Data Sheet 1_Characteristics and outcomes of patients with LAM receiving sirolimus in France based on real-life data.pdf

LAM is a rare multi-cystic lung disease for which treatment with sirolimus is indicated in cases of moderate or severe lung disease or declining lung function. The aim of this study was to describe patients treated with sirolimus for LAM and their outcomes. This retrospective observational study was based on data from the French national health insurance data system (SNDS). All adult women receiving sirolimus were identified in France between 2014 and 2021. In the absence of a specific LAM code in the system, an algorithm was developed to identify patients treated for possible LAM exclusion of other sirolimus indications (transplantation, graft-versus-host disease), or probable LAM (among possible LAM, patients hospitalized for pneumothorax, pleural drainage, pleurisy, ascites, chronic respiratory failure, lung transplantation, or angiomyolipoma). Over the entire study period, 638 patients were considered as treated with sirolimus for possible LAM, including 208 patients treated for “probable” LAM and 33 patients for TSC-LAM. Median [Q1; Q3] age at index date was 45.0 years [34.0; 58.5] for patients with probable LAM and 40.0 years [28.0; 56.0] for patients with TSC-LAM. Overall, the number of incident patients varied from 28 to 96 each year for possible LAM, from 11 to 33 each year for probable LAM and from 1 to 4 patients each year for TSC-LAM patients. In 2021, the incidence rate of patients treated with sirolimus for probable LAM in France was estimated at 0.9 per 1,000,000 French adult women and the prevalence rate at 6.3 per 1,000,000 French adult women. The 5-year survival after sirolimus initiation was 84% (95% CI: 76%; 90%) for probable LAM patients, and 77% (95% CI: 48%; 91%) for TSC-LAM patients. This study provides an updated epidemiological estimate of LAM patients treated with sirolimus in France between 2014 and 2021. Even though some of the results should be interpreted cautiously in the light of limitations related to the use of claims database, evolution of the disease and missing safety data, the information retrieved in this study is very valuable, as few studies provide real-world information on LAM populations.

淋巴管肌瘤病（Lymphangioleiomyomatosis，LAM）是一种罕见的多囊性肺部疾病，针对中重度肺部病变或肺功能进行性下降的患者，临床推荐采用西罗莫司（sirolimus）进行治疗。本研究旨在描述接受西罗莫司治疗的LAM患者队列及其临床转归。本研究为回顾性观察性研究，数据来源于法国国家健康保险数据系统（Système National des Données de Santé，SNDS）。研究纳入2014—2021年间法国境内所有接受西罗莫司治疗的成年女性患者。由于该系统未设置专门的LAM疾病编码，本研究开发了一套识别算法以筛选疑似LAM患者：首先排除存在其他西罗莫司治疗指征（如器官移植、移植物抗宿主病）的人群，再从疑似LAM队列中进一步甄别确诊可能性较高的患者——即因气胸、胸腔引流、胸膜炎、腹水、慢性呼吸衰竭、肺移植或血管平滑肌脂肪瘤住院的患者。整个研究周期内，共计638例患者被判定为因疑似LAM接受西罗莫司治疗，其中208例为“确诊可能性较高”的LAM患者，33例为结节性硬化症相关淋巴管肌瘤病（Tuberous Sclerosis Complex-LAM，TSC-LAM）患者。确诊可能性较高的LAM患者的索引日期中位年龄[四分位数间距Q1; Q3]为45.0岁[34.0; 58.5]，TSC-LAM患者为40.0岁[28.0; 56.0]。整体而言，疑似LAM患者的年新发例数波动于28~96例，确诊可能性较高的LAM患者年新发例数介于11~33例，TSC-LAM患者年新发例数为1~4例。2021年，法国境内因西罗莫司治疗的确诊可能性较高的LAM患者发病率估算为每100万成年女性0.9例，患病率为每100万成年女性6.3例。确诊可能性较高的LAM患者在启动西罗莫司治疗后的5年生存率为84%（95%置信区间：76%~90%），TSC-LAM患者为77%（95%置信区间：48%~91%）。本研究提供了2014—2021年间法国境内接受西罗莫司治疗的LAM患者的最新流行病学估算数据。尽管部分研究结果需结合本研究的局限性谨慎解读——包括使用医保索赔数据库带来的潜在偏倚、疾病病程演变特征及缺失的安全性数据，但本研究获取的信息仍具有极高临床价值：目前针对LAM人群的真实世界研究数据仍较为匮乏。