背景:退行性脊髓型颈椎病(DCM)是一种进行性慢性脊髓损伤,估计影响50名成年人中的1名。没有标准化指导,临床研究研究可以自行决定选择结果,往往不足以代表疾病,限制了研究之间的可比性。利用通过多方利益相关者共识形成的标准最低数据集可以解决这些问题。这结合了定义核心结果集(COS)(关键结果列表)和核心数据元素(CDE)的过程,解释结果所需的关键抽样特征列表.然后在核心测量集(CMS)中进一步定义“如何测量和/或报告这些结果”。这可以包括报告结果数据的标准化时间点的建议。这项研究定义了COS,CDE,和CMS用于DCM研究。
结果:使用一系列修改的Delphi过程开发了最小数据集。第一阶段涉及建立一个国际DCM利益相关者小组。第二阶段涉及开发一系列成果,数据元素,并形成域。第3阶段使用两阶段Delphi流程对结果和CDE进行了优先级排序。阶段4使用共识会议确定最终DCM最小数据集。使用COS,第5阶段最终确定了每个结果的测量结构的定义。在第6阶段,对文献进行了系统回顾,范围和定义测量工具的心理测量属性。阶段7使用修改后的Delphi过程来通知候选测量工具的候选列表。然后通过共识会议(第8阶段)形成最终测量集。为了支持实施,然后将数据集整合到模板临床研究表格(CRF)中,用于未来的临床试验(第9阶段).总的来说,28个结果和6个领域(疼痛,神经功能,生活影响,放射学,经济影响,和不良事件)被输入到最终的COS中。32个结果和4个领域(个人,疾病,调查,和干预)进入最终CDE。最后,4个结果工具(mJOA,NDI,SF-36v2和SAVES2)被鉴定为CMS,建议进行评估手术后结果的试验,包括基线测量和手术后6个月。
结论:AOSpineRECODE-DCM产生了目前用于DCM临床试验的最小数据集。这些可在https://myopathy.org/minimum-dataset/上找到。虽然预计CDE和COS具有强大而持久的相关性,人们承认,新的测量工具,随着越来越多地过渡到研究未接受手术的患者,可能需要更新和调整,特别是关于CMS。
BACKGROUND: Degenerative cervical myelopathy (DCM) is a progressive chronic spinal cord injury estimated to affect 1 in 50 adults. Without standardised guidance, clinical research studies have selected outcomes at their discretion, often underrepresenting the disease and limiting comparability between studies. Utilising a standard minimum data set formed via multi-stakeholder
consensus can address these issues. This combines processes to define a core outcome set (COS)-a list of key outcomes-and core data elements (CDEs), a list of key sampling characteristics required to interpret the outcomes. Further \"how\" these outcomes should be measured and/or reported is then defined in a core measurement set (CMS). This can include a recommendation of a standardised time point at which outcome data should be reported. This study defines a COS, CDE, and CMS for DCM research.
RESULTS: A minimum data set was developed using a series of modified Delphi processes. Phase 1 involved the setup of an international DCM stakeholder group. Phase 2 involved the development of a longlist of outcomes, data elements, and formation into domains. Phase 3 prioritised the outcomes and CDEs using a two-stage Delphi process. Phase 4 determined the final DCM minimal data set using a
consensus meeting. Using the COS, Phase 5 finalised definitions of the measurement construct for each outcome. In Phase 6, a systematic review of the literature was performed, to scope and define the psychometric properties of measurement tools. Phase 7 used a modified Delphi process to inform the short-listing of candidate measurement tools. The final measurement set was then formed through a
consensus meeting (Phase 8). To support implementation, the data set was then integrated into template clinical research forms (CRFs) for use in future clinical trials (Phase 9). In total, 28 outcomes and 6 domains (Pain, Neurological Function, Life Impact, Radiology, Economic Impact, and Adverse Events) were entered into the final COS. Thirty two outcomes and 4 domains (Individual, Disease, Investigation, and Intervention) were entered into the final CDE. Finally, 4 outcome instruments (mJOA, NDI, SF-36v2, and SAVES2) were identified for the CMS, with a recommendation for trials evaluating outcomes after surgery, to include baseline measurement and at 6 months from surgery.
CONCLUSIONS: The AO Spine RECODE-DCM has produced a minimum data set for use in DCM clinical trials today. These are available at https://myelopathy.org/minimum-dataset/. While it is anticipated the CDE and COS have strong and durable relevance, it is acknowledged that new measurement tools, alongside an increasing transition to study patients not undergoing surgery, may necessitate updates and adaptation, particularly with respect to the CMS.