Caregiver Perspective of Benefits and Side Effects of Anti-Seizure Medications in CDKL5 Deficiency Disorder from an International Database.

IF 7.4 2区 医学 Q1 CLINICAL NEUROLOGY CNS drugs Pub Date : 2024-09-01 Epub Date: 2024-07-26 DOI:10.1007/s40263-024-01105-z
Kingsley Wong, Mohammed Junaid, Solomon Alexander, Heather E Olson, Elia M Pestana-Knight, Rajsekar R Rajaraman, Jenny Downs, Helen Leonard
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Abstract

Background and objective: CDKL5 deficiency disorder presents as a challenging condition with early-onset refractory seizures, severe developmental delays, and a range of other neurological symptoms. Our study aimed to explore the benefits and side effects of anti-seizure medications (ASMs) in managing seizures among individuals with CDKL5 deficiency disorder, drawing on data from the International CDKL5 Disorder Database.

Methods: Data for this retrospective cohort study were obtained from the International CDKL5 Disorder Database, which contains responses from a baseline questionnaire administered between 2012 and 2022 and a follow-up questionnaire administered between 2018 and 2019. Families of eligible individuals were asked to provide information on ASMs that were previously and currently taken, the dose prescribed, the age at starting the medications, and the age at discontinuation for past medications. The outcome variables of interest were perceived seizure-related benefits for the current and past use of ASMs and caregiver-reported side effects. Rescue medications and infrequently used ASMs were excluded from the analysis. Descriptive statistics were used to summarise the characteristics of the study population.

Results: The study included 399 children and adults with CDKL5 deficiency disorder, descriptively analysing the perceived benefits and side effects of 23 unique ASMs based on caregiver reports. The study identified levetiracetam, topiramate, sodium valproate, vigabatrin, phenobarbital and clobazam as the most used ASMs. Notably, cannabidiol showed highly beneficial outcomes with few side effects, whereas levetiracetam and phenobarbital exhibited less favourable benefit-to-side-effect ratios. Dual therapy involving sodium valproate and levetiracetam was only used a small number (n = 5) of times but appeared effective in reducing seizure activity with relatively few side effects. Compared with monotherapy, polytherapy had a relatively higher likelihood of reported side effects than benefits.

Conclusions: The study, leveraging a large sample size that exceeds that of previous research, emphasises the complex nature of seizure management in CDKL5 deficiency disorder. Our findings underscore the necessity of ongoing research to optimise treatment strategies, considering both the efficacy of seizure control and the potential for adverse effects. The study also points to the need for future investigations into the therapeutic potential of emerging treatments such as ganaxolone and the unresolved efficacy of cannabis products in seizure management.

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从国际数据库看护理人员对 CDKL5 缺乏症患者服用抗癫痫药物的益处和副作用的看法。
背景和目的:CDKL5 缺乏症是一种具有挑战性的疾病,患者早期会出现难治性癫痫发作、严重发育迟缓以及一系列其他神经系统症状。我们的研究旨在利用CDKL5缺乏症国际数据库的数据,探讨抗癫痫药物(ASM)在控制CDKL5缺乏症患者癫痫发作方面的益处和副作用:这项回顾性队列研究的数据来自国际CDKL5障碍数据库,该数据库包含2012年至2022年期间进行的基线问卷调查和2018年至2019年期间进行的后续问卷调查的答复。符合条件者的家属被要求提供有关以前和现在服用的 ASMs、处方剂量、开始服药的年龄以及过去停药的年龄等信息。我们关注的结果变量是当前和过去使用的 ASMs 对癫痫发作的相关益处以及护理人员报告的副作用。分析中不包括急救药物和不经常使用的 ASM。描述性统计用于总结研究人群的特征:该研究纳入了399名患有CDKL5缺乏症的儿童和成人,根据护理人员的报告,对23种独特的ASM的益处和副作用进行了描述性分析。研究发现,左乙拉西坦、托吡酯、丙戊酸钠、维格巴曲林、苯巴比妥和氯巴扎姆是使用最多的ASMs。值得注意的是,大麻二酚的疗效显著且副作用较小,而左乙拉西坦和苯巴比妥的疗效与副作用比率则不太理想。丙戊酸钠和左乙拉西坦的双重疗法只使用了少量(n = 5),但似乎能有效减少癫痫发作活动,且副作用相对较少。与单药疗法相比,多药疗法的副作用报告率相对高于益处报告率:这项研究的样本量超过了以往研究的样本量,强调了 CDKL5 缺乏症癫痫发作治疗的复杂性。我们的研究结果突出表明,有必要持续开展研究,以优化治疗策略,同时考虑癫痫发作控制的疗效和潜在的不良反应。这项研究还指出,今后有必要对新出现的治疗方法(如甘纳唑酮)的治疗潜力以及大麻产品在癫痫发作控制方面尚未解决的疗效进行调查。
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来源期刊
CNS drugs
CNS drugs 医学-精神病学
CiteScore
12.00
自引率
3.30%
发文量
82
审稿时长
6-12 weeks
期刊介绍: CNS Drugs promotes rational pharmacotherapy within the disciplines of clinical psychiatry and neurology. The Journal includes: - Overviews of contentious or emerging issues. - Comprehensive narrative reviews that provide an authoritative source of information on pharmacological approaches to managing neurological and psychiatric illnesses. - Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. - Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in neurology and psychiatry. - Original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in CNS Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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