[Rinsho ketsueki] The Japanese journal of clinical hematology最新文献_第5页

[Validation of gradient-boosted models for non-invasive diagnosis of myelodysplastic neoplasms: a single-center analysis]. [验证梯度增强模型对骨髓增生异常肿瘤的非侵入性诊断：单中心分析]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.535

Toma Suzuki, Akio Mori, Emi Yokoyama, Minoru Kanaya, Koh Izumiyama, Makoto Saito, Masanobu Morioka, Takeshi Kondo

{"title":"[Validation of gradient-boosted models for non-invasive diagnosis of myelodysplastic neoplasms: a single-center analysis].","authors":"Toma Suzuki, Akio Mori, Emi Yokoyama, Minoru Kanaya, Koh Izumiyama, Makoto Saito, Masanobu Morioka, Takeshi Kondo","doi":"10.11406/rinketsu.66.535","DOIUrl":"https://doi.org/10.11406/rinketsu.66.535","url":null,"abstract":"Bone marrow examination is essential for the definitive diagnosis of myelodysplastic neoplasms (MDS). However, non-invasive diagnostic approaches are needed for patients who cannot tolerate the procedure, especially elderly patients. This study aimed to validate the diagnostic accuracy of non-invasive gradient-boosted models (GBMs) in Japanese patients with MDS. When used alone, GBMs had sensitivity of 63.2% and specificity of 43.9% for diagnosis of MDS. When combined with WT1 mRNA expression levels (WT1 levels)(cutoff: 150 copies/µg RNA), specificity remained similar at 50.0%, while sensitivity improved to 81.6%. MDS was correctly diagnosed in 87.2% of patients with probable or indeterminate MDS by GBMs who had WT1 levels higher than 150 copies/µg RNA. When patients with vitamin B12 deficiency were excluded from analysis, this percentage increased to 91.9%. These findings suggest that the combination of GBMs, WT1 levels, and vitamin B12 deficiency could non-invasively identify patients likely to have MDS.","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 7","pages":"535-540"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144796394","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

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.355

引用次数: 0

[Gene therapy for hemophilia]. [血友病的基因治疗]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.465

Yoshitaka Miyakawa

引用次数: 0

[Lessons from the first-in-human clinical trial of iPSC-derived platelets: aiming to understand platelet biogenesis]. 【ipsc衍生血小板的首次人体临床试验的经验教训：旨在了解血小板的生物发生】。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.509

Koji Eto

{"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":"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.","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}

引用次数: 0

[Diffuse large B-cell lymphoma presenting with anti-MAG/SGPG IgM gammopathy and neurolymphomatosis at onset]. [弥漫性大b细胞淋巴瘤，发病时表现为抗mag /SGPG IgM γ病变和神经淋巴瘤病]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.305

Masaki Yoshida, Kuniko Takano, Yasuko Mori, Yosuke Kodama, Mizuki Arimatsu, Manami Iwanaga, Masanori Sakata, Kazuki Okuhiro, Shuhei Honda, Yui Moroga, Masao Ogata

引用次数: 0

[Hemostatic therapy for hemorrhagic cholecystitis in a patient with hemophilia A with inhibitors]. [a型血友病患者出血性胆囊炎的抑制剂止血治疗]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.312

Yumi Oshima, Makiko Mizuguchi, Yasunobu Okamoto, Kumiko Kagawa, Hironobu Shibata, Yusuke Arakawa, Toshihiro Omoya, Hiroyoshi Watanabe, Shuji Ozaki

{"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":"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.","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}

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[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.271

引用次数: 0

[Drug-drug interactions between antifungal agents and molecular-targeted agents]. [抗真菌药物和分子靶向药物之间的药物相互作用]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.1215

Takeo Yamagiwa

{"title":"[Drug-drug interactions between antifungal agents and molecular-targeted agents].","authors":"Takeo Yamagiwa","doi":"10.11406/rinketsu.66.1215","DOIUrl":"https://doi.org/10.11406/rinketsu.66.1215","url":null,"abstract":"Drug interactions occur when the concomitant use of multiple drugs enhances or reduces their pharmacological effects, or causes unexpectedly strong adverse reactions. Major mechanisms include alterations in cytochrome P450 (CYP) enzyme activity, chelation within the gastrointestinal tract, and inhibition of renal transporters. In the treatment of hematologic malignancies, combination of molecular targeted therapies with immunosuppressants often increases the risk of infections due to drug-induced cytopenia and immunosuppression. Azole antifungal agents, frequently used for the prevention and treatment of fungal infections, are known to inhibit CYP enzymes. This inhibition can increase the plasma concentrations of targeted therapies, thereby enhancing their toxicity and increasing the risk of severe adverse effects. The degree of interaction is influenced not only by specific drug combinations but also by interindividual variability. Advanced pharmacokinetic approaches such as concentration ratio-interaction risk methods, physiologically based pharmacokinetic modeling, and population pharmacokinetic analysis allow for the prediction and evaluation of such interactions. These tools enable more precise drug selection and dosage adjustments tailored to individual patients, supporting treatment personalization and promoting safer, more effective drug therapy in clinical practice.","PeriodicalId":93844,"journal":{"name":"[Rinsho ketsueki] The Japanese journal of clinical hematology","volume":"66 9","pages":"1215-1221"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145208710","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

[Diffuse large B-cell lymphoma: current treatment strategy and future outlook]. [弥漫大b细胞淋巴瘤：目前的治疗策略和未来展望]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.1055

Shinichi Makita

引用次数: 0

[Future perspectives on hematopoietic stem cell gene therapy]. [造血干细胞基因治疗的未来展望]。

[Rinsho ketsueki] The Japanese journal of clinical hematology Pub Date : 2025-01-01 DOI: 10.11406/rinketsu.66.891

Masahumi Onodera

引用次数: 0