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Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea. 韩国 B 型血友病患者治疗方案和用药的临床数据。
IF 2.3
Blood Research Pub Date : 2024-08-08 DOI: 10.1007/s44313-024-00024-8
Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi
{"title":"Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea.","authors":"Young Shil Park, Ji Kyoung Park, Jeong A Park, Hee Jo Baek, Jae Hee Lee, Chur Woo You, Chuhl Joo Lyu, Eun Jin Choi","doi":"10.1007/s44313-024-00024-8","DOIUrl":"10.1007/s44313-024-00024-8","url":null,"abstract":"<p><strong>Background: </strong>To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.</p><p><strong>Methods: </strong>In this retrospective chart review, data of patients with hemophilia B from eight university hospitals were collected. Demographic and clinical data, treatment data, such as regimen and number of injections, dose of factor IX concentrate, and bleeding data were reviewed. Descriptive analyses were performed with annual data for 2019, 2020, and 2021, as well as the three years consecutively.</p><p><strong>Results: </strong>The medical records of 150 patients with hemophilia B between January 1, 2019, and December 31, 2021, were collected. Among these, 72 (48.0%) were severe, 47 (31.3%) were moderate, and 28 (18.7%) were mild. The results showed approximately two times more patients receiving prophylaxis as those receiving on-demand therapy, with 66.1% of patients receiving prophylaxis in 2019, 64.9% in 2020, and 72.1% in 2021. Annualized bleeding rates were 2.2% (± 3.1) in 2019, 1.8% (± 3.0) in 2020, and 1.8% (± 2.9) in 2021 among patients receiving prophylaxis. For the doses of factor IX concentrate, patients receiving prophylaxis received an average of 41.6 (± 11.9) IU/Kg/Injection in 2019, 45.7 (± 12.9) IU/Kg/Injection in 2020, and 60.1 (± 24.0) IU/Kg/Injection in 2021.</p><p><strong>Conclusions: </strong>Clinically, prophylaxis is more prevalent than reported. Based on insights gained from current clinical evidence, it is expected that the unmet medical needs of patients can be identified, and physicians can evaluate the status of patients and actively manage hemophilia B using more effective treatment strategies.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11310372/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141903148","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Functional iron deficiency anemia in patients with cancer. 癌症患者的功能性缺铁性贫血。
IF 2.3
Blood Research Pub Date : 2024-08-07 DOI: 10.1007/s44313-024-00030-w
Jeong Suk Koh, Ik-Chan Song
{"title":"Functional iron deficiency anemia in patients with cancer.","authors":"Jeong Suk Koh, Ik-Chan Song","doi":"10.1007/s44313-024-00030-w","DOIUrl":"10.1007/s44313-024-00030-w","url":null,"abstract":"<p><p>Anemia is frequently observed in patients with cancer owing to anticancer chemotherapy, radiation therapy, and inflammatory responses. This often leads to functional iron deficiency, characterized by adequate iron stores but impaired use of iron for red blood cell production. This condition, termed functional iron deficiency anemia (IDA), is identified by a ferritin level of 30-500 µg/dL and a transferrin saturation < 50%. Functional iron deficiency often develops with the prolonged use of erythropoiesis-stimulating agents, leading to a diminished response to anemia treatment. Although oral iron supplementation is common, intravenous iron is more effective and recommended in such cases. Recent studies have shown that ferric carboxymaltose (FCM) is effective in treating functional IDA in patients with cancer. However, because of its potential to induce asymptomatic severe phosphate deficiency, it is important to closely monitor phosphate levels in patients receiving FCM.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11306885/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141898564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Proper application of anticoagulation therapy on cancer-associated venous thrombosis. 正确应用抗凝疗法治疗癌症相关静脉血栓。
IF 2.3
Blood Research Pub Date : 2024-08-02 DOI: 10.1007/s44313-024-00029-3
Ho-Young Yhim
{"title":"Proper application of anticoagulation therapy on cancer-associated venous thrombosis.","authors":"Ho-Young Yhim","doi":"10.1007/s44313-024-00029-3","DOIUrl":"10.1007/s44313-024-00029-3","url":null,"abstract":"<p><p>Cancer-associated venous thromboembolism (VTE) significantly impacts morbidity and mortality. The introduction of direct oral anticoagulants over the past decade has revolutionized VTE treatment in patients with active cancer, offering potential advantages over traditional therapies. However, uncertainties persist regarding the optimal selection and dosage of anticoagulants, particularly in patients with specific risk factors for bleeding, such as certain cancer types (e.g., upper gastrointestinal cancer, genitourinary cancer, primary or metastatic brain tumor, and hematologic malignancies) and specific patient characteristics (e.g., renal dysfunction and thrombocytopenia). Recent data on the thrombotic risk associated with low thrombotic burden VTE, such as subsegmental pulmonary embolism and isolated distal deep vein thrombosis, underscore the need for updated management strategies in daily clinical practice. This review aims to explore these issues and highlight the evolving landscape of cancer-associated VTE management.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11296996/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141876264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative efficacy of VMP vs. Rd in newly diagnosed, autologous stem cell transplant-ineligible multiple myeloma patients: a prematurely terminated randomized controlled study, CAREMM-2002 study. VMP与Rd对新诊断的、符合自体干细胞移植条件的多发性骨髓瘤患者的疗效比较:一项提前终止的随机对照研究,CAREMM-2002研究。
IF 2.3
Blood Research Pub Date : 2024-07-17 DOI: 10.1007/s44313-024-00025-7
Cheong Yoon Huh, Sung-Soo Park, Jung Yeon Lee, Chang-Ki Min
{"title":"Comparative efficacy of VMP vs. Rd in newly diagnosed, autologous stem cell transplant-ineligible multiple myeloma patients: a prematurely terminated randomized controlled study, CAREMM-2002 study.","authors":"Cheong Yoon Huh, Sung-Soo Park, Jung Yeon Lee, Chang-Ki Min","doi":"10.1007/s44313-024-00025-7","DOIUrl":"10.1007/s44313-024-00025-7","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11254880/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141628063","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment with ropeginterferon alfa-2b in patients with hydroxyurea resistant or intolerant polycythemia vera in South Korea: one-year results from a phase 2 study. 在韩国,对羟基脲耐药或不耐受的多发性红细胞症患者使用罗京干扰素 alfa-2b 治疗:一项 2 期研究的一年结果。
IF 2.3
Blood Research Pub Date : 2024-07-09 DOI: 10.1007/s44313-024-00026-6
Seug Yun Yoon, Sung-Eun Lee
{"title":"Treatment with ropeginterferon alfa-2b in patients with hydroxyurea resistant or intolerant polycythemia vera in South Korea: one-year results from a phase 2 study.","authors":"Seug Yun Yoon, Sung-Eun Lee","doi":"10.1007/s44313-024-00026-6","DOIUrl":"10.1007/s44313-024-00026-6","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11233476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141560040","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advancements in the understanding and management of histiocytic neoplasms. 组织细胞瘤的认识和治疗进展。
IF 2.3
Blood Research Pub Date : 2024-07-04 DOI: 10.1007/s44313-024-00022-w
Kyung-Nam Koh, Su Hyun Yoon, Sung Han Kang, Hyery Kim, Ho Joon Im
{"title":"Advancements in the understanding and management of histiocytic neoplasms.","authors":"Kyung-Nam Koh, Su Hyun Yoon, Sung Han Kang, Hyery Kim, Ho Joon Im","doi":"10.1007/s44313-024-00022-w","DOIUrl":"10.1007/s44313-024-00022-w","url":null,"abstract":"<p><p>Histiocytic neoplasms are rare diseases involving macrophages, dendritic cells, and monocytes. They include Langerhans cell histiocytosis (LCH), Erdheim-Chester disease (ECD), Rosai-Dorfman disease (RDD), juvenile xanthogranuloma (JXG), and histiocytic sarcoma. Histiocytic neoplasms are characterized by varied clinical courses and prognoses, necessitating a nuanced understanding of their classification, epidemiology, and clinical manifestations. Genetic studies have revealed somatic mutations, predominantly in the MAPK pathway, suggesting a clonal neoplastic nature. This review covers the current understanding of histiocytic neoplasms, molecular pathophysiology, with a particular focus on mutations in genes such as BRAF, MAP2K1, and the PI3K-AKT signaling pathways, and evolving treatment strategies, especially focusing on LCH, ECD, RDD, and JXG. The treatment landscape has evolved with advancements in targeted therapies. BRAF inhibitors, such as vemurafenib and dabrafenib, have shown efficacy, especially in high-risk LCH cases; however, challenges remain, including relapse post-treatment discontinuation, and adverse effects. MEK inhibitors have also demonstrated effectiveness, and cobimetinib has recently been approved for use in adults. Further research is required to determine the optimal treatment duration and strategies for managing therapy interruptions. Advancements in molecular genetics and targeted therapies have revolutionized the management of histiocytic neoplasms. However, ongoing research is crucial for optimizing patient outcomes.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.3,"publicationDate":"2024-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11224208/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141499323","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Preoperative consultation for determining the appropriate transfusion strategy. 术前咨询,以确定适当的输血策略。
IF 2.2
Blood Research Pub Date : 2024-06-07 DOI: 10.1007/s44313-024-00021-x
Ka-Won Kang
{"title":"Preoperative consultation for determining the appropriate transfusion strategy.","authors":"Ka-Won Kang","doi":"10.1007/s44313-024-00021-x","DOIUrl":"10.1007/s44313-024-00021-x","url":null,"abstract":"<p><p>Surgical patients are at risk of postoperative complications and mortality, necessitating preoperative patient optimization through the identification and correction of modifiable risk factors. Although preoperative platelet transfusions aim to reduce the risk of bleeding, their efficacy remains uncertain. Similarly, red blood cell transfusion in patients with anemia does not reduce the risk of postoperative mortality and may exacerbate complications. Therefore, developing individualized strategies that focus on correcting preoperative complete blood count abnormalities and minimizing transfusion requirements are essential. This review aimed to examine complete blood count abnormalities and appropriate transfusion strategies to minimize postoperative complications.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2024-06-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11161442/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141284995","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Correction: MicroRNA-765 is upregulated in myelodysplastic syndromes and induces apoptosis via PLP2 inhibition in leukemia cells. 更正:MicroRNA-765在骨髓增生异常综合征中上调,并通过抑制白血病细胞中的PLP2诱导细胞凋亡。
IF 2.2
Blood Research Pub Date : 2024-05-27 DOI: 10.1007/s44313-024-00020-y
Seong-Ho Kang, Ji Seon Choi
{"title":"Correction: MicroRNA-765 is upregulated in myelodysplastic syndromes and induces apoptosis via PLP2 inhibition in leukemia cells.","authors":"Seong-Ho Kang, Ji Seon Choi","doi":"10.1007/s44313-024-00020-y","DOIUrl":"10.1007/s44313-024-00020-y","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2024-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11130100/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141155447","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Assessment of the phenotypic severity of hemophilia A: using rotational thromboelastometry (ROTEM) and APTT-clot waveform analysis. 评估血友病 A 的表型严重程度:使用旋转血栓弹性测定法 (ROTEM) 和 APTT-凝血波形分析法。
IF 2.2
Blood Research Pub Date : 2024-05-14 DOI: 10.1007/s44313-024-00018-6
Deepika Gupta, Vandana Arya, Jasmita Dass, Nitin Gupta, Manas Kalra, Anupam Sachdeva, Jyoti Kotwal
{"title":"Assessment of the phenotypic severity of hemophilia A: using rotational thromboelastometry (ROTEM) and APTT-clot waveform analysis.","authors":"Deepika Gupta, Vandana Arya, Jasmita Dass, Nitin Gupta, Manas Kalra, Anupam Sachdeva, Jyoti Kotwal","doi":"10.1007/s44313-024-00018-6","DOIUrl":"10.1007/s44313-024-00018-6","url":null,"abstract":"<p><strong>Background: </strong>Hemophilia A (HA) is an X-linked inherited bleeding disorder caused by reduced factor VIII (FVIII) levels. Approximately 10-15% of patients with severe HA (SHA) do not present with the anticipated bleeding pattern. Here, we assessed the phenotypic severity of hemophilia A using rotational thromboelastometry (ROTEM) and activated partial thromboplastin time-clot waveform analysis (APTT-CWA).</p><p><strong>Methods: </strong>Patients diagnosed with hemophilia A were enrolled. Clinical phenotype assignment was performed according to the published literature, and patients were classified into four phenotypic subgroups. The whole blood sample was first run on ROTEM in INTEM mode using platelet-poor plasma, APTT was run, and the APTT-CWA graph was simultaneously recorded.</p><p><strong>Results: </strong>A total of 66 patients were recruited for this study. Statistically significant differences were observed between the four phenotypically categorized groups using ROTEM and APTT-CWA. On comparing patients with mild/moderate-to-severe phenotypes (Group II) with SHA without inhibitors (Group IV), no significant difference was found for all parameters of ROTEM or APTT-CWA. The MCF, MA30, MAXV, and Alpha angle values using ROTEM were found to be the lowest in patients with SHA with inhibitors, which helped differentiate them from those with SHA without inhibitors. However, these two groups could not be differentiated using the APTT-CWA parameters.</p><p><strong>Conclusion: </strong>ROTEM can be used to distinguish patients with SHA with inhibitors from those with SHA without inhibitors using a combination of parameters with high sensitivity and specificity. However, APTT-CWA cannot be used to differentiate these patient groups.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2024-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11093952/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140921185","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pathologic characteristics of histiocytic and dendritic cell neoplasms. 组织细胞和树突状细胞肿瘤的病理特征。
IF 2.2
Blood Research Pub Date : 2024-05-07 DOI: 10.1007/s44313-024-00015-9
Sun Och Yoon
{"title":"Pathologic characteristics of histiocytic and dendritic cell neoplasms.","authors":"Sun Och Yoon","doi":"10.1007/s44313-024-00015-9","DOIUrl":"10.1007/s44313-024-00015-9","url":null,"abstract":"<p><p>Histiocytic and dendritic cell neoplasms comprise diverse tumors originating from the mononuclear phagocytic system, which includes monocytes, macrophages, and dendritic cells. The 5th edition of the World Health Organization (WHO) classification updating the categorization of these tumors, reflecting a deeper understanding of their pathogenesis.In this updated classification system, tumors are categorized as Langerhans cell and other dendritic cell neoplasms, histiocyte/macrophage neoplasms, and plasmacytoid dendritic cell neoplasms. Follicular dendritic cell neoplasms are classified as mesenchymal dendritic cell neoplasms within the stroma-derived neoplasms of lymphoid tissues.Each subtype of histiocytic and dendritic cell neoplasms exhibits distinct morphological characteristics. They also show a characteristic immunophenotypic profile marked by various markers such as CD1a, CD207/langerin, S100, CD68, CD163, CD4, CD123, CD21, CD23, CD35, and ALK, and hematolymphoid markers such as CD45 and CD43. In situ hybridization for EBV-encoded small RNA (EBER) identifies a particular subtype. Immunoprofiling plays a critical role in determining the cell of origin and identifying the specific subtype of tumors. There are frequent genomic alterations in these neoplasms, especially in the mitogen-activated protein kinase pathway, including BRAF (notably BRAF V600E), MAP2K1, KRAS, and NRAS mutations, and ALK gene translocation.This review aims to offer a comprehensive and updated overview of histiocytic and dendritic cell neoplasms, focusing on their ontogeny, morphological aspects, immunophenotypic profiles, and molecular genetics. This comprehensive approach is essential for accurately differentiating and classifying neoplasms according to the updated WHO classification.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":null,"pages":null},"PeriodicalIF":2.2,"publicationDate":"2024-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11076448/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140857730","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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