Subependymal giant cell astrocytoma

From HemOnc.org - A Hematology Oncology Wiki
Jump to navigation Jump to search

Section editor
SeemaNagpal.jpg
Seema Nagpal, MD
Stanford University
Palo Alto, CA

If you are looking for other subtypes of brain cancer, please go to the CNS cancers category page.

1 regimens on this page
1 variants on this page


All lines of therapy

Everolimus monotherapy

Regimen

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) 2009-2013 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

  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
    1. 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 PubMed
    2. 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 PubMed
  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 dosing details in abstract PubMed NCT00789828
    1. 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 PubMed
    2. 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 PubMed