Department of Psychiatry, Social Psychiatry and Psychotherapy, Hannover Medical School, Carl-Neuberg-Straße 1, 30625, Hannover, Germany.
Orphanet J Rare Dis. 2022 Aug 26;17(1):324. doi: 10.1186/s13023-022-02470-y.
Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder based on a loss of paternally expressed genes in chromosome segment 15q11-13. Behavioral traits such as temper outbursts, stereotypic, and ritualistic behavior, as well as an increased risk of psychosis accompany the syndrome, representing a major issue in the treatment of adults with PWS. Up to now, no treatment guideline for these conditions in PWS exist. This study aimed to retrospectively analyze the effect and adverse effects of treatment with aripiprazole for temper outbursts in 10 adults with PWS.
Aripiprazole was prescribed for temper outbursts (n = 10). Treatment outcome was assessed using the Clinical Global Impression-Severity (CGI-S) and -Improvement Scale (CGI-I). Treatment success (CGI-I < 3) was observed in 70% of cases, with adverse effects from mild to partly serious extent in 60% of cases. The major adverse effect observed was increased daytime sleepiness. In total, 50% of the individuals were treated successfully for temper outbursts. The BMI did not change significantly in the successfully treated group after 6 months of treatment.
Aripiprazole can be a treatment option for temper outbursts in people with PWS. Although a high rate of side effects was detected, their severity led to discontinuation in only 20% of the cases. Furthermore, the absence of weight gain makes aripiprazole interesting especially for the PWS population.
普拉德-威利综合征(PWS)是一种罕见的神经发育障碍,源于 15q11-13 号染色体片段上父源表达基因的缺失。该综合征伴有行为特征,如脾气爆发、刻板和仪式性行为,以及精神病风险增加,这是 PWS 成年患者治疗的主要问题。到目前为止,PWS 患者的这些病症还没有治疗指南。本研究旨在回顾性分析阿立哌唑治疗 10 例 PWS 患者脾气爆发的疗效和不良反应。
阿立哌唑被开用于治疗脾气爆发(n=10)。使用临床总体印象严重程度(CGI-S)和改善量表(CGI-I)评估治疗效果。70%的病例治疗效果良好(CGI-I<3),60%的病例出现轻度至中度不良反应。主要观察到的不良反应是白天嗜睡增加。总共有 50%的个体成功地接受了脾气爆发的治疗。在治疗 6 个月后,成功治疗组的 BMI 没有明显变化。
阿立哌唑可以作为 PWS 患者脾气爆发的治疗选择。尽管检测到较高的副作用发生率,但只有 20%的病例因副作用严重而停药。此外,体重增加不明显使得阿立哌唑特别适合 PWS 人群。