新型抗癫痫药物吡仑帕奈治疗癫痫患儿的安全性评价

Cost-effectiveness analysis on 5 strategies of treatment for schizophrenia in China

目的:评估新型抗癫痫药物吡仑帕奈(perampanel, PER)添加治疗癫痫患儿的安全性。方法:回顾性收集并评估癫痫患儿在吡仑帕奈治疗3个月后的安全性。以所有患儿癫痫治疗期间任何一种不良事件的类型和频率来评价吡仑帕奈的安全性。结果:本研究共纳入88例癫痫患儿。在整个PER治疗期间,41例(46.59%)癫痫患儿至少经历了1次药物不良反应。最常见的不良反应为嗜睡(26.14%)、头晕(19.32%)、精神行为异常(9.09%)和恶心/呕吐(4.55%)。不良反应导致4例患儿停止使用PER。服用PER后发生药物不良反应组患儿的血浆药物浓度显著高于未发生不良反应组患儿[(0.56±0.46)μg·mL~(-1) vs (0.39±0.27)μg·mL~(-1),P<0.05]。PER血浆谷浓度>0.98μg·mL~(-1)的癫痫患儿在发生药物不良反应组中的比例显著高于未发生不良反应组(21.95%vs 4.26%,P<0.05)。结论:PER的安全性较差,发生不良反应的比例较高,在用药过程中随时监测其血浆药物浓度非常有必要。

Objective:To evaluate the cost-effectiveness of different strategies of treatment for schizophrenia and to provide evidence for clinical decision-making in China.Methods:A Markov model with three health states(stable,relapse,and dead)was developed to simulate the progression of a cohort of patients with schizophrenia.From the perspective of healthcare system,the medication costs and inpatient costs were collected.The cumulative costs and health outcomes over 5 years were evaluated for the treatment strategies with daily oral administration(Olanzapine),as well as one injection every half(Risperidone for Depot Suspension),one(Paliperidone Palmitate Injection 1M),three(Paliperidone Palmitate Injection 3M),and six months(Paliperidone Palmitate Injection 6M).The incremental cost-effectiveness ratios were calculated for the comparison of different strategies for treating schizophrenia.The robustness of results was assessed by sensitivity analyses.Results:Daily oral administration was the least costly,while every-l-month injection was the most effective.The strategies of giving one injection every half month and every six months were the more costly and less effective than every-l-month injection strategy.The cost per QALY gained was 66078.9 CNY for every-3-month injection strategy and that for every-1-month injection strategy was 143417.8 CNY,compared with the daily oral medication.Sensitivity analysis suggested good reliability of the model outcomes.Conclusion:With the threshold of three times per capita Gross Domestic Product(257094.0 CNY)in 2022 in China,the treatment strategy of giving every-1-month injection strategy and every-3-month injection strategy are cost-effective,among which the strategy of giving once every three months is the most cost-effective.The results might provide evidence for clinical treatment of schizophrenia in China.