Abstract：
BACKGROUND: Trihexyphenidyl and clonazepam are commonly used to treat dystonia in children with cerebral palsy (CP). However, there is a notable gap in the literature when it comes to studies that combine these first-line agents for the management of dystonia.
METHODS: This open-label, randomized controlled trial aimed to compare the efficacy of adding oral clonazepam to trihexyphenidyl (THP + CLZ) versus using trihexyphenidyl alone (THP) in reducing the severity of dystonia, as measured by the Barry-Albright Dystonia (BAD) score. The study was conducted over a 12-week therapy period in children with dystonic CP aged two to 14 years.
RESULTS: Each group enrolled 51 participants. The THP + CLZ group showed significantly better improvement in dystonia severity at 12 weeks compared with the THP group alone (-4.5 ± 2.9 vs -3.4 ± 1.7, P = 0.02). Furthermore, the THP + CLZ group exhibited superior improvement in the severity of choreoathetosis, upper limb function, pain perception by the child, and quality of life, with P values of 0.02, 0.009, 0.01, and 0.01, respectively. The number of participants experiencing treatment-emergent adverse events was comparable in both groups (P = 0.67). Importantly, none of the participants in any of the groups reported any serious adverse events.
CONCLUSIONS: A combination of oral THP + CLZ proves to be more efficacious than using THP alone for the treatment of dystonic CP in children aged two to 14 years in terms of reducing the severity of dystonia.
METHODS: This open-label, randomized controlled trial aimed to compare the efficacy of adding oral clonazepam to trihexyphenidyl (THP + CLZ) versus using trihexyphenidyl alone (THP) in reducing the severity of dystonia, as measured by the Barry-Albright Dystonia (BAD) score. The study was conducted over a 12-week therapy period in children with dystonic CP aged two to 14 years.
RESULTS: Each group enrolled 51 participants. The THP + CLZ group showed significantly better improvement in dystonia severity at 12 weeks compared with the THP group alone (-4.5 ± 2.9 vs -3.4 ± 1.7, P = 0.02). Furthermore, the THP + CLZ group exhibited superior improvement in the severity of choreoathetosis, upper limb function, pain perception by the child, and quality of life, with P values of 0.02, 0.009, 0.01, and 0.01, respectively. The number of participants experiencing treatment-emergent adverse events was comparable in both groups (P = 0.67). Importantly, none of the participants in any of the groups reported any serious adverse events.
CONCLUSIONS: A combination of oral THP + CLZ proves to be more efficacious than using THP alone for the treatment of dystonic CP in children aged two to 14 years in terms of reducing the severity of dystonia.
摘要：
背景：三己苯基和氯硝西泮通常用于治疗脑瘫（CP）儿童的肌张力障碍。然而,在结合这些一线药物治疗肌张力障碍的研究方面,文献中存在显著差距.
方法：这个开放标签，随机对照试验旨在比较口服氯硝西泮与三己苯基(THP+CLZ)与单用三己苯基(THP)在减轻肌张力障碍严重程度方面的疗效,根据Barry-Albright肌张力障碍(BAD)评分。这项研究是在2至14岁的肌张力障碍性CP儿童中进行的，为期12周的治疗期。
结果：每组共纳入51名参与者。与单独使用THP组相比，THP+CLZ组在12周时的肌张力障碍严重程度显着改善（-4.5±2.9vs-3.4±1.7，P=0.02）。此外，THP+CLZ组表现出较好的改善，上肢功能,孩子的疼痛感知，和生活质量，P值分别为0.02、0.009、0.01和0.01。两组中出现治疗紧急不良事件的参与者数量相当(P=0.67)。重要的是,任何组的参与者均未报告任何严重不良事件.
结论：在减轻肌张力障碍严重程度方面，口服THP+CLZ的组合被证明比单独使用THP治疗2至14岁儿童的肌张力障碍CP更有效。
方法：这个开放标签，随机对照试验旨在比较口服氯硝西泮与三己苯基(THP+CLZ)与单用三己苯基(THP)在减轻肌张力障碍严重程度方面的疗效,根据Barry-Albright肌张力障碍(BAD)评分。这项研究是在2至14岁的肌张力障碍性CP儿童中进行的，为期12周的治疗期。
结果：每组共纳入51名参与者。与单独使用THP组相比，THP+CLZ组在12周时的肌张力障碍严重程度显着改善（-4.5±2.9vs-3.4±1.7，P=0.02）。此外，THP+CLZ组表现出较好的改善，上肢功能,孩子的疼痛感知，和生活质量，P值分别为0.02、0.009、0.01和0.01。两组中出现治疗紧急不良事件的参与者数量相当(P=0.67)。重要的是,任何组的参与者均未报告任何严重不良事件.
结论：在减轻肌张力障碍严重程度方面，口服THP+CLZ的组合被证明比单独使用THP治疗2至14岁儿童的肌张力障碍CP更有效。
