Transplantation Conditioning

移植调理
  • 文章类型: Journal Article
    急性脑病,临床表现为谵妄,是造血细胞移植(HCT)的常见但通常未被识别的并发症。在任何年龄的患者中发现谵妄,并在自体或同种异体HCT后观察到。虽然主要在清髓性条件受体的初次移植住院期间进行了研究,最近的HCT调查发现,移植后和接受低强度调节的患者出现谵妄.急性脑病可由感染性并发症引起,药物,组织损伤和/或器官功能障碍。意识改变了,温和或深刻,通常是其唯一的临床表现。识别谵妄对于整体HCT护理至关重要,因为发生谵妄的患者住院时间和恢复时间更长,并且有其他不良HCT后结局的风险。鉴于这种常见并发症的关键性质和HCT扩展到更脆弱的人群,美国移植和细胞治疗学会(ASTCT)建议,现在是时候加强对HCT后认知变化的研究,并为临床护理目的建立涵盖全部意识改变的标准化定义,并为未来的研究提供基准终点.为了捕获一系列特别在HCT患者中发现的急性神经认知变化(通常称为急性脑病),ASTCT提出了新的诊断,移植相关畸形和脑病(TAME)。TAME诊断包括符合DSM-5谵妄标准的HCT患者和不符合完全DSM-5谵妄标准(亚综合征谵妄)的急性神经认知改变患者。早期TAME被定义为发生在HCT后的调节过程中或≤100天,而晚期TAME发生在HCT后>100天,在有其他HCT相关并发症的患者中。本手稿将建立明确的诊断标准,并讨论可能影响TAME发展的因素,以及TAME的工作和管理。
    Acute encephalopathy, manifesting clinically as delirium, is a common but often unrecognized complication of hematopoietic cell transplantation (HCT). Delirium can occur in patients of any age and is observed after autologous or allogeneic HCT. Although delirium has been studied primarily during initial HCT hospitalizations in recipients of myeloablative conditioning, recent investigations have identified delirium later post-transplantation and in recipients of reduced-intensity conditioning. Acute encephalopathy can be driven by infectious complications, medications, tissue damage, and/or organ dysfunction. Altered consciousness, either mild or profound, is often its only clinical manifestation. Identifying delirium is essential to overall HCT care, because patients who experience delirium have longer hospitalization and recovery times and are at risk for other poor post-HCT outcomes. Given the critical nature of this common complication and the ongoing expansion of HCT for more vulnerable populations, the American Society of Transplantation and Cellular Therapy (ASTCT) recommends intensifying research into post-HCT cognitive changes and establishing standardized definitions that encompass the full spectrum of altered consciousness for clinical care purposes and to provide benchmark endpoints for future research studies. To capture a range of acute neurocognitive changes specifically found in HCT patients (often referred to as acute encephalopathy), the ASTCT proposes a new diagnosis, transplantation-associated altered mentation and encephalopathy (TAME). The TAME diagnosis includes HCT patients who meet Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for delirium and those with acute neurocognitive changes who do not meet all the DSM-5 criteria for delirium (subsyndromal delirium). Early TAME is defined as occurring during conditioning or ≤100 days post-HCT, whereas late TAME occurs >100 days post-HCT in patients with additional HCT-related complications. This manuscript establishes clear diagnostic criteria and discusses factors that can potentially impact the development of TAME, as well as the workup and management of TAME.
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  • 文章类型: Journal Article
    肾上腺脑白质营养不良(ALD)是一种罕见X连锁隐性遗传病,其中脑型ALD(cALD)是最严重的类型,常于儿童期隐匿起病,极易误诊且进展迅速。异基因造血干细胞移植是治疗cALD的有效方法。中华医学会儿科学分会血液学组、神经学组、内分泌遗传代谢学组、放射学分会联合中华儿科编辑委员会组织制定本共识,旨在对cALD患者的诊断、治疗及多学科随访提供指导意见。.
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  • 文章类型: English Abstract
    法语国家骨髓移植和细胞治疗协会(SFGM-TC)于2022年9月在里尔组织了第13次造血干细胞移植临床实践协调程序研讨会,法国。本次研讨会的目的是更新自体造血干细胞移植治疗自身免疫性疾病的动员和调节方案,并指定移植的禁忌症,调理方案选择,在动员和疾病特异性监测之前停止免疫抑制治疗。
    The Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) organized the 13th workshop on hematopoietic stem cell transplantation clinical practices harmonization procedures in September 2022 in Lille, France. The aim of this workshop is to update the mobilization and conditioning protocols for autologous hematopoietic stem cell transplantation for autoimmune diseases, and to specify contraindications for transplant, conditioning regimen selection, immunosuppressive treatment discontinuation before mobilization and disease-specific surveillance.
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  • 文章类型: Journal Article
    NCCN造血细胞移植(HCT)指南为使用自体和同种异体HCT治疗成年患者的恶性疾病提供了一种基于证据和共识的方法。对于某些类型的恶性肿瘤患者,HCT是一种潜在的治愈性治疗选择;然而,复发性恶性肿瘤和移植相关并发症往往会限制HCT受者的长期生存.这些指南的目的是提供有关HCT方面的指导,包括移植前受体评估,造血细胞动员,和移植物抗宿主病的治疗-同种异体HCT的主要并发症-使患者和临床医生能够根据个体患者的病情评估治疗方案。这些NCCN指南见解提供了HCTNCCN指南的重要最新更新的摘要,包括纳入新开发的HCT条件原则部分。
    The NCCN Guidelines for Hematopoietic Cell Transplantation (HCT) provide an evidence- and consensus-based approach for the use of autologous and allogeneic HCT in the management of malignant diseases in adult patients. HCT is a potentially curative treatment option for patients with certain types of malignancies; however, recurrent malignancy and transplant-related complications often limit the long-term survival of HCT recipients. The purpose of these guidelines is to provide guidance regarding aspects of HCT, including pretransplant recipient evaluation, hematopoietic cell mobilization, and treatment of graft-versus-host disease-a major complication of allogeneic HCT-to enable the patient and clinician to assess management options in the context of an individual patient\'s condition. These NCCN Guidelines Insights provide a summary of the important recent updates to the NCCN Guidelines for HCT, including the incorporation of a newly developed section on the Principles of Conditioning for HCT.
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  • 文章类型: Journal Article
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  • 文章类型: English Abstract
    急性GVHD是造血干细胞移植的潜在严重并发症,可影响移植后预后的发病率和死亡率。在法语国家骨髓移植和细胞治疗学会(SFGM-TC)的第12次实践协调研讨会的框架内,急性GVHD的诊断模式更新.常规预防(取决于捐赠者,conditioning,和干细胞来源)和治疗方案(取决于受影响的器官和强度)明确了aGVHD,并讨论了新的治疗选择。
    Acute GVHD is a potentially severe complication of hematopoietic stem cell transplantation, responsible for morbidity and mortality that can affect the prognosis after transplantation. Within the framework of the 12th workshop of practice harmonization of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy (SFGM-TC), diagnostic modalities of acute GVHD are updated. The conventional prevention (depending on donor, conditioning, and stem cell source) and treatment schemes (depending on affected organ and intensity) of aGVHD are clarified, and new therapeutic options are discussed.
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  • 文章类型: Review
    骨髓增生异常综合征(MDS)的唯一治疗方法是异基因造血细胞移植(HCT)。在这里,在证据的背景下对这种治疗方式进行了审查和严格评估。使用特定标准来搜索已发表的文献,并对证据的质量和强度以及建议的强度进行分级。由移植和非移植医生组成的MDS专家小组制定了共识治疗建议。这篇综述总结了HCT的标准MDS适应症,并解决了争议领域。最近的前瞻性试验证实,与非移植方法相比,同种异体HCT在晚期或高危MDS患者中具有生存益处。在表现良好的老年患者中,HCT的使用正在增加。然而,有高危细胞遗传学或分子突变的患者仍有高复发风险.目前尚不清楚在移植前或移植后施用新疗法是否可以降低选定人群中疾病复发的风险。正在进行的和未来的研究将调查疾病风险分层的修订方法,患者选择,和移植后方法优化MDS患者的同种异体HCT结果。
    The sole curative therapy for myelodysplastic syndrome (MDS) is allogeneic hematopoietic cell transplantation (HCT). Here this therapeutic modality is reviewed and critically evaluated in the context of the evidence. Specific criteria were used for searching the published literature and for grading the quality and strength of the evidence and the strength of the recommendations. A panel of MDS experts comprising transplantation and nontransplantation physicians developed consensus treatment recommendations. This review summarizes the standard MDS indications for HCT and addresses areas of controversy. Recent prospective trials have confirmed that allogeneic HCT confers survival benefits in patients with advanced or high-risk MDS compared with nontransplantation approaches, and the use of HCT is increasing in older patients with good performance status. However, patients with high-risk cytogenetic or molecular mutations remain at high risk for relapse. It is unknown whether administration of novel therapies before or after transplantation may decrease the risk of disease relapse in selected populations. Ongoing and future studies will investigate revised approaches to disease risk stratification, patient selection, and post-transplantation approaches to optimize allogeneic HCT outcomes for patients with MDS.
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  • 文章类型: English Abstract
    新技术的出现使更频繁地识别骨髓增生异常综合征(MDS)和急性白血病(AL)的遗传易感性成为可能。目前最常见和最有特点的是CEBPA突变,RUNX1、GATA2、ETV6和DDX41,在存在这些具有高等位基因频率的突变之一的情况下,或在个人或家族史提示血液异常的情况下,如非免疫性血小板减少症,建议寻找遗传性血液系统恶性肿瘤(HHM)的可能性。的确,对这些HHM的早期识别可以更好地适应患者及其亲属的管理,因为异基因造血干细胞移植(HSCT)经常被提出用于这些病理。根据目前的数据,除了GATA2突变,突变的体质或体细胞性质似乎并不影响血液病的预后。因此,同种异体移植物的适应症将根据通常的标准确定。然而,在寻找家庭捐赠者时,重要的是要确保捐赠者没有遗传性疾病。为了保证在短时间内进行HSC同种异体移植的可能性,可能有必要启动平行程序以寻找无关的供体.鉴于在这种情况下HSC移植方式的信息有限,重要的是评估疾病的获益/风险以及决定条件处理类型(清髓性或降低强度)的程序.鉴于中期和长期继发性癌症风险的经验有限,建议降低强度调理可能是合适的,如在更好表征的综合征性血液系统疾病如范可尼贫血或端粒疾病的情况下。总之,更频繁地唤起HHM似乎很重要,特别是在有家族史的情况下,某些突变或持续性血液异常,为了讨论HSC同种异体移植的具体模式,特别是在寻找捐助者和评估程序的某些方式方面,比如调理。应该注意的是,HHM的发现,特别是如果同种异体HSC移植的迹象被保留,将提高伦理和心理的考虑,不仅为病人,还有他的家人.涉及分子生物学家的多学科方法,遗传学家,血液学家和心理学家是必不可少的。
    The advent of new technologies has made it possible to identify genetic predispositions to myelodysplastic syndromes (MDS) and acute leukemias (AL) more frequently. The most frequent and best characterized at present are mutations in CEBPA, RUNX1, GATA2, ETV6 and DDX41 and, either in the presence of one of these mutations with a high allelic frequency, or in the case of a personal or family history suggestive of blood abnormalities such as non-immune thrombocytopenia, it is recommended to look for the possibility of a hereditary hematological malignancy (HHM). Indeed, early recognition of these HHMs allows better adaptation of the management of patients and their relatives, as allogeneic hematopoietic stem cell transplantation (HSCT) is very often proposed for these pathologies. According to current data, with the exception of the GATA2 mutation, the constitutional or somatic nature of the mutations does not seem to influence the prognosis of hematological diseases. Therefore, the indication for an allograft will be determined according to the usual criteria. However, when searching for a family donor, it is important to ensure that there is no hereditary disease in the donor. In order to guarantee the possibility of performing the HSC allograft within a short period of time, it may be necessary to initiate a parallel procedure to find an unrelated donor. Given the limited information on the modalities of HSC transplantation in this setting, it is important to assess the benefit/risk of the disease and the procedure to decide on the type of conditioning (myeloablative or reduced intensity). In view of the limited experience with the risk of secondary cancers in the medium and long-term, it may be appropriate to recommend reduced intensity conditioning, as in the case of better characterized syndromic hematological diseases such as Fanconi anemia or telomere diseases. In summary, it seems important to evoke HHM more frequently, particularly in the presence of a family history, certain mutations or persistent blood abnormalities, in order to discuss the specific modalities of HSC allografting, particularly with regard to the search for a donor and the evaluation of certain modalities of the procedure, such as conditioning. It should be noted that the discovery of HHM, especially if the indication of an allogeneic HSC transplant is retained, will raise ethical and psychological considerations not only for the patient, but also for his family. A multidisciplinary approach involving molecular biologists, geneticists, hematologists and psychologists is essential.
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  • 文章类型: English Abstract
    遗传性代谢疾病(IMD)是罕见的遗传疾病,包括溶酶体和过氧化物酶体疾病。溶酶体疾病与一种或多种溶酶体酶或转运蛋白的缺乏有关。溶酶体疾病是进行性的,涉及几种组织,最常见的是神经损伤。在过氧化物酶体疾病中,X连锁肾上腺脑白质营养不良(ALD)是一种神经退行性疾病,结合了神经系统和肾上腺的损害。对于这些疾病,酶替代疗法(ERT),异基因造血细胞移植(allo-HCT)和基因治疗代表了各种可能的治疗选择,单独使用或组合使用。这次研讨会的目的是描述适应症,模态,以及allo-HCT的随访以及ERT围移植的使用。这些罕见疾病的所有移植适应症都与合并症有关,并且必须遵守必须在专门的国家多学科咨询会议上讨论的标准。I-H型粘多糖贮积症(MPS-IH)和ALD的大脑形式有一些共识。对于其他IMD,移植没有明显的益处。理想的供体是非杂合HLA相同的同胞。推荐的调理是结合氟达拉滨和白消安的清髓性。在MPS-IH中,ERT必须在诊断时开始,并持续到达到完全嵌合状态和正常酶测定。根据公布的建议(PNDS)进行移植前评估和移植后随访。标准随访由移植和转诊小组联合进行。
    Inherited Metabolic Diseases (IMD) are rare genetic diseases, including both lysosomal and peroxisomal diseases. Lysosomal diseases are related to the deficiency of one or more lysosomal enzymes or transporter. Lysosomal diseases are progressive and involve several tissues with most often neurological damage. Among peroxisomal diseases, X-linked adrenoleukodystrophy (ALD) is a neurodegenerative disease combining neurological and adrenal damage. For these diseases, enzyme replacement therapy (ERT), allogeneic hematopoietic cell transplantation (allo-HCT) and gene therapy represent various possible treatment options, used alone or in combination. The purpose of this workshop is to describe the indications, modalities, and follow-up of allo-HCT as well as the use of ERT peri-transplant. All indications for transplant in these rare diseases are associated with comorbidities and are subject to criteria that must be discussed in a dedicated national multidisciplinary consultation meeting. There are some consensual indications in type I-H mucopolysaccharidosis (MPS-IH) and in the cerebral form of ALD. For other IMDs, no clear benefit from the transplant has been demonstrated. The ideal donor is a non-heterozygous HLA-identical sibling. The recommended conditioning is myeloablative combining fludarabine and busulfan. In MPS-IH, ERT has to be started at diagnosis and continued until complete chimerism and normal enzyme assay are achieved. The pre-transplant assessment and post-transplant follow-up are made according to the published recommendations (PNDS). Standard follow-up is carried out jointly by the transplant and referral teams.
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