Min Jea Young, Knupp Kelly G, Patel Anup D, Shellhaas Reneé A, Zhang Manyao, Grinspan Zachary M
Department of Population Health Sciences, Weill Cornell Medical College, New York, NY, USA.
Department of Pediatrics and Neurology, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Epilepsy Res. 2021 Oct;176:106733. doi: 10.1016/j.eplepsyres.2021.106733. Epub 2021 Jul 24.
There are three recommended first-line treatments for infantile spasms, adrenocorticotropic hormone (ACTH), oral corticosteroids, and vigabatrin, though non-standard treatments such as topiramate are sometimes selected. Is it uncertain how treatment selection influences health services outcomes.
We conducted a retrospective cohort study of Medicaid beneficiaries newly diagnosed with infantile spasms from 2009-2010. We included infants with a new diagnosis of infantile spasms between age 2-9 months who filled ACTH (reference), prednisolone, vigabatrin, or topiramate prescriptions. Multivariable Cox proportional hazards regression compared time to first emergency department (ED) visit or hospitalization across treatment groups during 2 years of follow-up. Monthly costs for each treatment were examined in 6-month intervals and compared in a multivariable generalized linear model.
Among 256 children with infantile spasms, 116 received ACTH, 62 prednisolone, 15 vigabatrin, and 63 topiramate. The rate of ED visit or hospitalization per person-year did not differ significantly for prednisolone (0.9 [95 % CI 0.7-1.2]; adjusted hazard ratio [aHR] 0.84, 95 % CI 0.57-1.24), vigabatrin (0.8 [95 % CI 0.4-1.5]; aHR 0.91, 95% CI 0.45-1.84), or topiramate (1.7 [95 % CI 1.3-2.3]; aHR 1.15, 95 % CI 0.80-1.65), when compared to ACTH (1.1 [95 % CI 0.9-1.3]). The median payment for ACTH was $96,406 (interquartile range 70,742-138,476) during the first 6 months. The adjusted mean total payment in the first 6 months was 73% lower for prednisolone (95% CI -82, -61), 69% lower for vigabatrin (95% CI -84, -40), and 73% lower for topiramate (95% CI -82, -59). However, in subsequent 6-month intervals, costs associated with ACTH were not significantly different compared to other treatments.
Compared to other treatments for infantile spasms, use of ACTH was associated with greater cost in the first 6 months of treatment, but not with reduced ED visits or hospitalizations. The cost effectiveness of ACTH depends on its relative clinical efficacy, and merits additional research.
婴儿痉挛症有三种推荐的一线治疗方法，即促肾上腺皮质激素（ACTH）、口服糖皮质激素和氨己烯酸，不过有时也会选择托吡酯等非标准治疗方法。治疗选择如何影响卫生服务结果尚不确定。
我们对2009年至2010年新诊断为婴儿痉挛症的医疗补助受益人群进行了一项回顾性队列研究。我们纳入了年龄在2至9个月之间新诊断为婴儿痉挛症且开具了ACTH（对照）、泼尼松龙、氨己烯酸或托吡酯处方的婴儿。多变量Cox比例风险回归比较了随访2年期间各治疗组首次急诊就诊或住院的时间。每6个月检查一次每种治疗的月度费用，并在多变量广义线性模型中进行比较。
在256例婴儿痉挛症患儿中，116例接受了ACTH治疗，62例接受了泼尼松龙治疗，15例接受了氨己烯酸治疗，63例接受了托吡酯治疗。泼尼松龙组（0.9[95%CI 0.7 - 1.2]；调整后风险比[aHR]0.84，95%CI 0.57 - 1.24）、氨己烯酸组（0.8[95%CI 0.4 - 1.5]；aHR 0.91，95%CI 0.45 - 1.84）或托吡酯组（1.7[95%CI 1.3 - 2.3]；aHR 1.15，95%CI 0.80 - 1.65）的每人每年急诊就诊或住院率与ACTH组（1.1[95%CI 0.9 - 1.3]）相比，差异均无统计学意义。ACTH在最初6个月的中位费用为96,406美元（四分位间距70,742 - 138,476美元）。泼尼松龙在最初6个月的调整后平均总费用低73%（95%CI - 82，- 61），氨己烯酸低69%（95%CI - 84，- 40），托吡酯低73%（95%CI - 82，- 59）。然而，在随后的6个月期间，与ACTH相关的费用与其他治疗相比无显著差异。
与婴儿痉挛症的其他治疗方法相比，ACTH治疗在最初6个月的费用更高，但急诊就诊或住院次数并未减少。ACTH的成本效益取决于其相对临床疗效，值得进一步研究。
