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Rapamycin and its analogues (rapalogs) for Tuberous Sclerosis Complex-associated tumors: a systematic review on

Teguh Haryo Sasongko1,2, Nur Farrah Dila Ismail3,4, Nik Mohamad Ariff Nik Abdul Malik3,4

  • 1Human Genome Center, School of Medical Sciences, Universiti Sains Malaysia, USM Health Campus, 16150, Kubang Kerian, Kelantan, Malaysia. teguhharyosasongko@yahoo.com.

Orphanet Journal of Rare Diseases
|August 12, 2015
PubMed
Abstract

Insights

Rapamycin and rapalogs effectively reduce the size of subependymal giant cell astrocytomas (SEGAs) and kidney angiomyolipomas in Tuberous Sclerosis Complex (TSC) patients. However, these drugs did not show significant efficacy in reducing liver angiomyolipomas.

Area of Science:

  • Oncology
  • Pharmacology
  • Genetics

Background:

  • Tuberous Sclerosis Complex (TSC) is a genetic disorder characterized by the growth of benign tumors in various organs.
  • TSC-associated tumors, particularly subependymal giant cell astrocytomas (SEGAs) and angiomyolipomas, can cause significant morbidity.
  • Rapamycin and its analogs (rapalogs) have emerged as potential therapeutic agents for managing TSC-associated tumors, offering an alternative to surgical interventions.

Purpose of the Study:

  • To determine the efficacy of rapamycin and rapalogs in reducing the size of TSC-associated solid tumors.
  • To evaluate the impact of these drugs on SEGAs, kidney angiomyolipomas, and liver angiomyolipomas in patients with Tuberous Sclerosis Complex.

Main Methods:

  • A meta-analysis was conducted using data from non-randomized studies.
  • Electronic searches of PubMed were performed to identify relevant studies.
  • Data from case reports and clinical trials were analyzed using Cochrane Review Manager Version 5.3.

Main Results:

  • Rapamycin and rapalogs significantly reduced the volume and diameter of SEGAs.
  • Significant reductions in volume and diameter were observed for kidney angiomyolipomas.
  • No significant reduction in tumor size was observed for liver angiomyolipomas; some cases showed slight enlargement.

Conclusions:

  • Rapamycin and rapalogs demonstrate efficacy in reducing the size of SEGAs and kidney angiomyolipomas in TSC patients.
  • These findings support the use of rapamycin and rapalogs as a treatment option for specific TSC-associated tumors.
  • Further research may be needed to explore alternative treatments for liver angiomyolipomas in TSC.

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