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关于当前提高儿科输血临床安全性趋势的总结性评论,重点关注造血干细胞移植不良并发症的预防以及提高细胞治疗干细胞扩增安全性/质量标准的新策略。

Concluding commentary on current trends to enhance the clinical safety of pediatric transfusion, focusing on prevention of untoward complications of HSC transplantation & newer strategies for improving the standards of safety/quality of stem cells expansion for cellular therapy.

作者信息

Sniecinski Irena, Seghatchian Jerard

机构信息

Department of Transfusion Medicine, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Research Institute, Duarte, CA, USA.

International Consultancy in Blood Components Quality/Safety improvement, Audit/Inspection, and DDR Strategies, London, UK.

出版信息

Transfus Apher Sci. 2018 Jun;57(3):378-383. doi: 10.1016/j.transci.2018.05.023. Epub 2018 May 16.

Abstract

Clinical practice and related diagnostic, development and research [DDR] strategies in pediatric transfusion and transplantation cover a broad range of multidisciplinary studies, performed by many professionals involved in this most challenging clinical field [1]. This commentary on the current position and future perspectives in pediatric transfusion field is aimed to highlight major unresolved transfusion complications in pediatric patients, namely red blood cell and platelet alloimmunisation, and new ones such as nosocomial infection, thrombosis and multi-organ failure. Some other safety related issues issues in clinical management of neonates/young infants with urgent medical conditions, requiring immediate transfusion or apheresis treatment, especially, those resulting from hematopoietic stem cell transplantation (HSCT), have been addressed. Pediatric HSCT has evolved along with its growth and progress in adult population. New sources of stem cells, and greater donor options including apheresis donation by identical or haploidentical young children, new immunosuppressive drug and cell therapy regimens for prevention and treatment of transplantation related graft versus host disease (GVHD), recent developments in gene and immune cell as well as regenerative therapies, requiring implementation of advanced laboratory methods designed for efficient and safe HSC cell engineering are also discussed. Finally, the use of novel blood components, obtained from allogeneic cord bloods or platelet concentrates in successful treatment of ulcerative lesions in inherited or acquired conditions and in expansion of stem cells, as the growth media clinical grade supplement will be presented. Management of these new and challenging clinical situations in pediatric patients requires an integrated approach involving many specialties with overall goal of improving treatment outcome and quality of life. This only could be accomplished by adhering to existing practice standards in current practices and timely developing guidelines for new clinical applications. It is hoped that this commentary on the pediatric theme, by bridging the gap from bench to bedside and bringing the input from the prospective clinical trials back to laboratories provides a step forward to help in educational aspects of better understanding the specifics of pediatric patient care more fitting for the future interventional treatments.

摘要

儿科输血与移植领域的临床实践及相关诊断、开发与研究[DDR]策略涵盖了广泛的多学科研究,由众多投身于这一极具挑战性临床领域的专业人员开展[1]。这篇关于儿科输血领域当前状况及未来展望的评论旨在突出儿科患者中主要未解决的输血并发症,即红细胞和血小板同种免疫,以及诸如医院感染、血栓形成和多器官功能衰竭等新出现的并发症。还探讨了患有紧急医疗状况、需要立即输血或进行单采治疗的新生儿/幼儿临床管理中的一些其他安全相关问题,特别是那些由造血干细胞移植(HSCT)导致的问题。儿科HSCT随着其在成人中的发展而不断演进。文中还讨论了新的干细胞来源、更多的供体选择,包括同基因或单倍体儿童的单采捐献、用于预防和治疗移植相关移植物抗宿主病(GVHD)的新型免疫抑制药物和细胞治疗方案、基因和免疫细胞以及再生疗法的最新进展,这些都需要实施先进的实验室方法以实现高效且安全的造血干细胞(HSC)细胞工程。最后,将介绍从异体脐带血或血小板浓缩物中获得的新型血液成分在成功治疗遗传性或后天性疾病的溃疡性病变以及作为生长培养基临床级补充物用于干细胞扩增方面的应用。管理儿科患者这些新的且具有挑战性的临床情况需要一种综合方法,涉及多个专业领域,总体目标是改善治疗效果和生活质量。这只有通过在当前实践中坚持现有实践标准并及时制定新临床应用的指南才能实现。希望这篇关于儿科主题的评论,通过弥合从实验台到病床的差距,并将前瞻性临床试验的成果反馈到实验室,能在教育方面向前迈出一步,有助于更好地理解更适合未来介入治疗的儿科患者护理细节。

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