Orcesi Simona, Ariaudo Giada, Mercuri Eugenio, Beghi Ettore, Rezzani Cristiana, Balottin Umberto
1Unit of Child Neurology and Psychiatry, C. Mondino National Neurological Institute, Pavia, Italy.
J Child Neurol. 2014 Feb;29(2):167-81. doi: 10.1177/0883073813511859. Epub 2013 Dec 18.
Improvement of quality of life in neuromuscular disorders is a primary objective, both in management of affected children and in the context of therapeutic trials. Quality of life is a subjective concept and it is crucial to gather information directly from patients. We created the SOLE Questionnaire for NMDs, a new instrument designed to investigate quality of life in children with neuromuscular disorders, and tested it in a study population of 78 patients and in 81 healthy children aged 5 to 13 years. The SOLE Questionnaire, characterized by a visual and neutral approach, was well received, practical, rapid to administer, and able to discriminate between patients and controls. We also confirmed the presence of disagreement about children's quality of life between children and their parents. We suggest that our new approach could help to improve understanding of quality of life in children with neuromuscular disorders.
改善神经肌肉疾病患者的生活质量是主要目标，无论是在受影响儿童的管理方面还是在治疗试验的背景下。生活质量是一个主观概念，直接从患者那里收集信息至关重要。我们创建了神经肌肉疾病SOLE问卷，这是一种旨在调查神经肌肉疾病儿童生活质量的新工具，并在78名患者和81名5至13岁健康儿童的研究人群中进行了测试。SOLE问卷以直观和中立的方式为特点，受到好评，实用，易于实施，并且能够区分患者和对照组。我们还证实了儿童与其父母之间在儿童生活质量方面存在分歧。我们建议，我们的新方法有助于增进对神经肌肉疾病儿童生活质量的理解。
