Researchers have shown that the immunosuppressant everolimus provides a potential new treatment option for patients with tuberous sclerosis and associated angiomyolipomas.
In a randomized, phase III trial, the drug was significantly more effective than placebo at reducing angiomyolipoma volume and also demonstrated an acceptable safety profile, they report.
"This finding opens an alternative avenue for the treatment of patients who are not amenable to surgical intervention," say John Bissler (Cincinnati Children's Hospital Medical Center, Ohio, USA) and colleagues. They point out that 40% of the patients in their trial had already undergone an invasive surgical procedure but still required intervention, highlighting the limitations of this approach.
As reported in The Lancet, 33 (42%) of 79 patients who received everolimus 10 mg/day had a reduction in angiomyolipoma of 50% or more and were free of angiomyolipoma progression after a median drug exposure of 38 weeks, while no such response was observed in the placebo group.
In an accompanying commentary, Prasanna Sooriakumaran and Christopher Anderson from St George's National Health Service Trust in London, UK, say this is "a highly significant and, we believe, clinically significant finding."
The median time to angiomyolipoma response was 2.9 months and the treatment effect remained consistent when patients were stratified by strata, gender, age, or race.
Six months after baseline assessment, angiomyolipomas had not progressed in 98% of patients who received everolimus, compared with 83% of the placebo group. And after 12 months, 92% of those who received everolimus remained progression-free, while only 25% who received placebo were progression-free.
The most common adverse events with everolimus treatment were stomatitis, nasopharyngitis, and acne-like lesions, which occurred in 48%, 24%, and 22% of patients, compared with 8%, 31%, and 5% of those who took the placebo treatment. However, these adverse events were mostly mild and generally managed while patients continued everolimus treatment, reports the team.
"We congratulate the authors of this study," say Soorakumaran and Anderson. "Their results are promising and are likely to herald a new approach to the treatment of this rare but serious disease."
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