Division of Neurosurgery, Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom.
Myelopathy.org, Cambridge, United Kingdom.
PLoS Med. 2024 Aug 22;21(8):e1004447. doi: 10.1371/journal.pmed.1004447. eCollection 2024 Aug.
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.
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.
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.
退行性颈脊髓病（DCM）是一种进行性慢性脊髓损伤，据估计，每 50 个成年人中就有 1 人会受到影响。如果没有标准化的指导，临床研究可能会自行选择结果，这往往会使疾病代表性不足，并限制研究之间的可比性。利用多利益相关者共识形成的标准最小数据集可以解决这些问题。这结合了定义核心结局集（COS）的过程 - 一组关键结局的列表 - 和核心数据元素（CDEs），这是解释结局所需的关键采样特征的列表。进一步“如何”测量和/或报告这些结果，然后在核心测量集中（CMS）进行定义。这可以包括推荐报告结果数据的标准时间点。本研究为 DCM 研究定义了 COS、CDE 和 CMS。
使用一系列改良 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个包含结局、数据元素和形成域的长名单。第三阶段使用两阶段 Delphi 过程优先考虑结局和 CDEs。第四阶段使用共识会议确定最终的 DCM 最小数据集。使用 COS，第五阶段最终确定了每个结局的测量结构的定义。在第六阶段，对文献进行了系统综述，以确定和定义测量工具的心理测量特性。第七阶段使用改良 Delphi 过程来告知候选测量工具的筛选。然后通过共识会议形成最终的测量集（第八阶段）。为了支持实施，数据集随后被整合到模板临床研究表格（CRFs）中，用于未来的临床试验（第九阶段）。总共 28 个结局和 6 个领域（疼痛、神经功能、生活影响、影像学、经济影响和不良事件）进入了最终的 COS。32 个结局和 4 个领域（个体、疾病、调查和干预）进入了最终的 CDE。最后，CMS 确定了 4 种结局工具（mJOA、NDI、SF-36v2 和 SAVES2），并建议评估手术后结局的试验应包括基线测量和术后 6 个月的测量。
AO Spine RECODE-DCM 为今天的 DCM 临床试验提供了最小数据集。这些可在 https://myelopathy.org/minimum-dataset/ 获得。虽然预计 CDE 和 COS 具有很强的持久相关性，但需要认识到，新的测量工具以及越来越多的过渡到不接受手术的研究患者，可能需要更新和调整，特别是在 CMS 方面。
