeted complement inhibitor therapy for paroxysmal nocturnal hemoglobinuria in an expanding treatment landscape]. [在不断扩大的治疗领域中优化靶向补体抑制剂治疗阵发性夜间血红蛋白尿的挑战]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.915

Shikiko Ueno

{"title":"[Challenges in optimizing targeted complement inhibitor therapy for paroxysmal nocturnal hemoglobinuria in an expanding treatment landscape].","authors":"Shikiko Ueno","doi":"10.11406/rinketsu.66.915","DOIUrl":"https://doi.org/10.11406/rinketsu.66.915","url":null,"abstract":"The advent of the C5 inhibitor eculizumab marked a paradigm shift in the management of PNH, transforming it from \"a rare disease with a poor prognosis\" to \"a manageable disease\". However, emerging challenges such as primary nonresponse due to C5 gene polymorphisms and residual extravascular hemolysis have prompted the development of new therapeutic agents. Three C5 inhibitors and three proximal complement inhibitors are now available. For complement inhibitor-naïve patients with PNH, C5 inhibitors are the first-line therapy. Selection of eculizumab, ravulizumab, or crovalimab should be based on factors such as dosing frequency, route of administration, C5 gene polymorphisms, and pregnancy status. When C5 inhibitor therapy does not yield sufficient improvement in anemia or quality of life, second-line treatment with proximal complement inhibitors should be considered. Selection of pegcetacoplan, danicopan, or iptacopan should be guided by the underlying pathology of anemia, lifestyle factors, and treatment adherence. This article provides an overview of the pharmacological mechanisms, efficacy, safety, and clinical applications of each drug, along with the future outlook, based on the latest findings.","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 9","pages":"915-925"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208555","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

[Coagulation function tests: principles and clinical application]. 【凝血功能试验：原理及临床应用】。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.1158

Nobuaki Suzuki

引用次数: 0

[Myeloid tumors and antitumor innate immunity]. [骨髓肿瘤与抗肿瘤先天免疫]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.883

Susumu Goyama

引用次数: 0

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.295

引用次数: 0

[Extramedullary relapse of acute myeloid leukemia with pericardial effusion after allogeneic hematopoietic stem cell transplantation]. [同种异体造血干细胞移植后急性髓性白血病髓外复发伴心包积液]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.170

Yurie Saitou, Hayato Matsumoto, Takuro Shimbo, Shin Matsuda

引用次数: 0

[Successful treatment with venetoclax and azacitidine in a patient with acute myeloid leukemia with BCR::ABL1 fusion]. 【venetoclax联合阿扎胞苷治疗急性髓系白血病合并BCR::ABL1融合1例成功】。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.184

Kenta Motegi, Akira Katsumi

引用次数: 0