Care and Public Health Research Institute (CAPHRI), Maastricht University, Maastricht, The Netherlands.
BMC Neurol. 2011 Jan 25;11:10. doi: 10.1186/1471-2377-11-10.
Epilepsy is a neurological disorder, characterized by recurrent unprovoked seizures which have a high impact on the individual as well as on society as a whole. In addition to the economic burden, epilepsy imposes a substantial burden on the patients and their surroundings. Patients with uncontrolled epilepsy depend heavily on informal care and on health care professionals. About 30% of patients suffer from drug-resistant epilepsy. The ketogenic diet can be a treatment of last resort, especially for children. The beneficial effect of the ketogenic diet has been proven, but information is lacking about its cost-effectiveness. In the current study we will evaluate the (cost-) effectiveness of the ketogenic diet in children and adolescents with intractable epilepsy.
METHODS/DESIGN: In a RCT we will compare the ketogenic diet with usual care. Embedded in this RCT will be a trial-based and model-based economic evaluation, looking from a societal perspective at the cost-effectiveness and cost-utility of the ketogenic diet versus usual care. Fifty children and adolescents (aged 1-18) with intractable epilepsy will be screened for eligibility before randomization into the intervention or the usual care group. The primary outcome measure is the proportion of children with a 50% or more reduction in seizure frequency. Secondary outcomes include seizure severity, side effects/complaints, neurocognitive, socio-emotional functioning, and quality of life. Costs and productivity losses will be assessed continuously by a prospective diary and a retrospective questionnaire. Measurements will take place during consults at baseline, at 6 weeks and at 4 months after the baseline period, and 3, 6, 9 and 12 months follow-up after the 4 months consult.
The proposed research project will be the first study to provide data about the cost-effectiveness of the ketogenic diet for children and adolescents with intractable epilepsy, in comparison with usual care. It is anticipated that positive results in (cost-) effectiveness of the proposed intervention will contribute to the improvement of treatment for epilepsy in children and adolescents and will lead to a smaller burden to society.
癫痫是一种神经系统疾病，其特征是反复发作的无诱因癫痫发作，对个体和整个社会都有很大的影响。除了经济负担外，癫痫还给患者及其周围环境带来了沉重的负担。未得到控制的癫痫患者严重依赖于非正式护理和医疗保健专业人员。大约 30%的患者患有耐药性癫痫。生酮饮食可能是最后的治疗方法，尤其是对儿童而言。生酮饮食的有益效果已得到证实，但缺乏其成本效益的信息。在当前的研究中，我们将评估生酮饮食对难治性癫痫儿童和青少年的（成本）效果。
方法/设计：我们将在一项 RCT 中比较生酮饮食与常规护理。这项 RCT 将嵌入一项基于试验和基于模型的经济评估，从社会角度评估生酮饮食与常规护理相比的成本效益和成本效用。在随机分组到干预组或常规护理组之前，将对 50 名患有难治性癫痫的儿童和青少年进行入选资格筛查。主要结局指标是癫痫发作频率减少 50%或更多的儿童比例。次要结局包括癫痫严重程度、副作用/投诉、神经认知、社会情感功能和生活质量。通过前瞻性日记和回顾性问卷连续评估成本和生产力损失。测量将在基线时、第 6 周和基线后 4 个月进行，然后在第 4 个月咨询后 3、6、9 和 12 个月进行随访。
拟议的研究项目将是第一项比较生酮饮食与常规护理治疗难治性癫痫儿童和青少年的成本效益的研究。预计该干预措施在（成本）效果方面的积极结果将有助于改善儿童和青少年癫痫的治疗，并减轻社会负担。
