Everolimus Reduces Angiomyolipoma Volume
Findings in patients with tuberous sclerosis or lymphangioleiomyomatosis-linked angiomyolipomata
FRIDAY, Jan. 11 (HealthDay News) -- For patients with tuberous sclerosis or sporadic lymphangioleiomyomatosis-associated angiomyolipomata, the angiomyolipoma response rate is significantly higher with everolimus than placebo, with an acceptable safety profile, according to a study published online Jan. 11 in The Lancet.
John J. Bissler, M.D., from the Cincinnati Children's Hospital Medical Center, and colleagues conducted a double-blind, placebo-controlled, phase 3 trial comparing the angiomyolipoma response rate for everolimus versus placebo in 118 patients with tuberous sclerosis or sporadic lymphangioleiomyomatosis-associated angiomyolipomata from 24 centers in 11 countries. Participants were assigned in a 2:1 ratio to everolimus (79 patients) or placebo (39 patients).
At the data cut-off, 98 patients were still receiving double-blind treatment, with discontinuation mainly due to disease progression (nine placebo patients) and adverse events (two everolimus patients and four placebo patients). The researchers found that the angiomyolipoma response rate was significantly higher with everolimus than placebo (42 versus 0 percent). In the everolimus and placebo groups, the most common adverse events were stomatitis (48 and 8 percent, respectively), nasopharyngitis (24 and 31 percent, respectively), and acne-like skin lesions (22 and 5 percent, respectively).
"The results of this trial show the benefit of everolimus for treating angiomyolipomas associated with tuberous sclerosis complex," the authors write.
Several authors are employees or consultants for Novartis, which markets everolimus and funded the study. Another author disclosed financial ties to the pharmaceutical industry.