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鞘内注射巴氯芬治疗儿童难治性痉挛型脑瘫:成本效益分析

Intrathecal baclofen therapy in children with intractable spastic cerebral palsy: a cost-effectiveness analysis.

作者信息

Hoving M A, Evers S M A A, Ament A J H A, van Raak E P M, Vles J S H

机构信息

Department of Neurology, University Hospital Maastricht, Maastricht, The Netherlands.

出版信息

Dev Med Child Neurol. 2008 Jun;50(6):450-5. doi: 10.1111/j.1469-8749.2008.02059.x. Epub 2008 Apr 14.

Abstract

In a Dutch national study, we recently established the effectiveness and safety of continuous intrathecal baclofen infusion (CITB) in children with intractable spastic cerebral palsy (CP). Because prospective studies on the cost-effectiveness of CITB in children with spastic CP are lacking, we conducted a cost-effectiveness analysis alongside our prospective national study. We compared the costs and health effects of CITB with those of standard treatment only, from the health care perspective for a 1-year period. Health effects were expressed in terms of a visual analogue scale for individual problems and quality-adjusted life years (QALYs). We included eight females and seven males, aged between 7 and 17 years (mean age 13y 8mo [SD 3y]). Eleven children had spastic CP and four had spastic-dyskinetic CP. One child was clsssified on the Gross Motor Function Classification System at Level III, two at Level IV, and 12 at Level V. CITB was more effective and more costly than standard treatment only. Gaining one QALY cost on average 32,737 euros. We conclude that based on the threshold-willingness to pay for one QALY in the Netherlands (80,000 euros), our results confirm the cost-effectiveness of CITB for carefully selected children with intractable spastic CP.

摘要

在一项荷兰全国性研究中,我们最近确定了鞘内持续注入巴氯芬(CITB)治疗难治性痉挛型脑瘫(CP)患儿的有效性和安全性。由于缺乏关于CITB治疗痉挛型CP患儿成本效益的前瞻性研究,我们在全国性前瞻性研究的同时进行了成本效益分析。从医疗保健角度,我们比较了CITB与仅采用标准治疗在1年期间的成本和健康效果。健康效果用个体问题的视觉模拟量表和质量调整生命年(QALYs)表示。我们纳入了8名女性和7名男性,年龄在7至17岁之间(平均年龄13岁8个月[标准差3岁])。11名儿童为痉挛型CP,4名儿童为痉挛 - 运动障碍型CP。1名儿童在粗大运动功能分类系统中为III级,2名儿童为IV级,12名儿童为V级。CITB比仅采用标准治疗更有效且成本更高。获得1个QALY平均花费32,737欧元。我们得出结论,基于荷兰为1个QALY支付意愿的阈值(80,000欧元),我们的结果证实了CITB对于精心挑选的难治性痉挛型CP患儿具有成本效益。

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