Cottin Vincent, Cases Antoine, Bourdin Virginie, Reynaud-Gaubert Martine, Hirschi Sandrine, Kerjouan Mallorie, Diesler Rémi, Chardès Brieux, Fiévez Stéphane, Assi Nada, Schmidt Aurélie, Denis Hélène, Wémeau-Stervinou Lidwine, Uzunhan Yurdagul
Department of Respiratory Medicine, National Reference Centre for Rare Pulmonary Diseases, member of ERN-LUNG, Louis Pradel Hospital, Hospices Civils de Lyon, Lyon, France.
UMR 754, INRAE, Claude Bernard University Lyon 1, Lyon, France.
Front Med (Lausanne). 2025 Jan 8;11:1494713. doi: 10.3389/fmed.2024.1494713. eCollection 2024.
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（LAM）是一种罕见的多囊性肺部疾病，对于中度或重度肺部疾病或肺功能下降的病例，建议使用西罗莫司进行治疗。本研究的目的是描述接受西罗莫司治疗的LAM患者及其治疗结果。这项回顾性观察研究基于法国国家医疗保险数据系统（SNDS）的数据。在2014年至2021年期间，在法国确定了所有接受西罗莫司治疗的成年女性。由于系统中没有特定的LAM编码，因此开发了一种算法，以识别接受治疗的可能患有LAM的患者，排除其他西罗莫司适应症（移植、移植物抗宿主病）或可能患有LAM的患者（在可能患有LAM的患者中，因气胸、胸腔引流、胸膜炎、腹水、慢性呼吸衰竭、肺移植或肾血管平滑肌脂肪瘤住院的患者）。在整个研究期间，638例患者被视为接受了西罗莫司治疗，可能患有LAM，其中208例患者接受了“可能”LAM的治疗，33例患者接受了结节性硬化症相关LAM（TSC-LAM）的治疗。可能患有LAM的患者在索引日期的中位年龄[四分位间距1；四分位间距3]为45.0岁[34.0；58.5]，TSC-LAM患者为40.0岁[28.0；56.0]。总体而言，可能患有LAM的患者每年的新发病例数从28例到96例不等，可能患有LAM的患者每年从11例到33例不等，TSC-LAM患者每年从1例到4例不等。2021年，法国接受西罗莫司治疗的可能患有LAM的患者的发病率估计为每100万法国成年女性中有0.9例，患病率为每100万法国成年女性中有6.3例。开始使用西罗莫司后的5年生存率，可能患有LAM的患者为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年至2021年期间法国接受西罗莫司治疗的LAM患者的最新流行病学估计。尽管鉴于与使用索赔数据库、疾病演变和缺失安全数据相关的局限性，部分结果应谨慎解释，但本研究中检索到的信息非常有价值，因为很少有研究提供关于LAM人群的真实世界信息。
