Sánchez Fernández Iván, Amengual-Gual Marta, Barcia Aguilar Cristina, Romeu Amanda, Sheikh Tahir, Torres Alcy, Chao Jessica, Jonas Rinat, Gaínza-Lein Marina, Harini Chellamani, Douglass Laurie
Division of Pediatric Neurology, Department of Pediatrics, Boston Medical Center, Boston University School of Medicine, Boston, Massachusetts, USA.
Pediatric Neurology Unit, Department of Pediatrics, Hospital Universitari Son Llàtzer, Universitat de les Illes Balears, Palma, Spain.
Epilepsia. 2023 Mar;64(3):630-640. doi: 10.1111/epi.17498. Epub 2023 Jan 31.
To describe the temporal trends in the cost and use of adrenocorticotropic hormone (ACTH), oral prednisolone, and vigabatrin, the first-line treatments for infantile epileptic spasms syndrome (IESS).
Retrospective observational study using the MarketScan Commercial database from 2006 to 2020. We identified patients with IESS diagnosed between birth and 18 months of age who received at least one of the first-line treatments within 60 days of diagnosis. Costs were adjusted for inflation using the Gross Domestic Product Implicit Price Deflator.
A total of 1131 patients received at least one first-line treatment (median [p -p ] age: 6.3 [4.5-8.3] months, 55% male), of whom 592 patients received ACTH, 363 patients received oral prednisolone, and 355 patients received vigabatrin. After adjusting for inflation, the median average wholesale price of a 14-day course of treatment increased for ACTH from $3718 in 2006 to $100 457 in 2020, ~2700% (by a factor of 27), whereas it decreased for oral prednisolone from $169 in 2006 to $89 in 2020, ~50% (by a factor of 0.5), and increased for vigabatrin from $1206 in 2009 (first year with data on vigabatrin used for IESS) to $4102 in 2020, ~340% (by a factor of 3.4). During the first 60 days after diagnosis, inpatient admission days and costs where higher for ACTH than for oral prednisolone and vigabatrin-5.0 (3.0-8.3) days vs 2.0 (0.0-5.0) days vs 2.0 (0.0-6.0) days, p < .0001; and $32 828 ($14 711-$67 216) vs $16 227 ($0-$35 829) vs $17 844 ($0-$47 642), p < .0001. ACTH use decreased from representing 78% of first-line treatments in 2006 to 18% in 2020 (p < .0001). Sensitivity analyses confirmed the robustness of the results.
The gap between the cost of ACTH and the cost of oral prednisolone or vigabatrin has widened markedly from 2006 to 2020, whereas the relative proportion of ACTH use has decreased.
描述婴儿痉挛症综合征（IESS）一线治疗药物促肾上腺皮质激素（ACTH）、口服泼尼松龙和氨己烯酸的成本及使用情况的时间趋势。
使用2006年至2020年的MarketScan商业数据库进行回顾性观察研究。我们确定了出生至18个月大之间被诊断为IESS且在诊断后60天内接受至少一种一线治疗的患者。使用国内生产总值隐含价格平减指数对成本进行通胀调整。
共有1131名患者接受了至少一种一线治疗（中位年龄[四分位间距]：6.3[4.5 - 8.3]个月，55%为男性），其中592名患者接受ACTH治疗，363名患者接受口服泼尼松龙治疗，355名患者接受氨己烯酸治疗。经通胀调整后，ACTH一个14天疗程的平均批发价中位数从2006年的3718美元增至2020年的100457美元，增长约2700%（增长27倍）；而口服泼尼松龙则从2006年的169美元降至2020年的89美元，下降约50%（下降0.5倍）；氨己烯酸从2009年（有IESS使用氨己烯酸数据的第一年）的1206美元增至2020年的4102美元，增长约340%（增长3.4倍）。在诊断后的前60天内，ACTH的住院天数和费用高于口服泼尼松龙和氨己烯酸——分别为5.0（3.0 - 8.3）天、2.0（0.0 - 5.0）天、2.0（0.0 - 6.0）天，p < 0.0001；费用分别为32828美元（14711 - 67216美元）、16227美元（0 - 35829美元）、17844美元（0 - 47642美元），p < 0.0001。ACTH的使用从2006年占一线治疗的78%降至2020年的18%（p < 0.0001）。敏感性分析证实了结果的稳健性。
从2006年到2020年，ACTH与口服泼尼松龙或氨己烯酸的成本差距显著扩大，而ACTH的使用相对比例下降。
