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Monoclonal gammopathy of renal significance from the perspective of nephrologists. 从肾脏病专家的角度看肾脏单克隆抗体病。
IF 2.3 Q2 HEMATOLOGY Pub Date : 2024-08-12 DOI: 10.1007/s44313-024-00027-5
Kootae Park, Soon Hyo Kwon

Kidney disease is a frequent complication of multiple myeloma and other malignancies associated with monoclonal gammopathies. Additionally, dysproteinemia-related kidney disease can occur independently of overt multiple myeloma or hematologic malignancies. Monoclonal gammopathy of renal significance (MGRS) is a spectrum of disorders in which a monoclonal immunoglobulin produced by a benign or premalignant B-cell or plasma cell clone causes kidney damage. MGRS-associated renal disease manifests in various forms, including immunoglobulin-associated amyloidosis, monoclonal immunoglobulin deposition diseases (light chain, heavy chain, and combined light and heavy chain deposition diseases), proliferative glomerulonephritis with monoclonal immunoglobulin deposits, C3 glomerulopathy with monoclonal gammopathy, and light chain proximal tubulopathy. Although MGRS is a nonmalignant or premalignant hematologic condition, it has significant renal implications that often lead to progressive kidney damage and, eventually, end-stage kidney disease. This review discusses the epidemiology, pathogenesis, and management of MGRS and focuses on the perspective of nephrologists.

肾脏疾病是多发性骨髓瘤和其他与单克隆丙种球蛋白病相关的恶性肿瘤的常见并发症。此外,蛋白尿异常相关的肾脏疾病也可能独立于明显的多发性骨髓瘤或血液恶性肿瘤而发生。肾脏单克隆抗体病(MGRS)是一种由良性或恶性前 B 细胞或浆细胞克隆产生的单克隆免疫球蛋白导致肾脏损伤的疾病。与 MGRS 相关的肾脏疾病有多种表现形式,包括免疫球蛋白相关性淀粉样变性、单克隆免疫球蛋白沉积病(轻链、重链以及轻重链联合沉积病)、伴有单克隆免疫球蛋白沉积的增生性肾小球肾炎、伴有单克隆丙种球蛋白病的 C3 肾小球病以及轻链近端肾小管病。虽然 MGRS 是一种非恶性或恶性前血液病,但它对肾脏有重大影响,往往会导致进行性肾损害,最终导致终末期肾病。这篇综述讨论了 MGRS 的流行病学、发病机制和治疗方法,重点从肾病学家的角度进行分析。
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引用次数: 0
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 Q2 HEMATOLOGY 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

Background: To investigate the clinical treatment status, such as treatment regimen, bleeding events, and drug dose, in patients with hemophilia B in South Korea.

Methods: 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.

Results: 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.

Conclusions: 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.

背景:调查韩国血友病 B 患者的临床治疗状况,如治疗方案、出血事件和药物剂量:调查韩国 B 型血友病患者的临床治疗状况,如治疗方案、出血事件和药物剂量:在这项回顾性病历审查中,收集了 8 家大学医院的 B 型血友病患者数据。方法:在这项回顾性病历审查中,收集了 8 家大学医院的血友病 B 患者数据,审查了人口统计学和临床数据、治疗数据(如治疗方案和注射次数、浓缩因子 IX 的剂量)以及出血数据。对 2019 年、2020 年和 2021 年以及连续三年的年度数据进行了描述性分析:收集了 150 名 B 型血友病患者在 2019 年 1 月 1 日至 2021 年 12 月 31 日期间的医疗记录。其中,72 人(48.0%)为重度,47 人(31.3%)为中度,28 人(18.7%)为轻度。结果显示,接受预防性治疗的患者约为按需治疗患者的两倍,2019 年接受预防性治疗的患者占 66.1%,2020 年占 64.9%,2021 年占 72.1%。在接受预防性治疗的患者中,2019 年的年化出血率为 2.2%(± 3.1),2020 年为 1.8%(± 3.0),2021 年为 1.8%(± 2.9)。就浓缩因子 IX 的剂量而言,接受预防性治疗的患者在 2019 年平均接受 41.6(± 11.9)IU/Kg/注射,2020 年平均接受 45.7(± 12.9)IU/Kg/注射,2021 年平均接受 60.1(± 24.0)IU/Kg/注射:在临床上,预防性治疗比报告的更为普遍。结论:临床上,预防性治疗比报告的更为普遍。根据从当前临床证据中获得的见解,预计可以确定患者未满足的医疗需求,医生可以评估患者的状况,并采用更有效的治疗策略积极管理 B 型血友病。
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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 Q2 HEMATOLOGY Pub Date : 2024-08-07 DOI: 10.1007/s44313-024-00030-w
Jeong Suk Koh, Ik-Chan Song

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.

由于抗癌化疗、放射治疗和炎症反应,癌症患者经常会出现贫血。这通常会导致功能性缺铁,其特点是铁储存充足,但利用铁制造红细胞的能力受损。这种情况被称为功能性缺铁性贫血(IDA),铁蛋白水平为 30-500 µg/dL,转铁蛋白饱和度为 0.5%。
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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 Q2 HEMATOLOGY Pub Date : 2024-08-02 DOI: 10.1007/s44313-024-00029-3
Ho-Young Yhim

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.

癌症相关静脉血栓栓塞症(VTE)严重影响发病率和死亡率。过去十年中,直接口服抗凝剂的引入彻底改变了活动性癌症患者的 VTE 治疗,与传统疗法相比具有潜在优势。然而,抗凝剂的最佳选择和剂量仍存在不确定性,尤其是对于具有特定出血风险因素的患者,如某些癌症类型(如上消化道癌症、泌尿生殖系统癌症、原发性或转移性脑肿瘤以及血液系统恶性肿瘤)和特定患者特征(如肾功能障碍和血小板减少症)。有关低血栓负荷 VTE(如亚段肺栓塞和孤立的远端深静脉血栓)相关血栓风险的最新数据强调了在日常临床实践中更新管理策略的必要性。本综述旨在探讨这些问题,并强调癌症相关 VTE 管理的演变情况。
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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 Q2 HEMATOLOGY Pub Date : 2024-07-17 DOI: 10.1007/s44313-024-00025-7
Cheong Yoon Huh, Sung-Soo Park, Jung Yeon Lee, Chang-Ki Min
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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 Q2 HEMATOLOGY Pub Date : 2024-07-09 DOI: 10.1007/s44313-024-00026-6
Seug Yun Yoon, Sung-Eun Lee
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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 Q2 HEMATOLOGY 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

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.

组织细胞瘤是涉及巨噬细胞、树突状细胞和单核细胞的罕见疾病。它们包括朗格汉斯细胞组织细胞增生症(Langerhans cell histiocytosis,LCH)、埃尔德海姆-切斯特病(Erdheim-Chester disease,ECD)、罗赛-多夫曼病(Rosai-Dorfman disease,RDD)、幼年黄原细胞瘤(Juvenile xanthogranuloma,JXG)和组织细胞肉瘤。组织细胞肿瘤的临床过程和预后各不相同,因此需要对其分类、流行病学和临床表现有细致的了解。遗传学研究发现,组织细胞瘤的体细胞突变主要发生在 MAPK 通路上,这表明组织细胞瘤具有克隆性。本综述涵盖目前对组织细胞瘤、分子病理生理学的认识,尤其侧重于 BRAF、MAP2K1 和 PI3K-AKT 信号通路等基因的突变,以及不断发展的治疗策略,尤其侧重于 LCH、ECD、RDD 和 JXG。随着靶向疗法的发展,治疗格局也在不断变化。BRAF抑制剂,如维莫非尼(vemurafenib)和达拉菲尼(dabrafenib),已显示出疗效,尤其是在高风险LCH病例中;然而,挑战依然存在,包括治疗中断后的复发和不良反应。MEK抑制剂也显示出了疗效,而科比米替尼(cobimetinib)最近已被批准用于成人患者。确定最佳治疗时间和管理治疗中断的策略还需要进一步的研究。分子遗传学和靶向疗法的进步彻底改变了组织细胞肿瘤的治疗。然而,持续的研究对于优化患者的治疗效果至关重要。
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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 Q2 HEMATOLOGY Pub Date : 2024-06-07 DOI: 10.1007/s44313-024-00021-x
Ka-Won Kang

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.

手术患者面临术后并发症和死亡的风险,因此有必要通过识别和纠正可改变的风险因素来优化患者的术前治疗。虽然术前输注血小板的目的是降低出血风险,但其疗效仍不确定。同样,为贫血患者输注红细胞也不能降低术后死亡风险,反而可能加重并发症。因此,制定以纠正术前全血细胞计数异常和尽量减少输血需求为重点的个体化策略至关重要。本综述旨在研究全血细胞计数异常和适当的输血策略,以尽量减少术后并发症。
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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 Q2 HEMATOLOGY 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":"59 1","pages":"20"},"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 Q2 HEMATOLOGY 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

Background: 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).

Methods: 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.

Results: 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.

Conclusion: 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.

背景:血友病 A(HA)是一种 X 连锁遗传性出血性疾病,由第八因子(FVIII)水平降低引起。大约 10-15% 的重度 HA(SHA)患者不会出现预期的出血模式。在此,我们使用旋转血栓弹力测定法(ROTEM)和活化部分凝血活酶时间-血栓波形分析法(APTT-CWA)评估了血友病 A 的表型严重程度:方法:招募确诊为 A 型血友病的患者。临床表型分配是根据已发表的文献进行的,患者被分为四个表型亚组。首先在 INTEM 模式下使用贫血小板血浆在 ROTEM 上检测全血样本,然后检测 APTT,并同时记录 APTT-CWA 图:本研究共招募了 66 名患者。使用 ROTEM 和 APTT-CWA 在四个表型分类组之间观察到了明显的统计学差异。将轻度/中度至重度表型患者(II 组)与无抑制剂的 SHA 患者(IV 组)进行比较,发现 ROTEM 或 APTT-CWA 的所有参数均无明显差异。使用 ROTEM 的 MCF、MA30、MAXV 和 Alpha 角值在使用抑制剂的 SHA 患者中最低,这有助于将他们与未使用抑制剂的 SHA 患者区分开来。然而,使用 APTT-CWA 参数却无法区分这两组患者:结论:ROTEM 可用于区分有抑制剂的 SHA 患者和无抑制剂的 SHA 患者,其参数组合具有较高的灵敏度和特异性。然而,APTT-CWA 并不能用于区分这两类患者。
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引用次数: 0
期刊
Blood Research
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