Broadstreet Health Economics and Outcomes Research, 201-343 Railway Street, Vancouver, BC, V6A 1A4, Canada.
Sarepta Therapeutics, Inc, 215 First Street, Cambridge, MA, 02142, USA.
Adv Ther. 2024 Jun;41(6):2519-2530. doi: 10.1007/s12325-024-02865-2. Epub 2024 May 2.
Duchenne muscular dystrophy (DMD) is a rare, severe progressive neuromuscular disease. Health insurance claims allow characterization of population-level real-world outcomes, based on observed healthcare resource use. An analysis of data specific to those with Medicaid insurance is presently unavailable. The objective was to describe the real-world clinical course of DMD based on claims data from Medicaid-insured individuals in the USA.
Individuals with DMD were identified from the MarketScan Multi-State Medicaid datasets (2013-2018). Diagnosis and procedure codes from healthcare claims were used to characterize the occurrence of DMD-relevant clinical observations; categories were scoliosis, cardiovascular-related, respiratory and severe respiratory-related, and neurologic/neuropsychiatric. Age-restricted analyses were conducted to focus on the ages at which DMD-relevant clinical observations were more likely to be captured, and to better understand the impact of both age and follow-up time.
Of 2007 patients with DMD identified, median (interquartile range) age at index was 14 (9-20) years, and median follow-up was 3.1 (1.6-4.7) years. Neurologic and neuropsychiatric observations were most frequently identified, among 49.3% of the cohort; followed by cardiovascular (48.5%), respiratory (38.1%), scoliosis (36.3%), and severe respiratory (25.0%). Prevalence estimates for each category were higher when analyzed within age-restricted subgroups; and increased as follow-up time increased.
This study is the first to use diagnosis and procedure codes from real-world Medicaid claims to document the clinical course in DMD. Findings were consistent with previously published estimates from commercially insured populations and clinical registries; and contribute to the expanding body of real-world evidence around clinical progression of patients with DMD.
杜氏肌营养不良症（DMD）是一种罕见的严重进行性神经肌肉疾病。医疗保险索赔允许根据观察到的医疗资源使用情况来描述人群水平的真实结果。目前尚无法从 Medicaid 保险中获得特定人群的数据进行分析。本研究的目的是根据美国 Medicaid 保险个人的索赔数据，描述 DMD 的真实临床过程。
从 MarketScan 多州 Medicaid 数据集（2013-2018 年）中确定 DMD 患者。使用医疗保健索赔中的诊断和程序代码来描述与 DMD 相关的临床观察结果的发生情况；类别包括脊柱侧凸、心血管相关、呼吸和严重呼吸相关以及神经/神经精神。进行年龄限制分析是为了重点关注更有可能捕捉到与 DMD 相关的临床观察的年龄，并更好地理解年龄和随访时间的双重影响。
在所确定的 2007 名 DMD 患者中，索引年龄的中位数（四分位距）为 14 岁（9-20 岁），中位数随访时间为 3.1 年（1.6-4.7 年）。49.3%的队列中最常发现神经和神经精神观察结果；其次是心血管（48.5%）、呼吸（38.1%）、脊柱侧凸（36.3%）和严重呼吸（25.0%）。在年龄限制亚组内进行分析时，每个类别的患病率估计值更高；并且随着随访时间的增加而增加。
这项研究是首次使用来自真实世界 Medicaid 索赔的诊断和程序代码来记录 DMD 的临床过程。研究结果与商业保险人群和临床登记处以前发表的估计结果一致；并为围绕 DMD 患者临床进展的真实世界证据不断扩大做出了贡献。
