Source: International Congress 2014 – New treatments for cough, asthma, COPD and ILDs

Abstract

Sirolimus is a mammalian target of rapamycin (mTOR) inhibitor, was recently shown to stabilize lung function and reduce the size of chylous effusions in patients with LAM. The optimal doses of mTOR inhibitors for the treatment of LAM are not clear, but in recent retrospective trail low-dose therapy with sirolimus was clinically beneficial in LAM patients (Ando K et al., Respir Invest 2013; 51: 175-183). We present here our first experience of low-dose everolimus treatment in LAM patients.Methods:Ten women with sporadic LAM (mean age 41.4 ± 6.4 years) with clinically significant lung disease (FEV1 42.3±16.7%, FVC 77.9± 22.6%, DLCO 33.3±11.7%) were included in our open-label study. Five patients had abdominal angiomyolipomas (AML), four patients had chylous effusion, and one - chylous ascite. Patients received everolimus 1-2 mg daily from 6 month to 3 years (in all patients the blood trough levels of everolimus was lower than 5 ng/mL).Results:There was a complete resolution of chylous complications in all four patients within 1 to 2 months after the initiation of therapy with everolimus. A significant shrinkage of ALM was noted in four women. PFTs also showed substantial improvement: after first 6 months of treatment FEV1 were 46.6±17.9%, FVC - 80.6±13.9% and DLCO - 40.0± 20.6%. The distance in 6-MWD increased by 56.5±19.3 meters. The therapy with low-dose everolimus was well tolerated, and only two patients experienced adverse events: headache (1) and hypercholesterolemia (1).Conclusions: Our findings suggest a potential role for low-dose everolimus treatment in patients with sporadic LAM.

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Citations should be made in the following way:
S. Avdeev, M. Makarova, Z. Merzhoeva, A. Chuchalin (Moscow, Russian Federation). Treatment with low-dose everolimus in patients with sporadic lymphangioleiomyomatosis (LAM). Eur Respir J 2014; 44: Suppl. 58, 1519

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