Department of Rare Diseases Research, Institute of Medical Science, St. Marianna University School of Medicine, Kawasaki, Japan.
Department of Neurology, Tokai Central Hospital, Kakamigahara, Japan.
Orphanet J Rare Dis. 2020 Jul 3;15(1):175. doi: 10.1186/s13023-020-01451-3.
Urinary dysfunction is one of the main features of human T-cell leukemia virus type 1-associated myelopathy/tropical spastic paraparesis (HAM/TSP). However, a comprehensive assessment of the severity is difficult because a standardized assessment measure is unavailable. Therefore, this study aimed to develop a novel symptom score for the assessment of urinary dysfunction in HAM/TSP. We interviewed 449 patients with HAM/TSP using four internationally validated questionnaires for assessment of urinary symptoms (27 question items in total): the International Prostate Symptom Score; the International Consultation on Incontinence Questionnaire-Short Form; the Overactive Bladder Symptom Score; and the Nocturia Quality-of-Life questionnaire. We developed a symptom score based on the data of 322 patients who did not use urinary catheters by selecting question items from questionnaires focused on descriptive statistics, correlation analysis, and exploratory factor analysis. The score distribution, reliability, and validity of the developed score were evaluated.
First, 16 questions related to quality of life, situations, or subjective assessment were omitted from the 27 questions. Exploratory factor analysis revealed that the remaining 11 questions pertained to three factors: frequent urination, urinary incontinence, and voiding symptoms. Three questions, which had similar questions with larger factor loading, were deleted. Finally, we selected eight question items for inclusion in the novel score. The score distribution exhibited no ceiling or floor effect. The Cronbach's alpha (0.737) demonstrated reliable internal consistency. The new score comprised two subscales with acceptable factorial validity (inter-factor correlation coefficient, 0.322): storage symptoms (frequent urination plus urinary incontinence) and voiding symptoms. The correlation between each item and the subscales suggested acceptable construct validity.
We developed a novel score, the HAM/TSP-Bladder Dysfunction Symptom Score, and demonstrated its reliability and validity. The applicability of this score to patients using catheters should be examined in future research.
尿功能障碍是人类 T 细胞白血病病毒 1 型相关脊髓病/热带痉挛性截瘫（HAM/TSP）的主要特征之一。然而，由于缺乏标准化的评估方法，全面评估其严重程度较为困难。因此，本研究旨在开发一种新的 HAM/TSP 尿功能障碍评估症状评分。我们使用四种国际公认的尿症状评估问卷（共 27 个问题项）对 449 例 HAM/TSP 患者进行访谈：国际前列腺症状评分；国际尿失禁咨询问卷-简短形式；膀胱过度活动症症状评分；和夜间多尿生活质量问卷。我们通过从关注描述性统计、相关性分析和探索性因素分析的问卷中选择问题项，为 322 例未使用导尿管的患者开发了一种症状评分。评估了所开发评分的评分分布、可靠性和有效性。
首先，从 27 个问题中删除了与生活质量、情况或主观评估相关的 16 个问题。探索性因素分析显示，其余 11 个问题涉及三个因素：尿频、尿失禁和排尿症状。删除了三个具有较大因子载荷的类似问题的问题。最后，我们选择了 8 个问题纳入新的评分。评分分布无上限或下限效应。Cronbach's alpha（0.737）表现出可靠的内部一致性。新的评分由两个具有可接受因子有效性的子量表组成（因子间相关系数，0.322）：存储症状（尿频加尿失禁）和排尿症状。每个项目与子量表之间的相关性表明具有可接受的结构有效性。
我们开发了一种新的评分，即 HAM/TSP-膀胱功能障碍症状评分，并证明了其可靠性和有效性。在未来的研究中，应检查该评分在使用导管的患者中的适用性。
