Subependymal giant cell astrocytoma

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Seema Nagpal, MD
Stanford University
Palo Alto, CA

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1 regimens on this page
1 variants on this page

All lines of therapy

Everolimus monotherapy

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Study Years 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-NR in abstract Phase 3 (E-RT-esc) Placebo Superior confirmed response rate

Targeted therapy

  • Everolimus (Afinitor) as follows:
    • Starting dose: 4.5 mg/m2 PO once per day
    • Goal dose: Titrated to achieve blood trough concentrations of 5 to 15 ng/mL

Continued indefinitely


  1. 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 PubMed NCT00411619
  2. 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 contains protocol PubMed NCT00789828