Sharawat Indar Kumar, Panda Prateek Kumar, Ramachandran Aparna, Bhadoria Ajeet Singh
Department of Pediatrics, All India Institute of Medical Sciences, Rishikesh, Uttarakhand, India.
Department of Neurology, IQRAA International Hospital and Research Centre, Kozhikode, Kerala, India.
J Neurosci Rural Pract. 2023 Jan-Mar;14(1):103-110. doi: 10.25259/JNRP-2022-6-31. Epub 2022 Dec 15.
This study aims to compare the cost-effectiveness of oral prednisolone and adrenocorticotropic hormone injection in West syndrome patients, the two most common hormonal therapies used for this condition.
In this prospective and observational study, we documented sociodemographic, epilepsy, and development-related variables at baseline and up to 6 months after starting hormonal therapy, in all consecutive eligible patients of WS between August 2019 and June 2021, apart from the direct medical and non-medical costs and indirect health-care costs. We selected cost per quality-adjusted life-year (QALY) gained, per one patient with spasm freedom, one positive responder (>50% reduction in spasms), one relapse-free patient, and one patient with development gain. We determined whether incremental cost-effectiveness ratio for these parameters crossed the threshold value in base-case analysis and alternate scenario analysis.
Out of 52 patients screened, 38 and 13 patients enrolled in ACTH and prednisolone group. On D28, 76% and 71% achieved spasm cessation ( = 0.78) and the total cost of treatment was INR 19783 and 8956 ( = 0.01), in ACTH and prednisolone group respectively. For all pre-specified parameters, the cost/effectiveness ratios including cost/QALY gain were higher in ACTH group and the corresponding ICER values for all these parameters crossed the threshold cost value of INR 148,777 in base-case analysis and also in alternative scenario analysis.
Treatment with oral prednisolone is more cost-effective as compared to ACTH injection for children with WS.
本研究旨在比较口服泼尼松龙和促肾上腺皮质激素注射对韦斯特综合征患者的成本效益,这是用于该病症的两种最常见的激素疗法。
在这项前瞻性观察研究中,我们记录了2019年8月至2021年6月期间所有连续符合条件的韦斯特综合征患者在基线以及开始激素治疗后长达6个月的社会人口统计学、癫痫和发育相关变量,此外还记录了直接医疗和非医疗成本以及间接医疗保健成本。我们选择了每获得一个质量调整生命年(QALY)的成本、每一名痉挛停止的患者、每一名阳性反应者(痉挛减少>50%)、每一名无复发患者以及每一名有发育进步患者的成本。我们在基础病例分析和替代情景分析中确定这些参数的增量成本效益比是否超过阈值。
在52名筛查患者中,38名和13名患者分别纳入促肾上腺皮质激素组和泼尼松龙组。在第28天,促肾上腺皮质激素组和泼尼松龙组分别有76%和71%的患者痉挛停止(P = 0.78),治疗总成本分别为19783印度卢比和8956印度卢比(P = 0.01)。对于所有预先指定的参数,促肾上腺皮质激素组的成本/效益比(包括成本/QALY增益)更高,并且在基础病例分析和替代情景分析中,所有这些参数的相应增量成本效益比均超过了148777印度卢比的阈值成本值。
对于韦斯特综合征儿童,口服泼尼松龙治疗比促肾上腺皮质激素注射更具成本效益。