Key result
Current treatment approaches for pulmonary lymphangioleiomyomatosis, including mTOR inhibitors, target established disease, but effective prevention remains impossible due to unknown etiology.
Why the study?
The study was conducted to collect and systematize data on treatment approaches to pulmonary lymphangioleiomyomatosis based on insights into disease pathogenesis.
This review highlights the evolution of LAM treatments from radical surgical and hormonal approaches to targeted mTOR inhibitors, emphasizing the need for further research into its etiology for effective prevention.
May guide LAM management decisions; leaves open prospective validation of reviewed approaches.
The aim of the work was to collect and systematize the data on the treatment approaches to pulmonary lymphangioleiomyomatosis (LAM) based on insights into the pathogenesis of the disease. Materials and methods. 70 original sources have been selected from analyzed 1 157 articles and monographs (including duplicates). The search for the sources was carried out in the databases of PubMed, eLibrary.ru, Cyberleninka for a fifty-year period of publications (from 1973 to August 2023), with an emphasis on more current publications and the ones in highly rated scientific journals. Results. The review presents the treatment approaches to LAM, based both on clinical observations of the disease course and on the experimental data on its probable pathogenesis. The collected data are presented in the chronological order, starting from radical methods based on the idea of an unconditional connection between the development of LAM and the female sex hormones. Special attention has been paid to the drugs from the group of mTOR inhibitors, including their safety profile. In addition, the results of the studies demonstrating new promising methods of the LAM drug therapy, both combining the use of mTOR inhibitors with other drugs, and the ones based on the isolated use of alternative groups of drugs, are presented in the work. Conclusion. The currently used methods of the drug therapy and the proposed new methods are aimed at only treating an already established disease, and the effective drug prevention of LAM now seems almost impossible due to the lack of a complete understanding about its pathogenesis and, more importantly, its etiology. This issue is the most relevant in determining further prospects for the development of pharmacotherapeutic approaches to LAM.
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Polovnikov et al. (2025) conducted a review in pulmonary lymphangioleiomyomatosis (LAM) (n=70). Treatment approaches (including mTOR inhibitors) was evaluated. Current treatment approaches for pulmonary lymphangioleiomyomatosis, including mTOR inhibitors, target established disease, but effective prevention remains impossible due to unknown etiology.
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