PDF(Pubmed)
Abstract：
Aim: Nusinersen, administered by intrathecal injection at a dose of 12 mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA). Evidence on real-world healthcare resource use (HRU) and costs among patients taking nusinersen remains limited. This study aimed to evaluate real-world HRU and costs associated with nusinersen use through US claims databases. Patients & methods: Using the Merative™ MarketScan® Research Databases, patients with SMA receiving nusinersen were identified from commercial (January 2017 to June 2020) and Medicaid claims (January 2017 to December 2019). Those likely to have complete information on the date of nusinersen initiation and continuous enrollment 12 months pre- and post-index (first record of nusinersen treatment) were retained. Number and costs (US$ 2020) of inpatient admissions and emergency department (ED) visits, unrelated to nusinersen administration, were evaluated for 12 months pre- and post-nusinersen initiation and stratified by age: pediatric (<18 years) and adult (≥18 years). Results: Overall, 103 individuals treated with nusinersen were retained: 59 were pediatric (mean age [range]: 9 [1-17] years), and 44 were adults (30 [18-63] years). Inpatient admissions decreased by 41% for pediatrics and 67% for adults in the 12 months post-treatment versus the 12 months pre-treatment. Average inpatient admission costs per patient for the pediatric cohort decreased by 63% ($22,903 vs $8466) and by 79% ($13,997 vs $2899) for the adult cohort when comparing the 12 months pre-index with the 12 months post-index period. Total ED visits and ED visit costs decreased by 8% and 35%, respectively, for the overall cohort over the 12-month period pre- and post-index. Conclusion: Using US claims databases, nusinersen treatment in pediatric and adult patients was associated with reductions in HRU and costs over a 12-month period post-treatment initiation relative to the pre-treatment period.
摘要：
目标:Nusinersen,鞘内注射给药剂量为12毫克，适用于所有年龄段的脊髓性肌萎缩症（SMA）的治疗。有关服用nusinersen的患者的实际医疗保健资源使用（HRU）和成本的证据仍然有限。这项研究旨在通过美国索赔数据库评估与nusinersen使用相关的实际HRU和成本。患者和方法：使用Merative™MarketScan®研究数据库，接受nusinersen治疗的SMA患者来自商业(2017年1月至2020年6月)和医疗补助(2017年1月至2019年12月).保留了那些可能具有nusinersen开始日期和连续入组12个月前和后指数（nusinersen治疗的第一个记录）的完整信息。住院和急诊科（ED）就诊的次数和费用（2020美元），与Nusinersen管理无关，对nusinersen开始治疗前后12个月进行评估，并按年龄分层：儿科（<18岁）和成人（≥18岁）。结果：总体而言，保留了103名接受nusinersen治疗的患者：59名儿童（平均年龄[范围]：9[1-17]岁），44人是成年人（30[18-63]岁）。与治疗前的12个月相比，儿科住院率下降了41％，成人住院率下降了67％。当比较12个月前指数与12个月后指数期时，儿科队列的平均住院费用降低了63％（22,903美元对8466美元），成人队列的平均住院费用降低了79％（13,997美元对2899美元）。ED总就诊次数和ED就诊费用分别下降了8%和35%，分别,对于指数前后12个月期间的整体队列。结论：使用美国索赔数据库，儿童和成人患者的nusinersen治疗与治疗开始后12个月期间的HRU和费用相对于治疗前的减少相关.
