Murugupillai Roshini, Ranganathan Shalini Sri, Wanigasinghe Jithangi, Muniyandi Ravi, Arambepola Carukshi
Department of Clinical Sciences, Faculty of Health-Care Sciences, Eastern University Sri Lanka, No:50, New Road, Batticaloa, Sri Lanka.
Department of Pharmacology, Faculty of Medicine, University of Colombo, PO Box 25, Kynsey Road, Colombo 08, Sri Lanka.
Epilepsy Behav. 2018 Mar;80:56-60. doi: 10.1016/j.yebeh.2017.12.011. Epub 2018 Feb 3.
Clinical trials of antiepileptic drugs frequently measure outcomes of seizure control, which demonstrate efficacy. Yet, functional status, quality of life, and long-term treatment effects reflecting effectiveness are scarcely assessed. We sought to use a consensus method to help identify which outcome criteria key stakeholders consider should be used to measure effectiveness in trials of antiepileptic treatments for children.
A two-round Delphi survey was used; parents of children with epilepsy and local, international experts comprising academics and clinicians participated in the survey. In the first round, 32 experts, 50 parents, and 15 children with epilepsy aged >13years suggested outcomes that they considered important in determining effectiveness of antiepileptic therapy in children, separately for preschool and school age. In the second round, 29 experts and 42 parents scored the importance of outcomes from the list suggested by at least 10% of round 1 respondents and also proposed five most important outcomes.
Complete seizure freedom (67%), seizure frequency (48%), ability to perform normal day-to-day activities (45%), and quality of life (40%) were identified as the most important outcomes of antiepileptic therapy in children of both age groups. Additionally, effect on developmental milestones (47%) and child's compliance to treatment regimen (39%) were identified as most important in preschool age group and school performance (49%); adverse effects (39%) were identified as most important in school age group.
For the first time, this study has identified outcome priorities regarding antiepileptic treatment in children based on the key stakeholders' perspectives. It could be used as a provisional list of outcomes for inclusion in a core outcome set for children with epilepsy.
抗癫痫药物的临床试验经常测量癫痫控制的结果，以证明其疗效。然而，反映有效性的功能状态、生活质量和长期治疗效果却很少被评估。我们试图采用一种共识方法，以帮助确定关键利益相关者认为哪些结果标准应被用于衡量儿童抗癫痫治疗的有效性。
采用两轮德尔菲调查；癫痫患儿的家长以及包括学者和临床医生在内的本地和国际专家参与了调查。在第一轮中，32名专家、50名家长和15名年龄大于13岁的癫痫患儿分别就学龄前和学龄儿童抗癫痫治疗有效性判定中他们认为重要的结果提出了建议。在第二轮中，29名专家和42名家长对第一轮中至少10%的受访者所建议清单中的结果重要性进行了评分，并还提出了五项最重要的结果。
完全无癫痫发作（67%）、癫痫发作频率（48%）、进行正常日常活动的能力（45%）和生活质量（40%）被确定为两个年龄组儿童抗癫痫治疗的最重要结果。此外，对发育里程碑的影响（47%）和儿童对治疗方案的依从性（39%）在学龄前儿童组中被确定为最重要的，而学业表现（49%）；不良反应（39%）在学龄儿童组中被确定为最重要的。
本研究首次基于关键利益相关者的观点确定了儿童抗癫痫治疗的结果优先级。它可作为一份临时结果清单，纳入癫痫患儿的核心结局集。
