A Mechanistic Target of Rapamycin (mTOR) Inhibitor, Everolimus Safely Ameliorated Lupus Nephritis in a Patient Complicated with Tuberous Sclerosis.

A 26-year-old woman with tuberous sclerosis complex (TSC) received outpatient treatment for the complication of systemic lupus erythematosus (SLE) at our hospital. She visited our hospital with a chief complaint of pitting edema in bilateral lower legs for three days. The urinalysis showed massive proteinuria with a lot of WBC casts. The blood tests revealed hypoalbuminemia, hypercholesterolemia, hypocomplementemia and elevated anti-ds-DNA antibody titer. Renal biopsy was not performed because of multiple renal angiomyolipomas, which was one of the features of TSC. She was diagnosed with a nephrotic state due to lupus nephritis (LN). Although she had a standard therapy with high-dose corticosteroid and mycophenolate mofetil (MMF) and tacrolimus, complete remission had not been achieved leading to steroid-dependent nephrotic syndrome. During the following-up, the angiomyolipomas became larger and had a risk of rupture at the age of 29. Everolimus, a mechanistic target of rapamycin (mTOR) inhibitor, was started for the treatment of angiomyolipomas, and MMF and tacrolimus were terminated instead. The activity of LN was surprisingly ameliorated, and the amount of corticosteroid successfully tapered. Everolimus has been continued for six years without severe side effects. Accumulating evidence suggests that the activated mTOR pathway plays a key role in the pathogenesis of SLE. We reported the long-term efficacy and safety of everolimus for refractory SLE in a patient with TSC for the first time. This case suggests that everolimus can be a promising option for the treatment of lupus nephritis.