Aggarwal Rohit, Cai Qian, Labson Daniel, Crivera Concetta, Zazzetti Federico
Division of Rheumatology and Clinical Immunology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
Global Market Access RWE, Johnson & Johnson, Titusville, NJ, USA.
Clin Rheumatol. 2025 Sep 10. doi: 10.1007/s10067-025-07651-1.
INTRODUCTION/OBJECTIVE: Oral glucocorticoids (OGC) are conventionally used as first-line treatment for dermatomyositis (DM) and polymyositis (PM). This study evaluated clinical and economic outcomes associated with long-term (LT) OGC use in DM/PM.
Adults with ≥ 2 medical claims of DM/PM 30‒365 days apart from January 1, 2016, to December 31, 2022, and ≥ 1 diagnosis code of a physician specialty of interest were selected from the MarketScan Commercial and Medicare Supplemental databases. Patients should have ≥ 1 OGC pharmacy claim (i.e., date of first claim defined as index date) on or after the diagnosis date, ≥ 12 months pre- and post-index continuous enrollment, and no diagnosis of inclusion body myositis during the study. The LT group included patients with continuous OGC use for ≥ 3 consecutive months within the 12-month post-index period, whereas short-term (ST) users included those with < 3 consecutive months of OGC use.
Two thousand two-hundred eighty patients were included (LT, 1313 [57.6%]; ST, 967 [42.4%]). Compared with ST, LT OGC patients had significantly higher incidences of OGC-related complications, such as heart failure (adjusted odds ratio [aOR], 1.8; 95% CI, 1.3‒2.7) and osteoporosis (aOR, 1.9; 95% CI, 1.4‒2.6), after covariate adjustment. LT users had increased odds for both all-cause (aOR, 1.7; 95% CI, 1.3‒2.2) and disease-related inpatient admission (aOR, 3.8; 95% CI, 2.3‒6.2) versus ST users. Higher adjusted average annual all-cause (by US $30,555; p < 0.01) and disease-related costs (by US $21,311; p < 0.01) were incurred by LT versus ST users.
DM/PM patients with LT OGC use had higher rates of associated medical conditions and higher healthcare resource utilization and costs than ST users. Key points • Long-term use of oral glucocorticoids (OGC) in patients with dermatomyositis (DM) and polymyositis (PM) is associated with increased risk of complications, and there is limited real-world evidence on the association of long-term use with clinical and economic outcomes. • This real-world claims-based study demonstrated that long-term OGC use in patients with DM/PM leads to higher rates of complications, advanced treatment use and healthcare resource utilization, and higher costs compared with short-term OGC use. • These findings highlight the limitations of OGC as a therapeutic tool and further support an unmet medical need for new treatment options among patients with DM/PM. • Disease management with novel treatments that have an advantageous safety profile and can be utilized any time throughout the disease course may provide safer, more efficacious alternatives to OGC.
引言/目的：口服糖皮质激素（OGC）传统上用作皮肌炎（DM）和多发性肌炎（PM）的一线治疗药物。本研究评估了DM/PM患者长期（LT）使用OGC的临床和经济结局。
从MarketScan商业保险和医疗保险补充数据库中选取2016年1月1日至2022年12月31日期间间隔30至365天有≥2次DM/PM医疗理赔记录且有≥1个感兴趣的医生专业诊断代码的成年人。患者在诊断日期或之后应有≥1次OGC药房理赔记录（即首次理赔日期定义为索引日期），索引前后连续参保≥12个月，且研究期间无包涵体肌炎诊断。LT组包括索引后12个月内连续使用OGC≥3个月的患者，而短期（ST）使用者包括OGC使用时间<3个月的患者。
共纳入2280例患者（LT组1313例[57.6%]；ST组967例[42.4%]）。经协变量调整后，与ST组相比，LT组OGC相关并发症的发生率显著更高，如心力衰竭（调整优势比[aOR]为1.8；95%置信区间[CI]为1.3至2.7）和骨质疏松症（aOR为1.9；95%CI为1.4至2.6）。与ST组使用者相比，LT组使用者全因住院（aOR为1.7；95%CI为1.3至2.2）和疾病相关住院的几率均增加（aOR为3.8；95%CI为2.3至6.2）。LT组使用者的调整后平均年度全因费用（高出30555美元；p<0.01）和疾病相关费用（高出21311美元；p<0.01）均高于ST组使用者。
与短期使用者相比，长期使用OGC的DM/PM患者相关疾病发生率更高，医疗资源利用和成本更高。要点 • 皮肌炎（DM）和多发性肌炎（PM）患者长期使用口服糖皮质激素（OGC）与并发症风险增加相关，关于长期使用与临床和经济结局之间关联的真实世界证据有限。 • 这项基于真实世界理赔数据的研究表明，与短期使用OGC相比，DM/PM患者长期使用OGC会导致更高的并发症发生率、更多的进阶治疗使用和医疗资源利用以及更高的成本。 • 这些发现凸显了OGC作为治疗工具的局限性，并进一步支持DM/PM患者对新治疗方案的未满足医疗需求。 • 使用安全性良好且可在疾病病程中随时使用的新型治疗方法进行疾病管理，可能为OGC提供更安全、更有效的替代方案。
