Stem cell transplantation

干细胞移植
  • 文章类型: Journal Article
    尤文肉瘤(ES)是骨和软组织的恶性肿瘤,最常见于儿童,青少年,和年轻人。复发/难治性ES(RR-ES)的管理仍存在争议和争议。作者利用了国家尤因肉瘤肿瘤委员会的专业知识,多学科虚拟肿瘤委员会,每月开会讨论具有挑战性的ES病例。在这次审查中,他们专注于选择适用于RR-ES患者管理的主题.涵盖的具体主题包括此类患者的初始方法和对护理目标的讨论,分子检测的作用,要考虑的化疗方案和新药物,维持治疗的作用,以及使用大剂量化疗和自体干细胞抢救。引用的数据通常限于子组分析和/或从多个源编译。虽然不打算取代治疗医生的临床判断,这些指南旨在为临床医师提供支持,并为RR-ES患者的治疗提供一些明确的建议.尤因肉瘤(ES)是一种骨和软组织癌,最常见于青少年和年轻人。本文利用国家尤因肉瘤肿瘤委员会的经验,一个多机构,多学科虚拟肿瘤委员会每月开会,讨论具有挑战性的ES病例,并解决与复发性ES患者治疗相关的问题。虽然不打算取代治疗医生的临床判断,并且受现有数据的限制,这些共识建议将支持治疗这种具有挑战性的恶性肿瘤患者的临床医生,当它复发时变得更加困难。
    Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue that most often occurs in children, adolescents, and young adults. Debate and controversy remain in the management of relapsed/refractory ES (RR-ES). The authors leveraged the expertise assembled by the National Ewing Sarcoma Tumor Board, a multidisciplinary virtual tumor board that meets monthly to discuss challenging cases of ES. In this review, they focus on select topics that apply to the management of patients with RR-ES. The specific topics covered include the initial approach of such patients and discussion of the goals of care, the role of molecular testing, chemotherapy regimens and novel agents to consider, the role of maintenance therapy, and the use of high-dose chemotherapy with autologous stem cell rescue. The data referenced are often limited to subgroup analyses and/or compiled from multiple sources. Although not intended to replace the clinical judgement of treating physicians, these guidelines are intended to support clinicians and provide some clarity and recommendations for the management of patients with RR-ES. PLAIN LANGUAGE SUMMARY: Ewing sarcoma (ES) is a bone and soft tissue cancer that most often occurs in teenagers and young adults. This article uses the experience of the National Ewing Sarcoma Tumor Board, a multi-institution, multidisciplinary virtual tumor board that meets monthly to discuss challenging cases of ES and to address questions related to the treatment of patients with relapsed ES. Although not intended to replace the clinical judgement of treating physicians and limited by available data, these consensus recommendations will support clinicians who treat patients with this challenging malignancy, made even more difficult when it recurs.
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  • 文章类型: Journal Article
    巨细胞病毒(CMV)感染和疾病是移植受体发病和死亡的重要原因。为了在临床试验中开发CMV结局的一致报告,制定了CMV感染和疾病的定义,最近一次于2017年发表。从那以后,有了重大发展,包括新的抗病毒药物的注册。因此,移植相关病毒感染论坛,由科学家组成,临床医生,监管者,和行业代表,已经产生了这些定义的更新版本,结合了最近的知识,目的是支持临床研究和药物开发。这还包括有关先前于2019年发布的耐药性和难治性CMV感染定义的更新。随着田野的发展,这些定义的更新的需要是明确的,和临床医生之间的合作努力,科学家,监管者,行业代表可以为这项工作提供平台。
    Cytomegalovirus (CMV) infection and disease are important causes of morbidity and mortality in transplant recipients. For the purpose of developing consistent reporting of CMV outcomes in clinical trials, definitions of CMV infection and disease were developed and most recently published in 2017. Since then, there have been major developments, including registration of new antiviral agents. Therefore, the Transplant Associated Virus Infections Forum, which consists of scientists, clinicians, regulators, and industry representatives, has produced an updated version of these definitions that incorporates recent knowledge with the aim of supporting clinical research and drug development. This also includes an update regarding the definition of resistant and refractory CMV infections previously published in 2019. As the field evolves, the need for updates of these definitions is clear, and collaborative efforts among clinicians, scientists, regulators, and industry representatives can provide a platform for this work.
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  • 文章类型: Journal Article
    AL淀粉样变性是最常见的全身性淀粉样变性形式。然而,出现症状的非特异性性质要求需要加强临床怀疑,以在适当的临床环境中检测无法解释的表现.早期检测和治疗至关重要,因为心脏受累程度是AL淀粉样变性患者生存的主要预后预测指标。在用适当的组织活检诊断AL淀粉样变性后,用硼替佐米迅速治疗,在有或没有达雷妥单抗的情况下,应开始基于环磷酰胺和地塞米松的一线诱导.治疗的目标是实现可能的最佳血液学反应,理想情况下涉及游离轻链<20mg/L,因为它提供了器官功能改善的最佳机会。如果患者在2个治疗周期内没有达到部分反应,或者在4个周期或自体干细胞移植后没有达到非常好的部分反应,则应改变治疗方法。作为实现深刻和长期的克隆反应转化为更好的器官反应和长期结果。早期多学科专家如肾内科医师的参与,心脏病学家,神经学家,建议胃肠病学家对受累器官进行最佳维护和支持,以对AL淀粉样变性患者进行最佳管理。
    AL amyloidosis is the most common form of systemic amyloidosis. However, the non-specific nature of presenting symptoms requires the need for a heightened clinical suspicion to detect unexplained manifestations in the appropriate clinical setting. Early detection and treatment are crucial as the degree of cardiac involvement emerges as a primary prognostic predictor of survival in a patient with AL amyloidosis. Following the diagnosis of AL amyloidosis with appropriate tissue biopsies, prompt treatment with a bortezomib, cyclophosphamide and dexamethasone-based first-line induction with or without daratumumab should be initiated. The goal of treatment is to achieve the best haematologic response possible, ideally with involved free light chain <20 mg/L, as it offers the best chance of organ function improvement. Treatment should be changed if patients do not achieve a partial response within 2 cycles of treatment or very good partial response after 4 cycles or after autologous stem cell transplant, as achievement of profound and prolonged clonal responses translates to better organ response and long-term outcomes. Early involvement of multidisciplinary subspecialists such as renal physicians, cardiologists, neurologists, and gastroenterologists for optimal maintenance and support of involved organs is recommended for optimal management of patients with AL amyloidosis.
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  • 文章类型: Journal Article
    背景:许多儿童接受异基因造血干细胞移植(HSCT)治疗恶性和非恶性疾病。不幸的是,肺部并发症经常发生在HSCT后,闭塞性细支气管炎综合征(BOS)是最常见的非感染性肺部并发症。当前的国际准则包含有关BOS的HSCT后监测的相互矛盾的建议,最近的美国国立卫生研究院研讨会强调了对HSCT后监测采取标准化方法的必要性。因此,本指南为儿童HSCT后BOS的检测提供了基于证据的方法.
    方法:一家跨国公司,多学科专家小组确定了关于监测的六个问题,儿童HSCT后BOS的评估。对文献进行了系统的回顾,以回答每个问题。建议的分级,评估,发展,采用评估方法对证据质量和建议强度进行评分。
    结果:小组成员考虑了每个建议的强度,并评估了应用干预措施的收益和风险。在制定建议时,小组考虑了患者和护理人员的价值观,护理的费用,和可行性。提出了有关筛查肺功能测试和诊断测试在疑似HSCT后BOS儿童中的作用的建议。在Delphi过程之后,还提出了儿科HSCT后BOS的新诊断标准.
    结论:本文件提供了一种基于证据的方法来检测儿童HSCT后BOS,同时还强调了执行每一项建议的考虑因素。Further,该文件描述了未来研究的重要领域。
    Background: Many children undergo allogeneic hematopoietic stem cell transplantation (HSCT) for the treatment of malignant and nonmalignant conditions. Unfortunately, pulmonary complications occur frequently post-HSCT, with bronchiolitis obliterans syndrome (BOS) being the most common noninfectious pulmonary complication. Current international guidelines contain conflicting recommendations regarding post-HSCT surveillance for BOS, and a recent NIH workshop highlighted the need for a standardized approach to post-HSCT monitoring. As such, this guideline provides an evidence-based approach to detection of post-HSCT BOS in children. Methods: A multinational, multidisciplinary panel of experts identified six questions regarding surveillance for, and evaluation of, post-HSCT BOS in children. A systematic review of the literature was undertaken to answer each question. The Grading of Recommendations, Assessment, Development, and Evaluation approach was used to rate the quality of evidence and the strength of recommendations. Results: The panel members considered the strength of each recommendation and evaluated the benefits and risks of applying the intervention. In formulating the recommendations, the panel considered patient and caregiver values, the cost of care, and feasibility. Recommendations addressing the role of screening pulmonary function testing and diagnostic tests in children with suspected post-HSCT BOS were made. Following a Delphi process, new diagnostic criteria for pediatric post-HSCT BOS were also proposed. Conclusions: This document provides an evidence-based approach to the detection of post-HSCT BOS in children while also highlighting considerations for the implementation of each recommendation. Further, the document describes important areas for future research.
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  • 文章类型: Journal Article
    近年来,马来西亚已经看到针对各种医疗条件的干细胞疗法激增。然而,马来西亚对干细胞研究和治疗的监管面临着一些挑战,例如不受监管的诊所的出现和缺乏具体的立法.一些紧急措施,包括制定具体法律,加强监测,以及提高公众意识和教育,是至关重要的。因此,干细胞治疗监管需要决策者的共同努力,监管机构和医疗保健专业人员。本评论讨论了马来西亚干细胞治疗法规的当前指南和挑战,并提出了一些未来的建议,可以为全球干细胞研究和治疗的负责任进展铺平道路。
    In recent years, Malaysia has seen a surge in stem cell therapy for various medical conditions. However, the regulation of stem cell research and therapy in Malaysia faces several challenges such as the emergence of unregulated clinics and a lack of specific legislation. Some urgent measures, including enactment of specific laws, strengthened monitoring, as well as increased public awareness and education, are crucial. Therefore, stem cell therapy regulation requires concerted efforts by the policymakers, regulator bodies and healthcare professionals. This commentary discusses the current guidelines and challenges in Malaysian stem cell therapy regulation and proposes some future recommendations that could pave the way for responsible progress of stem cell research and therapy globally.
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  • 文章类型: Journal Article
    骨髓细胞过少的全血细胞减少症是再生障碍性贫血(AA)的标志,并在仔细评估后确认诊断。在排除包括增生性骨髓增生异常综合征在内的替代诊断后。分子细胞基因组学的新兴应用有助于从遗传性骨髓衰竭(IBMF)中描绘免疫介导的AA。Camitta标准用于评估疾病的严重程度,随着年龄和人类白细胞抗原相容性供体的可用性是决定治疗决策的决定因素。血液和血小板输注支持的支持性护理,在整个疾病过程中,抗微生物预防和机会性感染的及时管理仍然是关键。新诊断的获得性严重/非常严重的AA患者的标准一线治疗是马抗胸腺细胞球蛋白和基于环孢素的免疫抑制治疗(IST),并使用eltrombopag或来自匹配的同胞供体的同种异体造血干细胞移植(HSCT)。在对IST缺乏反应后,应考虑成人的无关供体HSCT,并预先为患有严重感染的年轻人和现成的匹配的无关供体提供服务。IBMF的管理,怀孕和老年人的AA需要特别注意。鉴于AA的稀有性和管理的复杂性,强烈建议在多学科会议中进行适当讨论,并让专家中心参与,以改善患者预后.
    Pancytopenia with hypocellular bone marrow is the hallmark of aplastic anaemia (AA) and the diagnosis is confirmed after careful evaluation, following exclusion of alternate diagnosis including hypoplastic myelodysplastic syndromes. Emerging use of molecular cyto-genomics is helpful in delineating immune mediated AA from inherited bone marrow failures (IBMF). Camitta criteria is used to assess disease severity, which along with age and availability of human leucocyte antigen compatible donor are determinants for therapeutic decisions. Supportive care with blood and platelet transfusion support, along with anti-microbial prophylaxis and prompt management of opportunistic infections remain key throughout the disease course. The standard first-line treatment for newly diagnosed acquired severe/very severe AA patients is horse anti-thymocyte globulin and ciclosporin-based immunosuppressive therapy (IST) with eltrombopag or allogeneic haemopoietic stem cell transplant (HSCT) from a matched sibling donor. Unrelated donor HSCT in adults should be considered after lack of response to IST, and up front for young adults with severe infections and a readily available matched unrelated donor. Management of IBMF, AA in pregnancy and in elderly require special attention. In view of the rarity of AA and complexity of management, appropriate discussion in multidisciplinary meetings and involvement of expert centres is strongly recommended to improve patient outcomes.
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  • 文章类型: Journal Article
    尤因肉瘤(ES)是骨和软组织的恶性肿瘤,最常见于青少年和年轻人。尽管采取了国际协调的方法,几个细微差别,差异,在确定治疗ES的护理标准方面仍存在争议。在这次审查中,作者利用了国家尤因肉瘤肿瘤委员会组建而成的专业知识,一个多机构,多学科虚拟肿瘤委员会每月开会讨论ES的复杂和具有挑战性的病例。本报告的重点是适用于新诊断的ES患者管理的选定主题。涵盖的特定主题包括与氟脱氧葡萄糖-正电子发射断层扫描相比,用于初步评估的骨髓穿刺和活检的适应症,间隔压缩化疗在18岁及以上患者中的作用,转移性疾病患者在长春新碱/多柔比星/环磷酰胺中添加异环磷酰胺/依托泊苷的作用,大剂量化疗自体干细胞移植的数据和作用,维持治疗,和全肺照射。引用的数据通常限于子组分析和/或从多个源编译。虽然不打算取代治疗医生的临床判断,本指南旨在为ES患者的前期管理提供清晰度和建议.尤文肉瘤是一种骨和软组织恶性肿瘤,最常见于青少年和年轻人。对于这篇评论,作者使用了国家尤因肉瘤肿瘤委员会的经验,一个多机构,多学科虚拟肿瘤委员会每月开会讨论尤因肉瘤的复杂和具有挑战性的病例。虽然不打算取代治疗医生的临床判断,该指南的重点是为尤因肉瘤患者的前期治疗制定共识声明.
    Ewing sarcoma (ES) is a malignant tumor of bone and soft tissue that most often occurs in adolescents and young adults. Despite an international coordinated approach, several nuances, discrepancies, and debates remain in defining the standard of care for treating ES. In this review, the authors leverage the expertise assembled by formation of the National Ewing Sarcoma Tumor Board, a multi-institution, multidisciplinary virtual tumor board that meets monthly to discuss complicated and challenging cases of ES. This report is focused on select topics that apply to the management of patients with newly diagnosed ES. The specific topics covered include indications for bone marrow aspirate and biopsy for initial evaluation compared with fluorodeoxyglucose-positron emission tomography, the role of interval compressed chemotherapy in patients aged 18 years and older, the role of adding ifosfamide/etoposide to vincristine/doxorubicin/cyclophosphamide for patients with metastatic disease, the data on and role of high-dose chemotherapy with autologous stem cell transplantation, maintenance therapy, and whole-lung irradiation. The data referenced are often limited to subgroup analyses and/or compiled from multiple sources. Although not intended to replace the clinical judgement of treating physicians, the guidelines are intended to provide clarity and recommendations for the upfront management of patients with ES. PLAIN LANGUAGE SUMMARY: Ewing sarcoma is a malignant tumor of bone and soft tissue that most often occurs in adolescents and young adults. For this review, the authors used the experience of the National Ewing Sarcoma Tumor Board, a multi-institution, multidisciplinary virtual tumor board that meets monthly to discuss complicated and challenging cases of Ewing sarcoma. Although not intended to replace the clinical judgement of treating physicians, the guidelines will focus on the development of consensus statements for the upfront management of patients with Ewing sarcoma.
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  • 文章类型: English Abstract
    Cardiovascular diseases have been the leading cause of death in both urban and rural residents. Among them, heart failure, which develops from various heart diseases to the end stage, is the main cause of death. With the development of regenerative medicine, stem cell transplantation is expected to be a potential and promising treatment for heart failure. In recent years, basic research and clinical application research related to stem cells have been vigorously developed in China, and many latest achievements and progress have been obtained. However, relevant guidance documents are still needed to standardize and scale up clinical applications of stem cell transplantation. Therefore, experts from the Tissue Repair and Regeneration Branch of the Chinese Medical Association, by referring to the latest research results, discussed the treatment of heart failure by autologous stem cell transplantation and reached the following consensus. The key technical issues related to autologous stem cell transplantation were mainly expounded, and scientific suggestions were proposed to standardize and promote the clinical research and application of stem cells.
    心血管病死亡已占城乡居民总死亡原因的首位,其中各类心脏病发展到终末期的心力衰竭是死亡的主要原因。随着再生医学的发展,干细胞移植有望成为治疗心力衰竭较具潜力和应用前景的治疗方法。近年来,干细胞相关基础研究及临床应用研究在国内蓬勃开展,获得了许多最新的成果与进展,但干细胞移植在临床上规范化、规模化开展仍需要相关指导性文件。因此,中华医学会组织修复与再生分会组织专家,结合当前最新研究结果,对自体干细胞移植治疗心力衰竭进行会议讨论并达成以下共识,重点对自体干细胞移植过程中的相关关键技术问题进行阐述,提出了科学建议,以期规范和促进干细胞的临床研究及应用。.
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  • 文章类型: Journal Article
    与动物接触的儿科患者有很多好处,但是感染人畜共患病的风险,即使低,必须由临床医生考虑。为了评估对这个主题的认识,我们调查了意大利儿科肿瘤学中心,这导致了不同的反应。意大利儿科血液学-肿瘤学协会的传染病工作组和护士工作组,与意大利兽医全国联合会的兽医一起,起草了一份共识文件,以统一给予家庭的适应症,目的是保证患者和动物之间的安全互动,并改善临床医生与兽医和家庭的合作。
    Contact with animals in pediatric oncohematologic patients is associated with many benefits, but the risk of contracting zoonoses, even if low, must be considered by clinicians. In order to assess the awareness about this topic, we surveyed the Italian pediatric oncohematology centers, which resulted in heterogeneous responses. The Infectious Diseases Working Group and the Nurse Working Group of the Italian Association of Pediatric Hematology-Oncology, together with veterinarians from the National Federation of Italian Veterinarians, drew up a consensus document to unify the indications to be given to families with the aim of guaranteeing a safe interaction between patients and animals and improving the collaboration of clinicians with veterinarians and families.
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  • 文章类型: Practice Guideline
    Shwachman-Diamond综合征是一种罕见的疾病,可发展为恶性和非恶性血液并发症。总的来说,10%至20%的Shwachman-Diamond患者需要造血干细胞移植(HSCT),但是大多数中心都有有限的经验和不同的方法。欧洲血液和骨髓移植协会-重度再生障碍性贫血工作组促进了专家共识,就患有血液学并发症的Shwachman-Diamond患者的管理中的关键问题提出了建议。确定与提高生存率相关的主要项目是:定期和结构化血液学随访的重要性,通过使用降低强度的预处理方案可能降低移植相关的死亡率,全身照射的局限性,特别是对于非恶性严重的血细胞减少症/骨髓衰竭,克隆恶性演变的早期诊断和HSCT适应症的早期识别。最后,急性髓系白血病患者的HSCT效果不佳,不考虑移植前接受的细胞减灭性化疗治疗,强调了创新方法的必要性。©2023美国血液和骨髓移植协会。由ElsevierInc.发布。保留所有权利。
    Shwachman-Diamond syndrome is a rare disorder that can develop malignant and nonmalignant hematological complications. Overall, 10% to 20% of Shwachman-Diamond patients need hematopoietic stem cell transplantation (HSCT), but most centers have a limited experience and different approaches. The European Society for Blood and Marrow Transplantation-Severe Aplastic Anaemia Working Party promoted an expert consensus to propose recommendations regarding key issues in the management of Shwachman-Diamond patients with hematological complications. The main items identified as relevant for improving survival were: the importance of regular and structured hematologic follow-up, the potential reduction of transplant-related mortality by using reduced-intensity conditioning regimens, the limitation of total body irradiation, particularly for non-malignant severe cytopenia/bone marrow failure, the early diagnosis of clonal malignant evolution and early recognition of an indication for HSCT. Finally, the poor results of HSCT in patients with acute myeloid leukemia, irrespective of cytoreductive chemotherapy treatment received prior to transplantation, highlights the need for innovative approaches. © 2023 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
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