de Kinderen Reina J A, Postulart Debby, Aldenkamp Albert P, Evers Silvia M A A, Lambrechts Danielle A J E, Louw Anton J A de, Majoie Marian H J M, Grutters Janneke P C
Department of Health Services Research, CAPHRI School for Public Health and Primary Care, Maastricht University, Maastricht, The Netherlands; Department of Research and Development, Epilepsy Center Kempenhaeghe, Heeze, The Netherlands; MHENS School of Mental Health & Neuroscience, Maastricht University, Maastricht, The Netherlands.
Department of Research and Development, Epilepsy Center Kempenhaeghe, Heeze, The Netherlands.
Epilepsy Res. 2015 Feb;110:119-31. doi: 10.1016/j.eplepsyres.2014.12.005. Epub 2014 Dec 13.
The objective of this study was to estimate the expected cost-utility and cost-effectiveness of the ketogenic diet (KD), vague nerve stimulation (VNS) and care as usual (CAU), using a decision analytic model with a 5-year time horizon.
A Markov decision analytical model was constructed to estimate the incremental costs, quality-adjusted life years (QALYs) and successfully treated patient (i.e. 50% or more seizure reduction) of the treatment strategies KD, VNS and CAU, from a health care perspective. The base case considered children with intractable epilepsy (i.e. two or more antiepileptic drugs had failed) aged between 1 and 18 years. Data were derived from literature and expert meetings. Deterministic and probabilistic sensitivity analyses were performed.
Our results suggest that KD is more effective and less costly, and thus cost-effective compared with VNS, after 12 months. However, compared to CAU, neither KD nor VNS are cost-effective options, they are both more effective but also more expensive (€346,899 and €641,068 per QALY, respectively). At 5 years, VNS is cost-effective compared with KD and CAU (€11,378 and €68,489 per QALY, respectively) and has a 51% probability of being cost-effective at a ceiling ratio of €80,000 per QALY.
Our results suggest that on average the benefits of KD and VNS fail to outweigh the costs of the therapies. However, these treatment options should not be ignored in the treatment for intractable epilepsy in individual or specific groups of patients. There is a great need for high quality comparative studies with large patient samples which allow for subgroup analyses, long-term follow-up periods and outcome measures that measure effects beyond seizure frequency (e.g. quality of life). When this new evidence becomes available, reassessment of the cost-effectiveness of KD and VNS in children with intractable epilepsy should be carried out.
本研究的目的是使用一个为期5年的决策分析模型，评估生酮饮食（KD）、迷走神经刺激（VNS）和常规护理（CAU）的预期成本效用和成本效益。
构建一个马尔可夫决策分析模型，从医疗保健角度估计KD、VNS和CAU治疗策略的增量成本、质量调整生命年（QALY）和成功治疗的患者（即癫痫发作减少50%或更多）。基础病例为1至18岁的难治性癫痫儿童（即两种或更多抗癫痫药物治疗失败）。数据来源于文献和专家会议。进行了确定性和概率敏感性分析。
我们的结果表明，12个月后，KD比VNS更有效且成本更低，因此具有成本效益。然而，与CAU相比，KD和VNS都不是具有成本效益的选择，它们都更有效但也更昂贵（分别为每QALY 346,899欧元和641,068欧元）。在5年时，VNS与KD和CAU相比具有成本效益（分别为每QALY 11,378欧元和68,489欧元），并且在每QALY 80,000欧元的上限比率下具有51%的成本效益概率。
我们的结果表明，平均而言，KD和VNS的益处未能超过治疗成本。然而，在个体或特定患者群体的难治性癫痫治疗中，这些治疗选择不应被忽视。非常需要进行高质量的比较研究，样本量大，允许进行亚组分析、长期随访期以及测量癫痫发作频率以外的效果的结局指标（例如生活质量）。当获得这些新证据时，应重新评估KD和VNS在难治性癫痫儿童中的成本效益。
