Subependymal giant cell astrocytoma

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Last updated on 2024-07-23:1 regimens on this page
1 variants on this page
=All lines of therapy=
==Everolimus monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator	Comparative Efficacy
Krueger et al. 2010 (CCHMC 06-07-50)	2007-2008 	Phase 1/2 (RT) 	 	
Franz et al. 2012 (EXIST-1) 	2009-2013 	Phase 3 (E-RT-esc)	Placebo 	Higher confirmed response rate (primary endpoint)
====Targeted therapy====
*Everolimus (Afinitor) 4.5 mg/m2 PO once per day (see titration instructions below)
Continued indefinitely
====Dose and schedule modifications====
*Everolimus titrated to achieve blood trough concentrations of 5 to 15 ng/mL
===References===
#CCHMC 06-07-50: Krueger DA, Care MM, Holland K, Agricola K, Tudor C, Mangeshkar P, Wilson KA, Byars A, Sahmoud T, Franz DN. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med. 2010 Nov 4;363(19):1801-11. link to original article EuroPMC NCT00411619
##Update: Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN. Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. Neurology. 2013 Feb 5;80(6):574-80. Epub 2013 Jan 16. link to original article link to PMC article EuroPMC
##Update: Franz DN, Agricola K, Mays M, Tudor C, Care MM, Holland-Bouley K, Berkowitz N, Miao S, Peyrard S, Krueger DA. Everolimus for subependymal giant cell astrocytoma: 5-year final analysis. Ann Neurol. 2015 Dec;78(6):929-38. Epub 2015 Nov 9. link to original article link to PMC article EuroPMC
# EXIST-1: Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Kuperman R, Witt O, Kohrman MH, Flamini JR, Wu JY, Curatolo P, de Vries PJ, Whittemore VH, Thiele EA, Ford JP, Shah G, Cauwel H, Lebwohl D, Sahmoud T, Jozwiak S. Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet. 2013 Jan 12;381(9861):125-32. Epub 2012 Nov 14. Erratum in: Lancet. 2013 Jan 12;381(9861):116. link to original article dosing details in abstract have been reviewed by our editors EuroPMC NCT00789828
##Update: Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Kuperman R, Witt O, Kohrman MH, Flamini JR, Wu JY, Curatolo P, de Vries PJ, Berkowitz N, Anak O, Niolat J, Jozwiak S. Everolimus for subependymal giant cell astrocytoma in patients with tuberous sclerosis complex: 2-year open-label extension of the randomised EXIST-1 study. Lancet Oncol. 2014 Dec;15(13):1513-1520. Epub 2014 Nov 10. link to original article EuroPMC
##Update: Franz DN, Belousova E, Sparagana S, Bebin EM, Frost MD, Kuperman R, Witt O, Kohrman MH, Flamini JR, Wu JY, Curatolo P, de Vries PJ, Berkowitz N, Niolat J, Jóźwiak S. Long-Term Use of Everolimus in Patients with Tuberous Sclerosis Complex: Final Results from the EXIST-1 Study. PLoS One. 2016 Jun 28;11(6):e0158476. link to original article link to PMC article EuroPMC
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