Pasco Paul Matthew D, Jamora Roland Dominic G, Rosales Raymond L, Diesta Cid Czarina E, Ng Arlene R, Teleg Rosalia A, Go Criscely L, Lee Lillian, Fernandez Hubert H
Department of Neurosciences, College of Medicine-Philippine General Hospital, University of the Philippines Manila, Manila, Philippines.
Child Neuroscience Center, Philippine Children's Medical Center, Quezon City, Philippines.
NPJ Parkinsons Dis. 2017 Jul 25;3:24. doi: 10.1038/s41531-017-0026-0. eCollection 2017.
X-linked dystonia-parkinsonism(XDP) is a neurodegenerative disorder endemic to the Philippines. A rating scale was developed by the authors under the guidance of the Movement Disorder Society of the Philippines (MDSP) to assess XDP severity and progression, functional impact, and response to treatment in future clinical trials. Our main objective was to validate our new scale, the XDP-MDSP scale. The initial validation process included pragmatic testing to XDP patients followed by a modified Delphi procedure with an international advisory panel of dystonia, parkinsonism and scale development experts. Pearson correlation was used to assess construct validity of our new scale versus the assess construct validity of our new scale versus standard dystonia, parkinsonism, non-motor and functional scales; and also to assess divergent validity against behavioral and cognitive scales. The 37-item XDP-MDSP scale has five parts: I-dystonia, II-parkinsonism, III-non-motor features, IV-ADL, and V-global impression. After initial validation, the scale was administered to 204 XDP patients. Inter-domain correlation for the first four parts was acceptable. The correlation between these domains and the global rating was slightly lower. Correlations between Parts I, II, III, and IV versus standard dystonia, parkinsonism, non-motor and functional scales were acceptable with values ranging from 0.323 to 0.428. For divergent validity, a significant correlation was seen with behavioral scales. No significant correlation was noted with the cognitive scale. The proposed XDP-MDSP scale is internally valid but the global rating subscale may need to be modified or eliminated. While there is convergent validity, divergent validation was successful only on cognitive and not behavioral scales. The frequent co-occurrence of anxiety and depression, and its effect on the motor and functional state, may explain this finding.
X连锁肌张力障碍-帕金森综合征（XDP）是菲律宾特有的一种神经退行性疾病。作者在菲律宾运动障碍协会（MDSP）的指导下制定了一种评定量表，用于评估XDP的严重程度和进展、功能影响以及在未来临床试验中的治疗反应。我们的主要目标是验证我们新的量表——XDP-MDSP量表。初始验证过程包括对XDP患者进行实际测试，随后与肌张力障碍、帕金森综合征及量表开发专家组成的国际咨询小组进行改良德尔菲程序。采用Pearson相关性分析来评估我们新量表与标准肌张力障碍、帕金森综合征、非运动和功能量表的结构效度；同时评估与行为和认知量表的区分效度。这个包含37个条目的XDP-MDSP量表有五个部分：I-肌张力障碍、II-帕金森综合征、III-非运动特征、IV-日常生活活动能力以及V-整体印象。经过初始验证后，该量表应用于204例XDP患者。前四个部分的领域间相关性是可以接受的。这些领域与整体评分之间的相关性略低。第一、二、三、四部分与标准肌张力障碍、帕金森综合征、非运动和功能量表之间的相关性可以接受，值在0.323至0.428之间。对于区分效度，与行为量表有显著相关性。与认知量表未发现显著相关性。所提出的XDP-MDSP量表内部有效，但整体评分子量表可能需要修改或删除。虽然有聚合效度，但区分效度仅在认知量表上成功，行为量表上未成功。焦虑和抑郁的频繁共现及其对运动和功能状态的影响可能解释了这一发现。
