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儿童急性特发性血小板减少性紫癜(ITP)治疗的成本效用分析。

A cost-utility analysis of treatment for acute childhood idiopathic thrombocytopenic purpura (ITP).

作者信息

O'Brien Sarah H, Ritchey A Kim, Smith Kenneth J

机构信息

Pediatric Hematology/Oncology, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania 15213, USA.

出版信息

Pediatr Blood Cancer. 2007 Feb;48(2):173-80. doi: 10.1002/pbc.20830.

Abstract

BACKGROUND

The primary objective in the treatment of acute pediatric idiopathic thrombocytopenic purpura (ITP) is to rapidly increase the platelet count.

METHODS

We built a decision analytic model to evaluate the cost-utility of four commonly used treatment strategies: intravenous immunoglobulin G (IVIG) 0.8 g/kg, anti-D 75 mcg/kg, methylprednisolone (30 mg/kg for 3 days), and prednisone (4 mg/kg/day for 4 days). In our baseline model, all children were hospitalized upon presentation, and discharged once the platelet count reached > or =20,000. We performed a literature search to estimate time to platelet count > or =20,000 for each strategy, as well as the probability of side effects. We obtained cost data and quality of life measures from institutional and published data sources.

RESULTS

Total cost of one-time treatment for a 20 kg child was US dollars 786 with prednisone, US dollars 1,346 with methylprednisolone, US dollars 2,035 with anti-D, and US dollars 2,492 with IVIG. The strategies of IVIG and methylprednisolone were less effective and more expensive than anti-D and prednisone, respectively. Although anti-D caused the most rapid rise in platelet counts, the incremental cost-utility ratio (costs incurred by using anti-D instead of prednisone divided by health benefit of using anti-D instead of prednisone) was US dollars 7,616 per day of severe thrombocytopenia avoided, primarily due to the much higher medication cost of anti-D. Utilizing an outpatient model, the cost difference between anti-D and prednisone was even more striking.

CONCLUSIONS

The clinical benefit of anti-D is offset by a substantial cost increase. Although often overlooked in favor of newer agents, a brief course of high-dose prednisone is an inexpensive and effective treatment for acute ITP.

摘要

背景

治疗儿童急性特发性血小板减少性紫癜(ITP)的主要目标是迅速提高血小板计数。

方法

我们构建了一个决策分析模型,以评估四种常用治疗策略的成本效益:静脉注射免疫球蛋白G(IVIG)0.8 g/kg、抗-D 75 mcg/kg、甲泼尼龙(30 mg/kg,连用3天)和泼尼松(4 mg/kg/天,连用4天)。在我们的基线模型中,所有儿童就诊时均住院,血小板计数达到≥20,000时出院。我们进行了文献检索,以估计每种策略使血小板计数达到≥20,000的时间以及出现副作用的概率。我们从机构和已发表的数据来源获取成本数据和生活质量指标。

结果

一名20 kg儿童一次性治疗的总成本,泼尼松为786美元,甲泼尼龙为1,346美元,抗-D为2,035美元,IVIG为2,492美元。IVIG和甲泼尼龙策略分别比抗-D和泼尼松效果更差且成本更高。尽管抗-D使血小板计数上升最快,但增量成本效益比(使用抗-D而非泼尼松产生的成本除以使用抗-D而非泼尼松带来的健康效益)为每避免一天严重血小板减少症7,616美元,主要是因为抗-D的药物成本高得多。采用门诊模型时,抗-D和泼尼松之间的成本差异更为显著。

结论

抗-D的临床益处被成本大幅增加所抵消。尽管高剂量泼尼松的短期疗程常因青睐新药物而被忽视,但它是治疗急性ITP的一种廉价且有效的方法。

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