• 文章类型: Journal Article
    多发性骨髓瘤仍然是一种不治之症,尽管开发了许多药物类别和组合,有助于改善总体生存率。针对癌细胞表面抗原的免疫疗法,如嵌合抗原受体(CAR)T细胞疗法和T细胞重定向双特异性抗体,最近获得了监管部门的批准,并显示出前所未有的效力。然而,这些免疫疗法具有独特的作用机制和毒性,与以前的骨髓瘤治疗方法不同,因此,临床试验和早期访问计划的经验对于为患者管理提供具体建议至关重要,特别是当这些代理商在世界许多地方都可用时。这里,我们为使用双特异性抗体治疗骨髓瘤提供专家共识的临床实践指南.国际骨髓瘤工作组还参与收集接受此类免疫疗法治疗的患者的预期实时数据,目的是不断学习并调整临床实践,以优化接受免疫疗法的患者的管理。
    Multiple myeloma remains an incurable disease, despite the development of numerous drug classes and combinations that have contributed to improved overall survival. Immunotherapies directed against cancer cell-surface antigens, such as chimeric antigen receptor (CAR) T-cell therapy and T-cell-redirecting bispecific antibodies, have recently received regulatory approvals and shown unprecedented efficacy. However, these immunotherapies have unique mechanisms of action and toxicities that are different to previous treatments for myeloma, so experiences from clinical trials and early access programmes are essential for providing specific recommendations for management of patients, especially as these agents become available across many parts of the world. Here, we provide expert consensus clinical practice guidelines for the use of bispecific antibodies for the treatment of myeloma. The International Myeloma Working Group is also involved in the collection of prospective real-time data of patients treated with such immunotherapies, with the aim of learning continuously and adapting clinical practices to optimise the management of patients receiving immunotherapies.
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  • 文章类型: Journal Article
    静脉血栓栓塞(VTE)在多发性骨髓瘤的背景下提出了重大挑战,在新诊断的患者中,发病率高达10%,在复发/难治的情况下,发病率不同。准确的VTE风险评估和个性化的血栓预防策略是骨髓瘤支持治疗的重要组成部分。有三种经过验证的风险评估模型用于预测新诊断的骨髓瘤-SAVED的VTE风险。IMPEDE-VTE,PRISM。在这次审查中,我们深入研究了VTE风险预测模型在当前治疗中的实际应用.通过强调定制方法的必要性,我们强调了考虑患者特异性的重要性,疾病特异性,以及每种临床情景中特定治疗的风险因素,并使用这些数据来补充风险评估模型的输出。我们还提供了关于骨髓瘤VTE血栓预防的现有数据的摘要。并强调应强烈考虑直接口服抗凝剂的具体情况。我们的目标是通过分析特定患者病例来填补VTE预防和管理方面的关键空白,并为临床医生提供实用概述。
    Venous thromboembolism (VTE) poses a significant challenge in the context of multiple myeloma, with an incidence of up to 10% in newly diagnosed patients and varying frequency in the relapsed/refractory setting. Accurate VTE risk assessment and personalized thromboprophylaxis strategies are important parts of supportive care in myeloma. There are three validated risk assessment models for prediction of VTE risk in newly diagnosed myeloma-SAVED, IMPEDE-VTE, and PRISM. In this review, we delve into the practical applications of VTE risk prediction models in the context of current therapies. By emphasizing the necessity of a tailored approach, we underscore the importance of considering patient-specific, disease-specific, and treatment-specific risk factors in each clinical scenario, and using that data to complement the output from risk assessment models. We also provide a summary of currently available data on VTE thromboprophylaxis in myeloma, and highlight specific situations where direct oral anticoagulants should be strongly considered. Our objective is to fill the critical gaps in VTE prophylaxis and management through the analysis of specific patient cases and provide a practical overview for clinicians.
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  • 文章类型: English Abstract
    Renal impairment is a common complication of multiple myeloma (MM). All patients with MM should be assessed for the presence and severity of renal impairment. The clinicopathological manifestations of MM-related renal impairment are diverse and complex; accordingly, except for light-chain nephropathy, which can often be diagnosed without biopsy based solely on clinical criteria, a renal biopsy is needed for an accurate diagnosis. Supportive care, such as adequate hydration, is required for all patients with MM-related renal impairment. The guideline provide the principles for dose adjustment of the drugs used for MM with renal impairment, including proteasome inhibitors, immunomodulators, monoclonal antibodies, small molecule inhibitors, and alkylating agents, as well as those used for myeloma bone disease. Autologous stem cell transplantation (ASCT) and chimeric antigen receptor T-cell immunotherapy (CAR-T) are effective in patients with moderate renal impairment and are tolerated by the patients. The Chinese Hematology Association; the Chinese Geriatrics Association, Society of Hematology; and the Chinese Research Hospital Association, Society of Nephrology asked experts to collate information on current progress in clinical research relating to MM with renal impairment. This guideline was developed based on the gathered data combined with the latest international consensus and clinical practice guidelines.
    肾损伤是多发性骨髓瘤(MM)的常见合并症。所有MM患者均应评估是否存在肾损伤及其严重程度。MM肾损伤临床病理表现复杂多样,轻链管型肾病在临床表现典型时一般不需肾活检即可诊断,余患者应尽可能行肾活检明确诊断。所有MM肾损伤患者应尽早给予水化等支持治疗。指南详细列出了治疗MM的主要药物在肾损害时的减量原则,包括蛋白酶体抑制剂、免疫调节剂、单克隆抗体、小分子抑制剂、烷化剂、抗骨病治疗药物等。自体造血干细胞移植(ASCT)及嵌合抗原受体T细胞免疫疗法(CAR-T)在中度肾损伤患者有较好的耐受性及有效性。中国医师协会血液科医师分会、中国老年医学学会血液学分会及中国研究型医院学会肾脏病学专委会组织专家检索MM合并肾损伤相关的最新临床研究进展,并结合国外最新指南、共识及临床实践制订了本指南。.
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  • 文章类型: English Abstract
    Extramedullary plasmacytoma (EMP) is a special type of malignant plasmacytosis, which is complex and heterogeneous. Most EMP patients have poor prognosis and lack a stratified prognostic system or ideal treatment strategy supported by evidence-based medical evidence, which cannot meet clinical needs. In order to improve the understanding of this disease entity, Plasma Cell Disease Group, Chinese Society of Hematology, Chinese Medical Association and Chinese Myeloma Committee-Chinese Hematology Association developed the \"Chinese Expert Consensus on the diagnosis and treatment of extramedullary plasmacytoma\", which aims to standardize the clinical diagnosis and treatment of EMP and ultimately improve the overall survival of patients with plasmacytoma.
    髓外浆细胞瘤(extramedullary plasmacytoma, EMP)是恶性浆细胞病的一种特殊类型,表现复杂、异质性强。大部分EMP预后差,缺乏循证医学证据支持下的预后分层系统和理想的治疗策略,无法满足临床需求。为提高对这类疾病的认识,中华医学会血液学分会浆细胞疾病学组和中国医师协会多发性骨髓瘤专业委员会编写《中国髓外浆细胞瘤诊断与治疗专家共识》,旨在规范EMP的临床诊治,最终改善浆细胞瘤患者的总体生存。.
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  • 文章类型: Journal Article
    转移性肱骨病的治疗是基于对已发表的关于转移性疾病治疗的研究的系统评价。多发性骨髓瘤,淋巴瘤仅限于肱骨。该指南包含七个行动声明,以协助整形外科医生,骨科肿瘤学家,医师,以及任何其他参与肱骨转移性疾病手术管理的合格医疗保健专业人员。它还旨在作为决策者的信息资源,研究人员,和临床实践指南的开发者。除了提供务实的实践建议外,该指南还突出了文献中的差距,并为未来的研究和质量测量开发提供了信息。该指南已得到美国骨科医师学会的认可。
    Management of Metastatic Humeral Disease is based on a systematic review of published studies surrounding the management of metastatic disease, multiple myeloma, and lymphoma limited to the humerus. This guideline contains seven action statements to assist orthopaedic surgeons, orthopaedic oncologists, physicians, and any other qualified healthcare professionals involved in the surgical management of metastatic disease of the humerus. It is also intended to serve as an information resource for decision makers, researchers, and developers of clinical practice guidelines. In addition to providing pragmatic practice recommendations, this guideline also highlights gaps in the literature and informs areas for future research and quality measure development. This guideline has been endorsed by the American Academy of Orthopaedic Surgeons.
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  • 文章类型: Journal Article
    暂无摘要。
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  • 文章类型: Journal Article
    治疗进展大大提高了生存率,但是骨髓瘤是所有延迟诊断最严重的癌症之一,导致严重的疾病和早期死亡。这种延迟主要是因为骨髓瘤的症状特征非常低,在初级保健中,骨髓瘤是罕见的。然而,开始诊断的旅程只需要考虑骨髓瘤和送血来检测单克隆免疫球蛋白。实验室测试可靠地检测单克隆免疫球蛋白,这在99%的骨髓瘤病例中都存在,那么,为什么医疗保健系统会出现延迟诊断的问题?英国骨髓瘤早期诊断计划汇集了各种专业知识来调查这个问题,本文是由该计划的实验室最佳实践工作组编写的。它审查测试请求的证据,分析和报告,英国各地的实践差异很大。它提出了一个“GP骨髓瘤诊断工具”,以及如何将其集成到实验室实践中以及实验室最佳实践工具中。它建议改进请求并与血液学服务相结合,以进行报告和解释。在这里,实验室在为骨髓瘤诊断提供适当和及时的骨髓检查的有效和具有成本效益的途径方面发挥着核心作用。
    Treatment advances have greatly improved survival, but myeloma is among the worst of all cancers for delayed diagnosis, causing serious morbidities and early deaths. This delay is largely because the symptom profile of myeloma has very low specificity, and in primary care, myeloma is rare. However, initiating the journey to diagnosis simply requires considering myeloma and sending blood to test for monoclonal immunoglobulin. Laboratory tests reliably detect monoclonal immunoglobulin, which is present in 99% of myeloma cases, so why do health care systems have such a problem with delayed diagnosis? The Myeloma UK early diagnosis programme has brought together diverse expertise to investigate this problem, and this article was prepared by the programme\'s working group for laboratory best practice. It reviews evidence for test requesting, analysis and reporting, for which there is large variation in practice across the United Kingdom. It presents a \'GP Myeloma diagnostic tool\' and how it can be integrated into laboratory practice alongside a laboratory best practice tool. It proposes improved requesting and integration with haematology services for reporting and interpretation. Here the laboratory has a central role in creating efficient and cost-effective pathways for appropriate and timely bone marrow examination for myeloma diagnosis.
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  • 文章类型: Guideline
    在个性化医疗时代,越来越需要评估患者报告的结果(PRO),以成为患者护理的标准。患者报告的结果指标(PROM)对于评估基于患者自身观点的干预结果的重大且有意义的变化非常重要。公认的是,活动性多发性骨髓瘤(MM)的特征是疾病和治疗相关症状的高负担。生活质量(QoL)显著恶化。总的来说,在过去的十年里,重点已转移到获得最持久的缓解和最佳的QoL作为MM治疗的主要目标。患者对他们的QoL有相当大的价值,并且在治疗决策之前就QoL数据进行沟通,使他们能够做出明智的治疗选择。因此,优化MM患者的QoL是一个重要的治疗目标,将PRO纳入临床试验有可能改善治疗结果.在这方面,在临床试验中MM中使用和报告PROM的指导是必要的。在欧洲血液学协会的主持下,根据EHA的核心指南制定方法学,制定了MM患者使用和报告PRO的循证指南.本文件提供了MM临床试验中PROM选择的一般考虑因素,以及涵盖MM临床试验中PROM选择的一系列建议;给药方式;评估时间;最小化缺失数据的策略;样本量计算;结果报告;和结果解释。
    In the era of personalized medicine there is an increasing need for the assessment of patient-reported outcomes (PROs) to become a standard of patient care. Patient-reported outcome measures (PROM) are important in assessing significant and meaningful changes as a result of an intervention based on a patient\'s own perspective. It is well established that active multiple myeloma (MM) can be characterized by a high burden of disease and treatment-related symptoms, with considerable worsening of quality of life (QoL). In general, and over the past decade, the focus has shifted to obtaining the most durable remissions with the best QoL as primary goals for MM treatment. Patients place considerable value on their QoL and communicating about QoL data prior to treatment decisions allows them to make informed treatment choices. Consequently, optimization of QoL of patients with MM is an important therapeutic goal and the incorporation of PROs into clinical trials has the potential of improving treatment outcomes. In this regard, guidance for the use and reporting of PROMs in MM in clinical trials is warranted. Under the auspices of the European Hematology Association, evidence-based guidelines for the use and reporting of PROs in patients with MM have been developed according to the EHA\'s core Guidelines Development Methodology. This document provides general considerations for the choice of PROMs in MM clinical trials as well as a series of recommendations covering a selection of PROMs in MM clinical trials; the mode of administration; timing of assessments; strategies to minimize missing data; sample size calculation; reporting of results; and interpretation of results.
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  • 文章类型: Journal Article
    复发性/难治性多发性骨髓瘤(MM)的治疗已发展为包括几种新选择。这些包括与第二代蛋白酶体抑制剂(PI)的新组合;第二代免疫调节剂,单克隆抗体,CAR-T细胞,双特异性抗体,selinexor,维尼托克,和许多其他人。大多数MM患者经历了几个周期的缓解和复发,因此需要多种联合疗法。选择复发性/难治性MM的治疗方案需要考虑对特定类别的耐药状态。应考虑患者的特定因素,如年龄和其他合并症。MM的NCCN指南提供了一个框架,在该框架上做出有关工作的决策,治疗,以及新诊断和先前治疗的MM的随访。本手稿概述了NCCN指南中针对复发性/难治性疾病的MM的建议。
    The treatment of relapsed/refractory multiple myeloma (MM) has evolved to include several new options. These include new combinations with second generation proteasome inhibitors (PI); second generation immunomodulators, monoclonal antibodies, CAR T cells, bispecific antibodies, selinexor, venetoclax, and many others. Most patients with MM undergo several cycles of remissions and relapse, and therefore need multiple lines of combination therapies. Selecting treatment options for relapsed/refractory MM requires consideration of resistance status to specific classes, and patient-specific factors such as age and other comorbidities should be considered. The NCCN Guidelines for MM provide a framework on which to base decisions regarding workup, treatment, and follow-up of newly diagnosed and previously treated MM. This manuscript outlines the recommendations from NCCN Guidelines for MM specific to relapsed/refractory disease.
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  • 文章类型: Practice Guideline
    多发性骨髓瘤(MM)的特征是恶性浆细胞(PC)在骨髓中的积累。尽管在治疗方面取得了相当大的进步,MM被认为是一种不可治愈的慢性疾病,预后非常异质,主要取决于复杂性随时间演变的基因组改变。MM的细胞遗传学分析是在CD138+分选的PC上进行的,为了检测以下高危细胞遗传学异常:t(4;14),17p/TP53删除,1q21增益/放大,1p32删除,以及t(11;14),因为它的治疗意义。这个最小的面板可以放大以检测其他复发性异常,根据实验室选择的预后评分。尽管随着分子技术的改进,MM的遗传景观知识正在迅速发展,风险评分仍有待完善,因为它们需要更多的时间进行共识验证。GFCH在此概述了MM和相关PC疾病中与其预后因素相关的基因组学改变。当可用时,以及专家组的建议,以识别和表征这些更改。这项工作是对以前2016年建议的更新。
    Multiple myeloma (MM) is characterized by the accumulation of malignant plasma cells (PCs) in the bone marrow. Despite considerable advances in the treatment, MM is considered an incurable chronic disease with a very heterogeneous prognosis, mostly depending on genomic alterations whose complexity evolves over time. The cytogenetic analysis of MM is performed on CD138+ sorted PCs, in order to detect the following high risk cytogenetic abnormalities: t(4;14), 17p/TP53 deletion, 1q21 gain/amplification, 1p32 deletion, as well as t(11;14) because of its therapeutic implication. This minimal panel can be enlarged to detect other recurrent abnormalities, according to the prognostic score chosen by the laboratory. Although the knowledge of the genetic landscape of MM is evolving rapidly with improved molecular technologies, risk scores remain to be refined as they require more time for consensual validation. The GFCH present here the overview of genomics alterations identified in MM and related PCs diseases associated with their prognostic factor, when available, and recommendations from an expert group for identification and characterization of those alterations. This work is the update of previous 2016 recommendations.
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