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A phase II clinical trial of the Safety Of Simvastatin (SOS) in patients with pulmonary lymphangioleiomyomatosis and with tuberous sclerosis complex

Respiratory Medicine Feb 15, 2020

Krymskaya VP, Courtwright AM, Fleck V, et al. - By performing this phase II single arm trial, researchers evaluated whether simvastatin with either sirolimus or everolimus would be safe to treat patients with pulmonary lymphangioleiomyomatosis (LAM). They assessed the safety of escalating daily simvastatin (20–40 mg) in LAM patients previously managed with sirolimus or everolimus. Evaluation of adverse events as well as alterations in lipid panel profile, pulmonary function tests, and VEGF-D was performed. In this study, 20 mg simvastatin for two months followed by 40 mg for two months was administered to 10 LAM patients on a stable dose of mTORi (mechanistic target of rapamycin inhibitors) for > 3 months. Peripheral edema (30%), cough (30%), and diarrhea (30%) were documented as the adverse events that were most common. During the study span, a reduction was evident in total cholesterol and low density lipoproteins. FEV1 also got reduced but no significant alteration was evident in FVC, DLCO, or VEGF-D. Findings revealed safety as well as good tolerability of the combination of simvastatin with mTORi in LAM patients, from an adverse events perspective. However, FEV1 reduction was noted in relation to the addition of simvastatin.
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