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mTOR inhibitors
Source: Childhood Astrocytomas, Other Gliomas, and Glioneuronal/Neuronal Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 14, 2025 · Captured 2026-09-09.
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For children with tuberous sclerosis (TS) and symptomatic subependymal giant cell astrocytomas (SEGAs) or low-grade gliomas,[18] mammalian target of rapamycin (mTOR) inhibitors (e.g., everolimus and sirolimus) have been studied.
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Evidence (mTOR inhibitors):
Small series have shown significant reductions in the size of these tumors after administration of everolimus or sirolimus, often eliminating the need for surgery.[19]; [20][Level of evidence B4]; [21][Level of evidence C3]; [22][Level of evidence C1]
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A multicenter, phase III, placebo-controlled trial of 117 patients confirmed these earlier findings.[23][Level of evidence B3]
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A multicenter, phase III, placebo-controlled trial of 117 patients confirmed these earlier findings.[23][Level of evidence B3]
Thirty-five percent of the patients in the everolimus group had at least a 50% reduction in the size of the SEGA, versus no reduction in the placebo group.
In a study of patients who were treated with everolimus for 5 years, the following results were observed:[24]
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In a study of patients who were treated with everolimus for 5 years, the following results were observed:[24]
A reduction in the size of the mass was observed in about 50% of patients; in many cases, the reduction was sustained.
In a study of patients who were treated with everolimus for 5 years, the following results were observed:[24]
These patients also had a reduction in seizure frequency.
In a series of 23 patients with recurrent low-grade gliomas who were treated with everolimus, the following was observed:[25]
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In a series of 23 patients with recurrent low-grade gliomas who were treated with everolimus, the following was observed:[25]
Everolimus demonstrated modest activity, with a 2-year progression-free survival (PFS) rate of 39% and an overall survival rate of 93%.
A companion study completed by the Neurofibromatosis Clinical Trials Consortium evaluated 23 children with neurofibromatosis type 1 (NF1) and progressive low-grade gliomas who were treated with everolimus.[26]
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A companion study completed by the Neurofibromatosis Clinical Trials Consortium evaluated 23 children with neurofibromatosis type 1 (NF1) and progressive low-grade gliomas who were treated with everolimus.[26]
Of the 22 evaluable patients, 15 demonstrated either a partial response or tumor stabilization, 10 of whom remained free of progression for a median follow-up of 33 months.
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Preserved source evidence · Not medical advice