{"title":"","authors":"","doi":"10.11406/rinketsu.66.71","DOIUrl":"https://doi.org/10.11406/rinketsu.66.71","url":null,"abstract":"","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 1","pages":"71-72"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143384261","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}
{"title":"[Prophylactic efficacy of tixagevimab/cilgavimab in patients with hematological neoplasms: a single-center study].","authors":"Hirofumi Nakano, Shiori Nakashima, Yui Imai, Tomoyuki Uchida, Morihiro Inoue, Masao Hagihara","doi":"10.11406/rinketsu.66.3","DOIUrl":"10.11406/rinketsu.66.3","url":null,"abstract":"<p><p>We retrospectively analyzed the efficacy of tixagevimab/cilgavimab (Tix/Cil) in 142 patients (total of 157 injections) with hematological disorders. Fifteen patients (9.5%) were infected with coronavirus disease (COVID-19), and 3 of these remained infected even after repeated administration of Tix/Cil. Malignant lymphoma and multiple myeloma were the most frequent underlying disorders (frequencies of 18.9% and 17.5%, respectively). Whole genome sequencing of the Omicron variant was performed in 11 patients, and revealed sensitivity to Tix/Cil in only 2. In both cases, the severity of COVID-19 was moderate I or II. Since April 2023, when a Tix/Cil-resistant variant became dominant (frequency >70%), the incidence of breakthrough infections increased from 4% to 35%, and none responded to Tix/Cil. Vaccination, together with daily precautions against infection, is the current approach used to prevent COVID-19, particularly in patients with lymphoid malignancies, because Tix/Cil is no longer effective as prophylaxis.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 1","pages":"3-6"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143383987","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}
{"title":"","authors":"","doi":"10.11406/rinketsu.66.271","DOIUrl":"https://doi.org/10.11406/rinketsu.66.271","url":null,"abstract":"","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 4","pages":"271-293"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144032601","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}
{"title":"[Lessons from the first-in-human clinical trial of iPSC-derived platelets: aiming to understand platelet biogenesis].","authors":"Koji Eto","doi":"10.11406/rinketsu.66.509","DOIUrl":"https://doi.org/10.11406/rinketsu.66.509","url":null,"abstract":"<p><p>The iPLAT1 study was conducted from 2019 to 2020 as the first-in-human clinical trial of iPS cell-derived platelet products (iPSC-PLTs). The subject was a patient with aplastic anemia refractory to anti-HPA-1a antibody-induced platelet transfusions who had no matched HPA-1b/1b donor. Autologous iPSC-PLTs were manufactured from a megakaryocyte cell line, imMKCL, established from the patient's iPSCs. High-efficiency manufacturing of iPSC-PLTs was achieved by incorporating the concept of turbulent flow in bioreactor tanks to mimic in vivo conditions. After comprehensive non-clinical studies, the iPLAT1 study was conducted as a dose-escalation study and achieved the primary endpoint of safety. However, an increase in the platelet count after transfusion was not observed, raising the possibility of a failure in post-transfusion measurement or defective circulation of transfused iPSC-PLTs. Since then, my research team and I have been conducting reverse-translational research to improve imMKCLs and developing a larger-scale manufacturing system to improve turbulent flow in bioreactor tanks. We have also recently demonstrated properties of subset of immune megakaryocytes in imMKCLs. Building upon such efforts, we have newly begun R&D for next-generation iPSC-PLTs.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 6","pages":"509-516"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144577207","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}
{"title":"[Molecular targeted therapy for peripheral T-cell lymphoma].","authors":"Kazuyuki Shimada","doi":"10.11406/rinketsu.66.456","DOIUrl":"https://doi.org/10.11406/rinketsu.66.456","url":null,"abstract":"<p><p>Peripheral T-cell lymphoma (PTCL) is a highly heterogenous disease that accounts for 10 to 15% of malignant lymphomas. It encompasses a wide range of disease types, including nodal, extranodal, and leukemic forms. Recent molecular genetic findings about PTCL have significantly deepened our understanding of the disease, leading to the reclassification of previously distinct subtypes under a unified entity (e.g., T-follicular helper lymphoma). In terms of treatment, CHOP or CHOP-like therapy have been widely adopted as a first-line regimen. However, even in ALK-positive anaplastic large cell lymphoma, which generally has favorable outcomes, the prognosis of PTCL remains unsatisfactory. The extremely poor outcomes of relapsed and refractory disease have highlighted an urgent need for breakthrough therapies. In recent years, novel therapeutic approaches, including antibody-drug conjugates, epigenetic modifiers, and immune cell therapies, have improved clinical outcomes for some patients with PTCL. However, the optimal use of novel approaches remains unclear, and stratification based on molecular genetic findings is crucial to achieve more effective and precisely targeted treatment.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 6","pages":"456-463"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144577209","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}
{"title":"[Overview].","authors":"Satoshi Yoshihara","doi":"10.11406/rinketsu.66.422","DOIUrl":"https://doi.org/10.11406/rinketsu.66.422","url":null,"abstract":"","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 6","pages":"422-423"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144577213","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}
{"title":"[Diffuse large B-cell lymphoma presenting with anti-MAG/SGPG IgM gammopathy and neurolymphomatosis at onset].","authors":"Masaki Yoshida, Kuniko Takano, Yasuko Mori, Yosuke Kodama, Mizuki Arimatsu, Manami Iwanaga, Masanori Sakata, Kazuki Okuhiro, Shuhei Honda, Yui Moroga, Masao Ogata","doi":"10.11406/rinketsu.66.305","DOIUrl":"10.11406/rinketsu.66.305","url":null,"abstract":"<p><p>Anti-MAG/SGPG antibody is an IgM antibody against myelin-associated glycoprotein and sulfate-3-glucuronyl paragloboside, which constitute the myelin sheath, and peripheral neuropathy associated with this antibody causes symptoms such as sensory disturbances and ataxia. Anti-MAG/SGPG antibodies are known to be associated with IgM monoclonal gammopathy derived from plasma cell tumors. Chemotherapy with rituximab has been shown to yield neurological improvement in patients with peripheral neuropathy positive for anti-MAG/SGPG antibodies. Here we report a very rare case of diffuse large B-cell lymphoma presenting with neurolymphomatosis along with sensorimotor neuropathy associated with anti-MAG/SGPG antibody-positive IgM monoclonal gammopathy.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 5","pages":"305-311"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227925","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}
{"title":"[Hemostatic therapy for hemorrhagic cholecystitis in a patient with hemophilia A with inhibitors].","authors":"Yumi Oshima, Makiko Mizuguchi, Yasunobu Okamoto, Kumiko Kagawa, Hironobu Shibata, Yusuke Arakawa, Toshihiro Omoya, Hiroyoshi Watanabe, Shuji Ozaki","doi":"10.11406/rinketsu.66.312","DOIUrl":"https://doi.org/10.11406/rinketsu.66.312","url":null,"abstract":"<p><p>The patient was a 47-year-old man. He was diagnosed with hemophilia A in childhood, and received replacement therapy with FVIII products. However, this became ineffective due to inhibitor production, and he was treated with emicizumab. He developed fever and vomiting, and abdominal CT revealed an enlarged gallbladder and gallstones. Acute cholecystitis was diagnosed, and he was hospitalized and treated conservatively with antibiotics. On the 12th day of illness, the anemia progressed and a high-density area was observed in the gallbladder by CT scan, leading to a diagnosis of hemorrhagic cholecystitis. Since the inhibitor titer was low at 3.6 BU/ml, neutralization therapy with FVIII products was attempted. FVIII activity increased to 155%, and emergency laparoscopic subtotal cholecystectomy was performed successfully. After surgery, FVIII replacement therapy was continued and FVIII activity was maintained, but on the 19th day of illness, it decreased to 16%, and inhibitor levels rose again. Emicizumab is effective in preventing bleeding in hemophilia A with inhibitors, but the risk of bleeding is high if tissue damage leads to severe inflammation or necrosis. Neutralization therapy with FVIII products might be effective in hemorrhagic emergencies and minimally invasive surgery for hemophilia A patients with inhibitors when emicizumab is used to reduce inhibitor levels.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 5","pages":"312-317"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227928","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}
{"title":"","authors":"","doi":"10.11406/rinketsu.66.355","DOIUrl":"https://doi.org/10.11406/rinketsu.66.355","url":null,"abstract":"","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 5","pages":"355-368"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144227920","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}
{"title":"[Gene therapy for hemophilia].","authors":"Yoshitaka Miyakawa","doi":"10.11406/rinketsu.66.465","DOIUrl":"10.11406/rinketsu.66.465","url":null,"abstract":"<p><p>Gene therapies for hemophilia A and B were approved in the US and EU in 2022. A single infusion of adeno-associated virus vector containing FVIII and FIX gene increases clotting factor levels within a few weeks. Around 90% of hemophilia patients treated with gene therapy no longer need clotting factor injections and show a reduced annual bleeding rate. Gene therapy vectors for hemophilia have strong liver tropism and use liver-specific promoters. This review discusses the history of hemophilia gene therapy, phase 3 trials, and unmet needs.</p>","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 6","pages":"465-472"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144577206","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}