Subependymal giant cell astrocytoma
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Seema Nagpal, MD Stanford University Palo Alto, CA |
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All lines of therapy
Everolimus monotherapy
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Regimen
Study | Years of enrollment | Evidence | Comparator | Comparative Efficacy |
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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
References
- 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