Tuberous sclerosis complex lesions respond well to everolimus in EXIST

By Helen Albert, Senior medwireNews Reporter

Results from two phase III trials demonstrate significantly better skin lesion reduction with everolimus than placebo in patients with tuberous sclerosis complex (TSC).

Sergiusz Jóźwiak (The Children's Memorial Health Institute of Warsaw, Poland) presented the results of EXamining everolimus In a Study of TSC (EXIST) 1 and 2 at the European Academy of Dermatology and Venereology annual congress in Prague, Czech Republic.

EXIST-1 looked at the efficacy of the Mammalian Target of Rapamycin (mTOR) inhibitor for treatment of subependymal giant cell astrocytoma (SEGA) associated with TSC, and EXIST-2 examined the efficacy for treatment of renal angiomyolipoma (AML) associated with either TSC or sporadic lymphangioleiomyomatosis.

In EXIST-1, patients were randomly assigned to receive everolimus (n=78) at a starting dose of 4.5 mg/m2 per day (target trough, 5-15 ng/mL) or placebo (n=39) for up to 6 months, after which time they could enter a longer term extension phase.

In EXIST-2, patients received everolimus 10 mg/day (n=79) or placebo (n=39) until first documented AML progression, date of further anti-AML medication (including open label everolimus or surgery), or analysis cutoff date.

Patients with SEGA and AML both responded significantly better to treatment with everolimus than placebo in EXIST-1 and 2 (primary endpoint).

Looking specifically at skin lesion response according to a 7-point Physician's Global Assessment of Clinical Condition tool (secondary endpoint), Jóźwiak and team found that significantly more patients had at least a partial response to everolimus compared with placebo in both trials, at 41.7% versus 10.5% in EXIST-1 and 26.0% versus 0.0% in EXIST-2.

The researchers note that no patients had a complete clinical response or progressive disease in either arm in either trial.

Patients with a partial response to everolimus also had a higher minimum concentration of everolimus than patients with stable disease at weeks 12 and 24 in both trials.

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