Department of Orthopaedic Surgery, The University of Edinburgh, Edinburgh, United Kingdom.
Rush University Medical Center, Chicago, Illinois.
J Bone Joint Surg Am. 2019 May 15;101(10):904-911. doi: 10.2106/JBJS.18.00915.
BACKGROUND: The lack of a standardized system for describing cell therapies acts as a barrier to advancement in clinical and basic research and practice. The aim of this study was to establish an international expert consensus on strategies to improve standardization and transparency when describing cell therapies. The secondary aim was to develop a consensus among experts on the contents of a standardized tool for describing cell therapies. METHODS: The need for expert consensus on strategies to improve cell therapy communication was confirmed at the American Academy of Orthopaedic Surgeons/National Institutes of Health Optimizing Clinical Use of Biologics Symposium in 2018. A working group of 6 experts convened an international consensus process involving clinicians and basic scientists using validated Delphi methodology. This iterative process was used to define statements on communication of cell therapies and develop a standardized tool for describing cell therapies. RESULTS: Thirty-four experts completed 3 rounds survey with use of the Delphi process. After 3 rounds, 27 statements relating to existing nomenclature, solutions to improve communication, ideal characteristics of a framework, mandatory elements of a new framework, and future work to facilitate application reached consensus with >80% agreement and <5% disagreement. Consensus was reached on the contents of a tool for improving standardization and transparency when describing cell therapies. This tool, dubbed "DOSES," is based on the reporting of 5 core items: donor (i.e., autologous, allogeneic, xenogeneic), origin of tissue, separation from other cell types/preparation method, exhibited cell characteristics associated with behavior, and the site of delivery. CONCLUSIONS: This study has established expert consensus on the communication of cell therapies. The DOSES tool has been developed to improve standardization and transparency in describing cell therapies. CLINICAL RELEVANCE: The DOSES tool for describing cell therapies can be utilized by researchers, clinicians, regulators, and industry professionals to improve standardization and transparency when describing cell therapies. The use of this tool may allow clinicians and patients to better understand the characteristics of current and future cell preparations.
背景:缺乏用于描述细胞疗法的标准化系统,这阻碍了临床和基础研究与实践的发展。本研究旨在建立国际专家共识,以改善描述细胞疗法时的标准化和透明度策略。次要目的是在专家中就描述细胞疗法的标准化工具的内容达成共识。
方法:2018 年,在矫形外科医师学会/美国国立卫生研究院优化生物制剂临床应用研讨会上,确认了在细胞疗法交流方面需要专家达成共识。一个由 6 名专家组成的工作组采用经过验证的德尔菲法召集了一个国际共识过程,涉及临床医生和基础科学家。该迭代过程用于定义细胞疗法的交流声明,并开发描述细胞疗法的标准化工具。
结果:34 名专家完成了 3 轮德尔菲调查。经过 3 轮,27 项与现有命名法、改善沟通的解决方案、框架的理想特征、新框架的强制性要素以及促进应用的未来工作相关的声明,以>80%的一致性和<5%的分歧达成共识。在描述细胞疗法时,提高标准化和透明度的工具内容也达成了共识。该工具名为“DOSES”,基于对 5 个核心项目的报告:供体(即自体、同种异体、异种)、组织来源、与其他细胞类型的分离/准备方法、表现出与行为相关的细胞特征以及给药部位。
结论:本研究在细胞疗法的交流方面达成了专家共识。已经开发了 DOSES 工具,以提高描述细胞疗法的标准化和透明度。
临床相关性:描述细胞疗法的 DOSES 工具可被研究人员、临床医生、监管机构和行业专业人员用于在描述细胞疗法时提高标准化和透明度。该工具的使用可以使临床医生和患者更好地理解当前和未来细胞制剂的特征。
J Bone Joint Surg Am. 2019-5-15
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