Goldschmidt Deborah, Bensink Mark E, Zhou Zheng-Yi, Shi Sherry, Lin Yilu, Shi Lizheng
Analysis Group, Boston, Massachusetts, United States of America.
Travere Therapeutics, Inc., San Diego, California, United States of America.
PLoS One. 2024 Dec 13;19(12):e0315302. doi: 10.1371/journal.pone.0315302. eCollection 2024.
Focal segmental glomerulosclerosis (FSGS) is a rare glomerular disease that can lead to reduced kidney function and kidney failure (KF). The objective of this study was to describe the epidemiology, characteristics, clinical outcomes, healthcare resource utilization, and costs associated with focal segmental glomerulosclerosis (FSGS) in United States (US) veterans.
This retrospective cohort study included patients in the National Veterans Affairs Health Care Network with ≥2 FSGS-associated diagnostic codes that were 30-180 days apart (October 1999-February 2021). Annual FSGS incidence and prevalence per 1,000,000 US veterans were calculated. Patient and disease characteristics as of the index date (date of first FSGS diagnosis) and baseline (6-months pre-index) comorbidities were described. Kaplan-Meier analyses were used to assess overall survival and time from index to KF or death, dialysis, and kidney transplant. Post-index medication use, HRU, and direct healthcare costs were summarized.
The study included 2,515 veterans with FSGS who were followed for an average of 8.9 years. The mean age was 57.5 years, most patients were male (94.6%), and the most common comorbidity was hypertension (87.0%). The mean annual incidence and prevalence of FSGS during 2000-2020 were 19.6 and 164.7 per million veterans, respectively. Approximately half (51.5%) died during follow-up (median time: 11.6 years) and 76.9% had kidney failure (4.1 years). Overall, 43.3% underwent dialysis and 5.8% had a kidney transplant. During follow-up, statins and calcium channel blockers were commonly used (81.9% and 75.1%). During the first year post-index, 40% had an inpatient admission and 33% visited the emergency room; mean total healthcare cost per patient in the analysis was $36,543.
Among US veterans, FSGS is associated with considerable clinical and economic burdens. Better treatments for FSGS are needed to slow kidney disease progression, improve patient outcomes, and reduce the burden.
局灶节段性肾小球硬化（FSGS）是一种罕见的肾小球疾病，可导致肾功能下降和肾衰竭（KF）。本研究的目的是描述美国退伍军人中局灶节段性肾小球硬化（FSGS）的流行病学、特征、临床结局、医疗资源利用情况以及相关费用。
这项回顾性队列研究纳入了美国退伍军人事务医疗保健网络中诊断代码与FSGS相关且间隔30 - 180天的患者（1999年10月至2021年2月）。计算每百万美国退伍军人中FSGS的年发病率和患病率。描述了索引日期（首次FSGS诊断日期）时的患者和疾病特征以及基线（索引前6个月）合并症情况。采用Kaplan-Meier分析评估总生存率以及从索引日期到发生KF或死亡、透析和肾移植的时间。总结了索引后药物使用情况、医疗资源利用情况和直接医疗费用。
该研究纳入了2515名患有FSGS的退伍军人，平均随访8.9年。平均年龄为57.5岁，大多数患者为男性（94.6%），最常见的合并症是高血压（87.0%）。2000 - 2020年期间FSGS的年平均发病率和患病率分别为每百万退伍军人19.6例和164.7例。随访期间约一半（51.5%）患者死亡（中位时间：11.6年），76.9%患者出现肾衰竭（4.1年）。总体而言，43.3%的患者接受了透析，5.8%的患者进行了肾移植。随访期间，他汀类药物和钙通道阻滞剂使用较为普遍（81.9%和75.1%）。索引后第一年，40%的患者有住院记录，33%的患者去过急诊室；分析中每位患者的平均总医疗费用为36,543美元。
在美国退伍军人中，FSGS与相当大的临床和经济负担相关。需要更好的FSGS治疗方法来减缓肾脏疾病进展、改善患者结局并减轻负担。
