Afentou Nafsika, Frew Emma, Mehta Samir, Ives Natalie J, Woolley Rebecca L, Brettell Elizabeth A, Khan Adam R, Milford David V, Bockenhauer Detlef, Saleem Moin A, Hall Angela S, Koziell Ania, Maxwell Heather, Hegde Shivaram, Finlay Eric, Gilbert Rodney D, Jones Caroline, McKeever Karl, Cook Wendy, Webb Nicholas J A, Christian Martin T
Health Economics Unit, University of Birmingham, Birmingham, UK.
Birmingham Clinical Trials Unit, University of Birmingham, Birmingham, UK.
Pharmacoecon Open. 2022 Jul;6(4):605-617. doi: 10.1007/s41669-022-00334-6. Epub 2022 Jun 22.
Childhood steroid-sensitive nephrotic syndrome is a frequently relapsing disease with significant short- and long-term complications, leading to high healthcare costs and reduced quality of life for patients. The majority of relapses are triggered by upper respiratory tract infections (URTIs) and evidence shows that daily low-dose prednisolone at the time of infection may reduce the risk of relapse.
The aim of this study was to assess the cost effectiveness of a 6-day course of low-dose prednisolone at the start of a URTI when compared with placebo.
A state-transition Markov model was developed to conduct a cost-utility analysis with the outcome measured in quality-adjusted life-years (QALYs). Resource use and outcome data were derived from the PREDNOS2 trial. The analysis was performed from a UK National Health Service perspective and the results were extrapolated to adulthood. Model parameter and structural uncertainty were assessed using sensitivity analyses.
The base-case results showed that administering low-dose prednisolone at the time of a URTI generated more QALYs and a lower mean cost at 1 year compared with placebo. In the long-term, low-dose prednisolone was associated with a cost saving (£176) and increased effectiveness (0.01 QALYs) compared with placebo and thus remained the dominant treatment option. These findings were robust to all sensitivity analyses.
A 6-day course of low-dose prednisolone at the time of a URTI in children with steroid-sensitive nephrotic syndrome has the potential to reduce healthcare costs and improve quality of life compared with placebo.
儿童类固醇敏感性肾病综合征是一种频繁复发的疾病，伴有严重的短期和长期并发症，导致高昂的医疗费用并降低了患者的生活质量。大多数复发由上呼吸道感染（URTI）引发，且有证据表明在感染时每日服用低剂量泼尼松龙可能降低复发风险。
本研究的目的是评估与安慰剂相比，在URTI开始时给予6天低剂量泼尼松龙疗程的成本效益。
建立了一个状态转换马尔可夫模型来进行成本效用分析，结果以质量调整生命年（QALY）衡量。资源使用和结果数据来自PREDNOS2试验。分析从英国国家医疗服务体系的角度进行，结果外推至成年期。使用敏感性分析评估模型参数和结构不确定性。
基础病例结果显示，与安慰剂相比，在URTI时给予低剂量泼尼松龙在1年时产生了更多的QALY且平均成本更低。从长期来看，与安慰剂相比，低剂量泼尼松龙可节省成本（176英镑）并提高有效性（0.01 QALY），因此仍是主要的治疗选择。这些发现对所有敏感性分析均具有稳健性。
与安慰剂相比，在类固醇敏感性肾病综合征儿童的URTI时给予6天低剂量泼尼松龙疗程有可能降低医疗成本并改善生活质量。
