Aim: To develop a novel proxy-reported scale of motor function in infants and young children with early-onset neuromuscular disorders (NMD), entitled the Proxy Motor Outcome Measure (PMOM).
Design: A mixed method design was employed, applying both qualitative and quantitative research.
Methods: A framework technique using sensitivity analyses guided the development of the most appropriate and relevant subset of items, modelled after 30 neuromuscular disease instruments/scales. The PMOM was designed based on semi-structured interviews with 16 proxies; a focus group of 11 experts in neuromuscular diseases and scale development, 10 of whom also gave quantitative data using a two-round Delphi method survey; and cognitive interviews with five proxies. These processes were conducted between January 2014-March 2019.
Results: Nine themes and 32 subthemes were derived from the semi-structured interviews. Five domains and three subdomains of potential items were identified by the focus group. An initial version of the PMOM scale was created with 121 items. Using the two-round Delphi method, 43 items met agreement on pre-defined requirements. The second version of the PMOM scale included these 43 and two additional items based on expert feedback. Proxies gave 114 suggestions on cognitive interviews, 99 of which were successfully addressed by the research team. The final version of the PMOM scale included 43 items.
Conclusion: We developed a preliminary proxy-reported instrument, the PMOM, to evaluate motor function in infants and young children with early-onset NMD.
Impact: Proxies hold a wealth of knowledge on their child's motor function during early development, which may complement clinic-based motor function testing. However, there is no validated measure of motor function that incorporates the observation of proxies of infants and young children with NMD. Future work will be focused on assessing the reliability, validity and responsiveness of the PMOM scale and implementing this tool in clinical studies.
目的: 开发新型代理报告的早发性神经肌肉疾病 (NMD) 婴幼儿运动机能量表, 名为代用性运动功能量表(PMOM)。 设计: 采用混合法设计, 定性和定量研究相结合。 方法: 以30个神经肌肉疾病仪器/量表为模型, 使用敏感性分析的框架技术指导最合适和相关的项目子集的开发。基于对16位代理人的半结构化访谈,设计代理运动结果测量; 由11位神经肌肉疾病和量表开发专家组成的焦点小组, 其中10位专家还使用两轮德尔菲调查法提供了定量数据; 以及对5位代理人进行认知访谈。这些过程已在2014年1月至2019年3月期间实施。 结果: 9个主题和32个子主题来自半结构化访谈。焦点小组确定了潜在项目的五个领域和三个子领域。代理运动结果测量量表的初始版本由121个项目组成。43个项目采用两轮德尔菲法, 且符合预先规定的要求。代理运动结果测量量表的第二个版本包括了这43个项目和两个基于专家反馈的附加项目。代理人在认知访谈中给出了114条建议, 研究小组成功地采用了其中的99条建议。代理运动结果测量量表的最终版本包括43个项目。 结论: 我们开发了一种初步代理报告工具, 即代理运动结果测量, 用于评估早发性神经肌肉疾病婴幼儿的运动机能。 影响: 代理人非常了解孩子在早期发育的运动机能, 这可能是对临床运动机能测试的补充。然而, 目前还没有一种有效的运动机能测量方法, 包括观察患有早发性神经肌肉疾病婴幼儿的代理人。未来的工作将集中于评估代理运动结果测量量表的信度、效度和反应性, 并在临床研究中实施这一工具。.
Keywords: Delphi method; PMOM; cognitive interviews; infants and young children; instrument development; mixed methods; motor function; neuromuscular disorders; nursing; proxy-reported scale.
© 2020 John Wiley & Sons Ltd.