Department of Child and Adolescent Psychiatry, Psychotherapy and Psychosomatics, University Medical Center Hamburg-Eppendorf, Martinistrasse 52, 20246, Hamburg, Germany.
The Danish National Rehabilitation Centre for Neuromuscular Diseases, Kongsvang Allé 23, 8000, Aarhus C, Denmark.
J Neurol. 2017 Apr;264(4):709-723. doi: 10.1007/s00415-017-8406-2. Epub 2017 Feb 7.
Duchenne muscular dystrophy (DMD) is a progressive, genetically determined neuromuscular disease that affects males and leads to severe physical disability in early teenage years. Over the last decades, patient-reported outcomes such as Health-Related Quality of Life (HRQoL) gained great interest in clinical research. However, little is known about factors affecting HRQoL in boys with DMD. Data from the multi-center CARE-NMD project of boys with DMD from six European countries collected between 2011 and 2012 were analyzed (8-17 years old; n = 321). HRQoL was measured using the KIDSCREEN-10 index, the Pediatric Quality of Life Inventory (PedsQL) and the Neuromuscular Module of the PedsQL (NMM). Linear regression models served to examine influences of socio-demographic, disease- and treatment-specific as well as participation- and environment-related factors on overall and disease-specific HRQoL. Proportions of explained variance varied across models using different outcomes (18-34%). Overall HRQoL according to the KIDSCREEN-10 index was associated with household income, the frequency of attending a clinic with specialized staff, the number of days spent outside home, and the attitude of the local community, but no significant association with age occurred. Overall HRQoL according to the generic PedsQL and disease-specific HRQoL were both positively associated with age and influenced by the country of residence, the disease stage, number of days spent outside home, and the attitude of the local community. Our results may be relevant for clinical practice and planning interventions for this population, but should be confirmed by future research. Further questions for future studies on boys with DMD are proposed.
杜氏肌营养不良症（DMD）是一种进行性、遗传性神经肌肉疾病，主要影响男性，导致青少年早期严重身体残疾。在过去几十年中，健康相关生活质量（HRQoL）等患者报告的结果在临床研究中受到了极大的关注。然而，关于影响 DMD 男孩 HRQoL 的因素知之甚少。分析了 2011 年至 2012 年期间来自六个欧洲国家的 CARE-NMD 项目中 321 名 DMD 男孩的多中心数据（8-17 岁）。使用 KIDSCREEN-10 指数、儿科生活质量问卷（PedsQL）和 PedsQL 神经肌肉模块（NMM）来测量 HRQoL。线性回归模型用于检查社会人口统计学、疾病和治疗特异性以及参与和环境相关因素对整体和疾病特异性 HRQoL 的影响。使用不同结果的模型解释方差的比例不同（18-34%）。根据 KIDSCREEN-10 指数的整体 HRQoL 与家庭收入、定期接受专科医护人员诊所就诊的频率、在家外度过的天数以及当地社区的态度有关，但与年龄无显著关联。根据通用 PedsQL 和疾病特异性 HRQoL 的整体 HRQoL 均与年龄呈正相关，并受到居住地、疾病阶段、在家外度过的天数和当地社区的态度的影响。我们的研究结果可能与临床实践和该人群的干预措施规划有关，但应通过未来的研究加以证实。对 DMD 男孩未来研究提出了进一步的问题。
