Expanded access study of patients with advanced basal cell carcinoma treated with the Hedgehog pathway inhibitor, vismodegib

Journal of the American Academy of Dermatology

Volumn 70, Issue 1, 2014, Pages 60-69

  Chang, Anne Lynn S ^a   Solomon, James A ^b,c,d   Hainsworth, John D ^e   Goldberg, Leonard ^f   McKenna, Edward ^g   Day, Bann Mo ^g   Chen, Diana M ^g   Weiss, Glen J ^h  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b Ameriderm Research   (United States)

^c SARAH CANNON RESEARCH INSTITUTE   (United States)

^d UNIVERSITY OF ILLINOIS   (United States)

^e UNIVERSITY OF CENTRAL FLORIDA COLLEGE OF MEDICINE   (United States)

^f DERMSURGERY ASSOCIATES   (United States)

^g GENENTECH INC   (United States)

^h Virginia G Piper Cancer Center   (United States)

Author keywords

basal cell carcinoma; basal cell nevus syndrome; expanded access; Hedgehog pathway inhibitor; locally advanced; metastatic; vismodegib

Indexed keywords

VISMODEGIB;

ADULT; ADVANCED CANCER; AGED; ALOPECIA; ARTICLE; BASAL CELL CARCINOMA; CANCER GROWTH; CANCER RADIOTHERAPY; CANCER SURGERY; COMPASSIONATE USE; DIARRHEA; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; DYSGEUSIA; FATIGUE; FEMALE; FOLLOW UP; HUMAN; MAJOR CLINICAL STUDY; MALE; MIDDLE AGED; MULTICENTER STUDY; MULTIPLE CYCLE TREATMENT; MUSCLE SPASM; NAUSEA; PRIORITY JOURNAL; SYSTEMIC THERAPY; TOXICITY; TREATMENT DURATION; TREATMENT RESPONSE; VERY ELDERLY; YOUNG ADULT;

EID: 84890796086     PISSN: 01909622     EISSN: 10976787     Source Type: Journal    
DOI: 10.1016/j.jaad.2013.09.012     Document Type: Article

References (28)

1. H.W. Rogers, M.A. Weinstock, A.R. Harris, M.R. Hinckley, S.R. Feldman, and A.B. Fleischer Incidence estimate of nonmelanoma skin cancer in the United States, 2006 Arch Dermatol 146 2010 283 287
2. A.I. Rubin, E.H. Chen, and D. Ratner Basal-cell carcinoma N Engl J Med 353 2005 2262 2269
3. T.L. Diepgen, and V. Mahler The epidemiology of skin cancer Br J Dermatol 146 Suppl 2002 1 6
4. H.W. Walling, S.W. Fosko, P.A. Geraminejad, D.C. Whitaker, and C.J. Arpey Aggressive basal cell carcinoma: presentation, pathogenesis, and management Cancer Metastasis Rev 23 2004 389 402
5. D.D. Von Hoff, P.M. LoRusso, C.M. Rudin, J.C. Reddy, R.L. Yauch, and R. Tibes Inhibition of the Hedgehog pathway in advanced basal-cell carcinoma N Engl J Med 361 2009 1164 1172
6. G.J. Weiss, and R.L. Korn Metastatic basal cell carcinoma in the era of Hedgehog signaling pathway inhibitors Cancer 118 2012 5310 5319
7. S.H. Amin, R. Tibes, J.E. Kim, and C.P. Hybarger Hedgehog antagonist GDC-0449 is effective in the treatment of advanced basal cell carcinoma Laryngoscope 120 2010 2456 2459
8. E. Varga, I. Korom, Z. Raskó, E. Kis, J. Varga, and J. Oláh Neglected basal cell carcinomas in the 21st century J Skin Cancer 2011 2011 392151
9. F. Cirrone, and C.S. Harris Vismodegib and the Hedgehog pathway: a new treatment for basal cell carcinoma Clin Ther 34 2012 2039 2050
10. D. Göppner, and M. Leverkus Basal cell carcinoma: from the molecular understanding of the pathogenesis to targeted therapy of progressive disease J Skin Cancer 2011 2011 650258
11. N.R. Telfer, G.B. Colver, and C.A. Morton Guidelines for the management of basal cell carcinoma Br J Dermatol 159 2008 35 48
12. C.S.M. Wong, R.C. Strange, and J.T. Lear Basal cell carcinoma BMJ 327 2003 794 798
13. P.T. Ting, R. Kasper, and J.P. Arlette Metastatic basal cell carcinoma: report of two cases and literature review J Cutan Med Surg 9 2005 10 15
14. A. Sekulic, M.R. Migden, A.E. Oro, L. Dirix, K.D. Lewis, and J.D. Hainsworth Efficacy and safety of vismodegib in advanced basal-cell carcinoma N Engl J Med 366 2012 2171 2179
15. P. Pfeiffer, O. Hansen, and C. Rose Systemic cytotoxic therapy of basal cell carcinoma: a review of the literature Eur J Cancer 26 1990 73 77
16. A. Wysong, S.Z. Aasi, and J.Y. Tang Update on metastatic basal cell carcinoma: a summary of published cases from 1981 through 2011 JAMA Dermatol 149 2013 615 616
17. J. Reifenberger, M. Wolter, R.G. Weber, M. Megahed, T. Ruzicka, and P. Lichter Missense mutations in SMOH in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system Cancer Res 58 1998 1798 1803
18. J. Xie, M. Murone, S.-M. Luoh, A. Ryan, Q. Gu, and C. Zhang Activating smoothened mutations in sporadic basal-cell carcinoma Nature 391 1998 90 92
19. L. Daya-Grosjean, and S. Couvé-Privat Sonic Hedgehog signaling in basal cell carcinomas Cancer Lett 225 2005 181 192
20. I. Caro, and J.A. Low The role of the Hedgehog signaling pathway in the development of basal cell carcinoma and opportunities for treatment Clin Cancer Res 16 2010 3335 3339
21. E.H. Epstein Basal cell carcinomas: attack of the Hedgehog Nat Rev Cancer 8 2008 743 754
22. C.D. Peacock, and C.M. Rudin Skin deep and deeper: multiple pathways in basal cell carcinogenesis Cancer Prev Res (Phila) 3 2010 1213 1216
23. T. Brinkhuizen, K. van den Hurk, V.J. Winnepenninckx, J.P. de Hoon, A.M. van Marion, and J. Veeck Epigenetic changes in basal cell carcinoma affect SHH and WNT signaling components PLoS One 7 2012 e51710
24. P.M. LoRusso, C.M. Rudin, J.C. Reddy, R. Tibes, G.J. Weiss, and M.J. Borad Phase I trial of Hedgehog pathway inhibitor vismodegib (GDC-0449) in patients with refractory, locally advanced or metastatic solid tumors Clin Cancer Res 17 2011 2502 2511
25. J.T. Lear Oral Hedgehog-pathway inhibitors for basal cell carcinoma N Engl J Med 366 2012 2225 2226
26. US Food and Drug Association. FDA approves new treatment for most common type of skin cancer [press release]. Silver Spring (MD). January 30, 2012. Available from: URL: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ ucm289545.htm. Accessed October 16, 2013.
27. Erivedge (vismodegib) [US prescribing information] 2012 Genentech Inc South San Francisco, CA
28. P. Therasse, S.G. Arbuck, E.A. Eisenhauser, J. Wanders, R.S. Kaplan, and L. Rubinstein New guidelines to evaluate the response to treatment in solid tumors J Natl Cancer Inst 92 2000 205 216