Data_Sheet_1_Real-World Healthcare Resource Utilization and Cost Burden Assessment for Adults With Generalized Myasthenia Gravis in the United States.docx

IntroductionLimited evidence exists for healthcare resource utilization (HCRU) and costs associated with generalized myasthenia gravis (gMG), a rare autoimmune disorder, for adults in the United States.MethodsAdults with ≥1 diagnostic claim for MG between 2014 and 2019 were identified using Symphony Health's Integrated Dataverse®. Using a novel algorithm, HCRU and costs over 12 months following index dates were evaluated for patients with gMG including those with exacerbation events. For patients who experienced crisis events, HCRU and costs were analyzed during the 36 months preceding, during, and 12 months following the events.ResultsMean HCRU and costs were higher for newly diagnosed patients compared with previously diagnosed patients (hospitalizations: 0.46 vs. 0.34; all-cause costs: $26,419.20 vs. $24,941.47; direct costs for gMG treatments: $9,890.37 vs. $9,186.47) and further increased for patients with exacerbation events (hospitalizations: 0.72; all-cause costs: $43,734.15; direct costs for gMG treatments: $21,550.02). For patients who experienced crisis events, HCRU and costs markedly increased during the 12 months immediately before the crisis event (hospitalizations: 1.35; all-cause costs: $49,236.68) compared with the 2 preceding years and increased further during the 12 months following the crisis index date (hospitalizations: 2.78; all-cause costs: $173,956.99). Cost increases were, in large part, attributed to treatments received.DiscussionNew diagnosis, exacerbation, and crisis events were drivers of HCRU and cost for patients with gMG. Particularly, high costs of gMG-specific medications associated with intervention for exacerbation and crisis events contributed to increased all-cause costs.

关于美国成年人群普遍重症肌无力（gMG）这一罕见自身免疫性疾病相关的医疗资源利用（HCRU）及其成本，现有证据有限。研究方法：通过 Symphony Health 的综合数据平台®，识别出2014至2019年间具有≥1次MG诊断索赔的成年人。采用一种新颖的算法，评估了gMG患者（包括那些出现病情恶化事件的患者）在索引日期后的12个月内的HCRU和成本。对于经历危机事件的患者，分析了事件前36个月、事件期间及事件后12个月的HCRU和成本。研究结果：新诊断患者的平均HCRU和成本高于既往诊断患者（住院率：0.46 vs. 0.34；全因成本：26,419.20美元 vs. 24,941.47美元；gMG治疗直接成本：9,890.37美元 vs. 9,186.47美元），且在出现病情恶化事件的患者中进一步增加（住院率：0.72；全因成本：43,734.15美元；gMG治疗直接成本：21,550.02美元）。对于经历危机事件的患者，与危机事件前两年相比，危机事件前12个月的HCRU和成本显著增加（住院率：1.35；全因成本：49,236.68美元），且在危机索引日期后的12个月内进一步增加（住院率：2.78；全因成本：173,956.99美元）。成本增加在很大程度上归因于接受的治疗。讨论：新诊断、病情恶化及危机事件是gMG患者HCRU和成本增加的驱动因素。特别是，与干预病情恶化和危机事件相关的gMG特定药物的高成本，导致了全因成本的显著上升。