散发性和伴发结节性硬化症复合型 LAM 的临床、断层扫描和功能比较:一项回顾性队列研究

Martina Rodrigues de Oliveira, Mark Wanderley, Carolina Salim, Gonçalves Freitas, R. Kairalla, R. Chate, A. F. Amaral, F. E. Arimura, L. P. Samorano, Elieser Hitoshi Watanabe, C. R. Carvalho, B. Baldi
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引用次数: 0

摘要

淋巴管瘤病(LAM)是一种罕见疾病,可以偶发(S-LAM),也可以与结节性硬化综合征伴发(TSC-LAM)。人们对 LAM 的自然病史还不完全了解,包括这两种类型的临床病程是否存在差异。本研究旨在比较S-LAM和TSC-LAM的临床、功能和断层扫描特征,并评估肺功能的年变化率。这项回顾性队列研究纳入了1994年至2019年间随访的LAM患者。这项回顾性队列研究纳入了 1994 年至 2019 年随访的 LAM 患者,评估了临床、功能和影像学变量,并自动量化了肺囊肿。在纳入的107名患者中,77人患有S-LAM,30人患有TSC-LAM。虽然TSC-LAM患者的肾血管肌脂肪瘤、神经系统和皮肤病的发病率较高,但肺功能测试结果却相似。S-LAM患者的FEV1下降率更高,囊肿的范围也更大。气胸、六分钟步行测试不饱和以及肺囊肿程度较高是功能受损的预测因素。TSC-LAM患者的生命力和情绪健康受到的影响更大。我们的研究为 LAM 患者提供了广泛的临床、功能和断层扫描特征,为现有证据增添了有价值的信息,有助于更好地了解该疾病的两种形式。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical, tomographic, and functional comparison of sporadic and associated tuberous sclerosis complex forms of LAM: a retrospective cohort study
Lymphangioleiomyomatosis (LAM) is a rare disease that can occur sporadically (S-LAM) or associated with the tuberous sclerosis complex (TSC-LAM). The natural history of LAM is not completely understood, including whether there is a difference between the clinical courses of the two forms. This study aimed to compare the clinical, functional, and tomographic features between S-LAM and TSC-LAM, and evaluate the annual rates of change in lung function.This retrospective cohort study included patients with LAM followed up between 1994 and 2019. Clinical, functional and imaging variables were evaluated, and the lung cysts were automatically quantified. Quality of life and predictors of lung function impairment were accessed, and the annual rate of lung function decline was compared between S-LAM and TSC-LAM.Of the 107 patients included, 77 had S-LAM and 30 had TSC-LAM. Although patients with TSC-LAM had a higher prevalence of renal angiomyolipomas and neurological and dermatological manifestations, pulmonary function tests were similar. Patients with S-LAM had a greater rate of FEV1decline and a higher extent of cysts. Pneumothorax, desaturation in the six-minute walking test and a higher extent of lung cysts were predictors of functional impairment. A greater impact on vitality and emotional health was observed in the TSC-LAM.Greater functional decline and a higher cystic extension were found in patients with S-LAM. Our study provides a broad clinical, functional, and tomographic characterisation of patients with LAM, adding valuable information to the existing evidence to better understand the two forms of the disease.
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