摘要
Lymphangioleiomyomatosis (LAM) is a rare, low-grade malignant tumor primarily affecting female patient. For LAM patients in the end stage, lung transplantation is the only treatment option. The pharmacotherapy is an important way to alleviating loss of lung function. Although several types of pharmacotherapies for patients with LAM have been reported in previous studies, the optimal medication remain unclear. Therefore, we conducted a systematic review and network meta-analysis to evaluate and compare the efficacy and safety of the pharmacotherapy for patients with LAM.
A systematic review of clinical trials was conducted following PRISMA guidelines. Relevant studies were searched using PubMed, Embase and Web of Science from the inception to April 2025. The inclusion criteria for articles were: (1) clinical trials involving any pharmacotherapy for LAM, and (2) a clear description of lung function. Firstly, literature review was performed to present updated insights into the pharmacotherapy of LAM. Subsequently, a random effects network meta-analysis using a frequentist approach was performed at forced expiratory volume in the first second (FEV1), forward voice channel (FVC) and diffusion lung capacity for CO (DLCO). All the control groups were treated with Placebo.
15 studies with 474 patients were included in this meta-analysis. The medications including sirolimus, everolimus, letrozole, nintedanib. Among them, sirolimus was the most common medication (n=7) in the meta-analysis.
3 RCTs involving 129 patients and 3 medications (Sirolimus, Doxycycline and Letrozole) were used to perform a network Meta-analysis. In terms of improving patients' FEV1, Doxycycline was the most effective (average grade of 1.0, SUCRA score of 100), compared with Placebo (MD = 69.80, 95% CI [65.40,74.20]). Sirolimus showed the best improvement in FVC (average grade 1.0, SUCRA score 100).
This network meta-analysis indicates that sirolimus and doxycycline can improve FEV1 and FVC in patients with lymphangioleiomyomatosis. Further randomized controlled trials on drug treatments for lymphangioleiomyomatosis to develop more effective therapeutic regimens.
