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开发生长激素释放激素(GHRH)类似物治疗特发性矮小症的临床试验

The long term effectiveness and cost-effectiveness of initiating treatment for ocular hypertension.

机构信息

University Eye Clinic Maastricht, University Medical Center, Maastricht, the Netherlands.

出版信息

Acta Ophthalmol. 2014 Sep;92(6):513-23. doi: 10.1111/aos.12328. Epub 2013 Dec 16.

Abstract

PURPOSE

To investigate the long-term health and economic consequences of direct treatment initiation in ocular hypertension patients.

METHODS

A cost-effectiveness analysis with a societal perspective and a lifelong horizon was performed. The primary outcomes were the incremental quality-adjusted life years (QALYs) and costs of direct pressure-lowering treatment for ocular hypertension, compared to a strategy where treatment is postponed until conversion to glaucoma has been observed. We used a decision analytic model based on individual patient simulation to forecast disease progression and treatment decisions in both strategies in a representative heterogeneous patient population and in 18 patient subgroups stratified by initial intraocular pressure and additional risk factors for conversion.

RESULTS

The incremental discounted health gain of direct treatment was 0.27 QALYs, whereas the incremental discounted costs were -€ 649 during an average lifetime of 26 years. In the simulations of patient subgroups, the model outcomes moved towards higher health gains and lower incremental costs with increasing risk of conversion in the patient population. The incremental cost-effectiveness ratio of direct treatment ranged from € 15,425 per QALY gained in the lowest-risk subgroup to dominance in the highest-risk subgroup. Probabilistic sensitivity analysis indicated that uncertainty surrounding the model input parameters did not affect the conclusions.

CONCLUSION

Direct, early, pressure-lowering treatment is a dominant cost-effective treatment strategy over a strategy to start the same treatment approach later, after glaucoma has occurred for patients with ocular hypertension. Its implementation and consequences should be discussed with ophthalmologists and individual patients.

摘要

目的

探讨开角型青光眼患者直接降压治疗的长期健康和经济后果。

方法

采用基于社会视角和终身视角的成本效益分析。主要结果是与观察到青光眼转化后再进行降压治疗的策略相比,开角型青光眼患者直接降压治疗的增量质量调整生命年(QALYs)和成本。我们使用基于个体患者模拟的决策分析模型,在代表性的异质患者人群中以及根据初始眼内压和转化的其他危险因素分层的 18 个患者亚组中,预测两种策略下的疾病进展和治疗决策。

结果

直接治疗的增量折扣健康收益为 0.27 QALYs,而在 26 年的平均寿命内,增量折扣成本为-649 欧元。在患者亚组的模拟中,随着患者人群中转化风险的增加,模型结果朝着更高的健康收益和更低的增量成本方向发展。直接治疗的增量成本效益比在风险最低的亚组中每获得 1 个 QALY 的成本为 15425 欧元,在风险最高的亚组中则具有优势。概率敏感性分析表明,模型输入参数的不确定性并不影响结论。

结论

对于开角型青光眼患者,与在青光眼发生后再开始相同治疗方法的策略相比,直接、早期的降压治疗是一种具有成本效益的优势治疗策略。应该与眼科医生和个体患者讨论其实施和后果。

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