背景与目的:转化大型动物模型是将软骨修复方法转移到临床的必然要求。这些试验的指南已经由指导机构(FDA,ASTM,EMEA)包括研究描述符和研究结果的建议。然而,并不是在所有方面都能切实遵守这些建议。这项研究包括评估有关该领域专业人员在大型动物模型中进行软骨修复的实际相关性的建议方面。材料和方法:在一项基于在线的调查中,评估了先前发布的指南中有关研究设计的11个方面和有关研究结果的13个方面(0-10分,其中10个是最重要的)研究参与者。此外,调查包含与专业经验(年)有关的问题,专业焦点(临床前,临床,兽医,工业)和首选的用于软骨修复的翻译大动物模型。结果:调查参与者总数为37人。对研究设计参数最重要的是病变大小(9.54pts。,SD0.80),其次是研究持续时间(9.43pts。,SD1.21);和支架固定方法(9.08分。,SD1.30)以及病变深度(9.03pts。,SD1.77)。研究结果最重要的方面是组织学(9.41pts。,SD0.86)和缺陷填充(8.97分。,SD1.21),而基因表达被认为是最不重要的(6.11pts。,SD2.46)结果。总共62.2%的参与者是研究人员,18.9%临床医生,13.5%的兽医和5.4%的行业员工。结论:在转化研究中,指导机构发表的建议在社会上获得广泛的理论共识,包括临床和临床前定位的科学家。然而,在主要方面缺乏对实践研究的实施。在所有利益攸关方的参与下,对准则进行持续的重新评估,以及克服财务和基础设施限制的方法,可以支持对指导文件的接受,并有助于该领域的标准化。
Background and Objective: Translational large animal models are inevitable to transfer cartilage repair methods into clinical practice.
Guidelines for these trials have been published by guiding agencies (FDA, ASTM, EMEA) including recommendations for study descriptors and study outcomes. However, practical adherence to these recommendations is not achieved in all aspects. This study includes an assessment of the recommended aspects regarding practical relevance in large animal models for cartilage repair by professionals in the field. Materials and Methods: In an online based survey, 11 aspects regarding study design and 13 aspects regarding study outcome from previously published
guidelines were evaluated (0-10 points, with 10 being most important) by study participants. Additionally, the survey contained questions related to professional experience (years), professional focus (preclinical, clinical, veterinarian, industry) and the preferred translational large animal model for cartilage repair. Results: The total number of survey participants was 37. Rated as most important for study design parameters was lesion size (9.54 pts., SD 0.80) followed by study duration (9.43 pts., SD 1.21); and method of scaffold fixation (9.08 pts., SD 1.30) as well as depth of the lesion (9.03 pts., SD 1.77). The most important aspects of study outcome were considered histology (9.41 pts., SD 0.86) and defect filling (8.97 pts., SD 1.21), while gene expression was judged as the least important (6.11 pts., SD 2.46) outcome. A total of 62.2% of all participants were researchers, 18.9% clinicians, 13.5% veterinarians and 5.4% industry employees. Conclusions: In translational research, recommendations published by guiding agencies receive broad theoretical
consensus within the community, including both clinically and preclinically orientated scientists. However, implementation into practical research lacks in major aspects. Ongoing re-evaluation of the
guidelines under involvement of all stakeholders and approaches to overcome financial and infrastructural limitations could support the acceptance of the guidance documents and contribute to standardization in the field.