Pons Roser, Pearson Toni S, Perez-Dueñas Belen, Garcia-Cazorla Angels, Kurian Manju A, Dalivigka Zoi, Zouvelou Vasiliki, Outsika Chrysa, Kokkinou Eleftheria, Sigatullina-Bondarenko Maria, Darling Alejandra, O'Callaghan Maria Del Mar, Spaull Robert, Steel Dora B D, Salamou Evdokia, Forjaz Maria João, Rodriguez-Blazquez Carmen
First Department of Pediatrics, Agia Sofia Children's Hospital, National and Kapodistrian University of Athens, Athens, Greece.
Division of Neurology, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio, USA.
Mov Disord. 2025 Aug;40(8):1669-1679. doi: 10.1002/mds.30219. Epub 2025 May 13.
Parkinsonism in infancy is rare and is highly correlated with the presence of dystonia. Advances in treating and characterizing developmental and infantile degenerative parkinsonism have highlighted the need for a specialized assessment scale.
The aim of this study was to design and validate a scale that effectively assesses parkinsonism-dystonia in early life.
The Infantile Parkinsonism-Dystonia Rating Scale (IPDRS) was designed to capture the key clinical features of parkinsonism-dystonia in early life. It consists of 28 items across three subscales: Non-motor symptoms, Motor symptoms, and Dyskinesias. Thirty-two patients with hypokinetic movement disorder were scored following a standardized protocol. Filmed motor examinations were analyzed independently by three pediatric movement disorders specialists to evaluate interrater reliability. Twenty additional patients with primary neurotransmitter disorders were scored, and nine of them were evaluated at baseline and after treatment. Psychometric validation was conducted.
A total of 52 patients were scored using the IPDRS. Mean age was 3.1 years (standard deviation [SD]: 2.0), and the mean IPDRS score was 40.8 (SD: 13.17). Internal consistency analysis demonstrated a Cronbach's α of 0.21 for Non-motor symptoms subscale, 0.84 for Motor symptoms subscale, and 0.95 for Dyskinesia subscale. Kappa indexes exceeded 0.70 in seven items. Correlation coefficients for dystonia items with the Barry-Albright-Dystonia Scale ranged from 0.46 to 0.64. After treatment, all IPDRS scores changed significantly, with an effect size of 2.42.
The IPDRS appears to be a reliable and valid tool for assessing parkinsonism in early life. Further validation studies with a larger sample size are needed to confirm these findings and complete the validation process. © 2025 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
婴儿期帕金森症较为罕见，且与肌张力障碍的存在高度相关。在发育性和婴儿期退行性帕金森症的治疗和特征描述方面取得的进展凸显了制定专门评估量表的必要性。
本研究旨在设计并验证一种能有效评估早期帕金森症 - 肌张力障碍的量表。
婴儿期帕金森症 - 肌张力障碍评定量表（IPDRS）旨在捕捉早期帕金森症 - 肌张力障碍的关键临床特征。它由28个项目组成，分为三个子量表：非运动症状、运动症状和运动障碍。按照标准化方案对32例运动减少性运动障碍患者进行评分。三位儿科运动障碍专家独立分析拍摄的运动检查结果，以评估评分者间信度。对另外20例原发性神经递质疾病患者进行评分，其中9例在基线和治疗后进行评估。进行了心理测量学验证。
使用IPDRS对52例患者进行了评分。平均年龄为3.1岁（标准差[SD]：2.0），IPDRS平均得分为40.8（SD：13.17）。内部一致性分析显示，非运动症状子量表的Cronbach's α为0.21，运动症状子量表为0.84，运动障碍子量表为0.95。7个项目的Kappa指数超过0.70。肌张力障碍项目与巴里 - 奥尔布赖特肌张力障碍量表的相关系数在0.46至0.64之间。治疗后，所有IPDRS评分均有显著变化，效应大小为2.42。
IPDRS似乎是评估早期帕金森症的可靠且有效的工具。需要进行更大样本量的进一步验证研究以证实这些发现并完成验证过程。© 2025作者。《运动障碍》由Wiley Periodicals LLC代表国际帕金森和运动障碍协会出版。
