کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
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6222178 | 1607455 | 2014 | 6 صفحه PDF | دانلود رایگان |
ObjectivesTo evaluate the efficacy and side effects of oral mammalian target of rapamycin (mTOR) inhibitors in children and adolescents with tuberous sclerosis complex (TSC) and intractable epilepsy or subependymal giant cell astrocytoma (SEGA).Study designSingle-center series of 13 children and adolescents with TSC who received sirolimus or everolimus (mTOR inhibitors). The anticonvulsant response was evaluated in 7 patients with TSC and refractory seizures. Six patients with SEGAs were treated with either sirolimus or everolimus for nonsurgical management. SEGA volumes were assessed longitudinally using 1.5-T magnetic resonance imaging.ResultsOf the intractable seizure group (7 patients), 1 patient had >90% reduction, 4 had 50%-90% reduction, and 2 had <50% reduction. Three reported subjective improvements in learning. By 12Â months of treatment, there were statistically significant reductions in the SEGA volumes in 4 patients who received mTOR inhibitors (PÂ <Â .04). The mean SEGA volume after 6Â months of treatment was 2.18Â cm3, which represents 33% reduction in the mean baseline volume of 3.26Â cm3. The mTOR inhibitors were well tolerated. Adverse effects include dyslipidaemia (3 of 13), gingivitis (1 of 13), anorexia (1 of 13), and mild gastrointestinal side effects (1 of 13).ConclusionThis case series suggests that mTOR inhibitors can improve seizures in those with TSC and refractory epilepsy. They are also an effective treatment for reducing the volume of SEGAs in patients with TSC not amenable to surgery with an acceptable side effect profile.
Journal: The Journal of Pediatrics - Volume 164, Issue 5, May 2014, Pages 1195-1200