Review Evaluates Pharmacological Treatments for Rare Neoplastic Disease

A systematic review and meta-analysis of 10 studies to evaluate the efficacy and safety of pharmacological treatments for lymphangioleiomyomatosis (LAM) yielded mixed results, according to the findings published in Respiratory Research.

LAM is a low-grade multisystem neoplastic disease that results in cystic lung destruction, angiomyolipomas (AMLs), chylous fluid accumulation, and lymphangioleiomyomas. The disease, which almost exclusively affects adult females, can occur on its own or is associated with tuberous sclerosis complex. LAM is estimated to affect 5 in every 1 million people and has also been reported in adult males and children. For those in the end stage of the disease, lung transplantation is the only treatment option, but LAM can recur in transplanted lungs.

To conduct the review, researchers identified studies from sources such as PubMed, EMBASE, Cochrane Library, and others. In total, data from 330 patients enrolled in prospective clinical trials or randomized control trials were included in the review.

Pharmacological treatments included sirolimus, everolimus, doxycycline, triptorelin, and a combination of sirolimus and hydroxychloroquine. Adverse events and side effects occurred with each treatment option, but most “were reported to be low or moderate grade and were tolerable.”

Overall, the review supports “the application of sirolimus and everolimus in LAM, as these medications may stabilize lung function and alleviate renal AML.”

Specifically, 7 prospective studies reported that sirolimus was “effective at improving or stabilizing lung function and alleviating renal AML in LAM patients.” Three prospective studies on everolimus “reported similar effects…with regard to preserving lung function and reducing AMLs.” Researchers noted that further studies are required to determine the optimal timing, duration, and dosage of these treatments.

The review found no consistent evidence that doxycycline and triptorelin yielded any beneficial outcomes for patients, and further research needs to be conducted on the efficacy and safety of the combination therapy.

Researchers pointed out that because LAM is so rare, it is difficult to conduct the necessary studies needed to explore treatment options for the disease. “Generally, the prospect is still not optimistic in the treatment of LAM…the results of the current meta-analysis support only the opinion that [sirolimus and everolimus] are effective at stabilizing the condition,” the study authors noted.

However, results from 2 ongoing multicenter international trials “will help us better understand” the optimal usage of everolimus and sirolimus in patients with LAM, they wrote.

Reference

Qi W, Luo M, Xiang B, et al. The efficacy and safety of pharmacological treatments for lymphangioleiomyomatosis. Respir Res. 2020;21:55. doi: 10.1186/s12931-020-1316-3.