Scalise Roberta, Sgandurra Giuseppina, Menici Valentina, Capodagli Nicola, Di Pietro Roberta, Romeo Domenico M, Sini Francesca, Pagliano Emanuela, Foscan Maria, Cioni Giovanni, Battini Roberta
Department of Developmental Neuroscience, IRCCS Fondazione Stella Maris, Pisa, Italy.
Tuscan PhD Programme of Neuroscience, University of Florence, Pisa and Siena, Florence, Italy.
Front Neurol. 2021 Feb 26;12:612429. doi: 10.3389/fneur.2021.612429. eCollection 2021.
Tetrabenazine has been studied with a variety of hyperkinetic movement disorders, but there is limited and empirical literature on the potential efficacy of tetrabenazine in children with dyskinetic cerebral palsy (DCP). The purpose of this study was to evaluate the efficacy of tetrabenazine in a sample of children with DCP using the Movement Disorders-Childhood Rating Scale 4-18 Revised (MD-CRS 4-18 R). The study is a multicenter retrospective longitudinal study in which the participants were selected from the databases of each Center involved, according to detailed inclusion criteria. The study was performed on 23 children and adolescents (19 male and 4 females; mean age 8.28 years, SD 3.59) with DCP having been evaluated before starting the treatment (baseline), after 6 and 12 months of treatment and in a sub-cohort after >2 years follow-up. A linear mixed model was used to evaluate the effects of the different timings on each MD-CRS 4-18 R Index (Index I, Index II, and Global Index) adding age and type of movement disorder as random effect. A significant clinical improvement related to a reduction of MD-CRS 4-18 R Indexes was detected between the baseline and after 6 and 12 months of treatment. Findings support the efficacy of tetrabenazine in children with DCP through a standardized outcome measure (MD-CRS 4-18 R) and confirm the use of this scale as a suitable tool to detect changes in further randomized clinical trials.
丁苯那嗪已被用于多种运动亢进性运动障碍的研究，但关于丁苯那嗪对运动障碍型脑性瘫痪（DCP）患儿潜在疗效的文献有限且多为经验性报道。本研究旨在使用修订版儿童运动障碍评定量表4 - 18（MD - CRS 4 - 18 R）评估丁苯那嗪对一组DCP患儿的疗效。该研究是一项多中心回顾性纵向研究，根据详细的纳入标准从各参与中心的数据库中选取参与者。研究对象为23名患有DCP的儿童和青少年（19名男性，4名女性；平均年龄8.28岁，标准差3.59），在治疗开始前（基线）、治疗6个月和12个月后以及>2年随访后的一个亚组中进行评估。使用线性混合模型评估不同时间点对每个MD - CRS 4 - 18 R指数（指数I、指数II和总指数）的影响，并将年龄和运动障碍类型作为随机效应纳入。在基线与治疗6个月和12个月后之间，检测到与MD - CRS 4 - 18 R指数降低相关的显著临床改善。研究结果通过标准化结局测量（MD - CRS 4 - 18 R）支持了丁苯那嗪对DCP患儿的疗效，并证实该量表可作为进一步随机临床试验中检测变化的合适工具。
