UZ Leuven, Child Neurology, Leuven, Belgium.
Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
J Neuromuscul Dis. 2024;11(4):801-814. doi: 10.3233/JND-240012.
Patients with Duchenne muscular dystrophy (DMD) face a higher risk of neurobehavioral problems, yet an international consensus on screening, assessing, and managing these difficulties is lacking.
This report introduces the term Duchenne Muscular Dystrophy-Associated Neurobehavioral Difficulties (DuMAND) to comprehensively cover the spectrum of neurobehavioral issues in DMD patients, including behavior, psychiatric disorders, and various cognitive, academic, and psychosocial deficits. To facilitate screening, the DuMAND Checklist, a 43-item tool with five subscales, was developed.
DuMAND categories were derived through literature review, parent (48 mothers and 37 fathers), and expert (n = 28) input and feedback. The DuMAND Checklist subscales were developed iteratively, incorporating item selection, expert panel (n = 10) assessment for face validity, comprehensiveness, and a pilot validation study in a DMD sample (n = 20). DuMAND encompasses five categories: cognition and learning, social responsiveness, emotion regulation, externalizing behavior, and eating and sleeping. Preliminary validation of the DuMAND Checklist indicates acceptable-to-excellent internal consistency and construct validity.
By introducing the DuMAND concept, this study seeks to inspire a consensus approach for screening, assessing, and managing neurobehavioral issues in DMD. Incorporating screening, using the DuMAND Checklist, in addition to medical follow-up will facilitate early intervention, addressing a critical gap in identification of neurobehavioral disorders in DMD. Future research is needed to further evaluate psychometric properties of the DuMAND Checklist and investigate the natural course of DuMAND.
杜氏肌营养不良症（DMD）患者面临更高的神经行为问题风险，但缺乏对此类问题进行筛查、评估和管理的国际共识。
本报告引入“杜氏肌营养不良症相关神经行为障碍（DuMAND）”这一术语，全面涵盖 DMD 患者的神经行为问题，包括行为、精神障碍以及各种认知、学业和心理社会缺陷。为便于筛查，开发了包含五个子量表的 43 项 DuMAND 清单工具。
DuMAND 类别源自文献回顾、家长（48 位母亲和 37 位父亲）和专家（n=28）的输入和反馈。DuMAND 清单的子量表是通过迭代开发的，包括项目选择、专家小组（n=10）对面有效度、全面性的评估以及在 DMD 样本中进行的试点验证研究（n=20）。DuMAND 涵盖五个类别：认知和学习、社交反应、情绪调节、外化行为和饮食与睡眠。DuMAND 清单的初步验证表明具有可接受至优秀的内部一致性和结构效度。
通过引入 DuMAND 概念，本研究旨在为 DMD 患者的神经行为问题的筛查、评估和管理提供共识方法。在医疗随访之外纳入筛查，并使用 DuMAND 清单，将有助于早期干预，解决 DMD 患者神经行为障碍识别的关键空白。未来需要进一步研究以评估 DuMAND 清单的心理测量学特性，并研究 DuMAND 的自然病程。
