Rapamycin and rapalogs for tuberous sclerosis complex

TH Sasongko, K Kademane, SCS Hou… - Cochrane Database …, 2023 - cochranelibrary.com
TH Sasongko, K Kademane, SCS Hou, TXY Jocelyn, Z Zabidi-Hussin
Cochrane Database of Systematic Reviews, 2023cochranelibrary.com
Background Potential benefits of rapamycin or rapalogs for treating people with tuberous
sclerosis complex (TSC) have been shown. Currently everolimus (a rapalog) is only
approved for TSC‐associated renal angiomyolipoma and subependymal giant cell
astrocytoma (SEGA), but not other manifestations of TSC. A systematic review needs to
establish evidence for rapamycin or rapalogs for various manifestations in TSC. This is an
updated review. Objectives To determine the effectiveness of rapamycin or rapalogs in …
Abstract
Background
Potential benefits of rapamycin or rapalogs for treating people with tuberous sclerosis complex (TSC) have been shown. Currently everolimus (a rapalog) is only approved for TSC‐associated renal angiomyolipoma and subependymal giant cell astrocytoma (SEGA), but not other manifestations of TSC. A systematic review needs to establish evidence for rapamycin or rapalogs for various manifestations in TSC. This is an updated review.
Objectives
To determine the effectiveness of rapamycin or rapalogs in people with TSC for decreasing tumour size and other manifestations and to assess the safety of rapamycin or rapalogs in relation to their adverse effects.
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