Hedgehog pathway inhibitor therapy for locally advanced and metastatic basal cell carcinoma a systematic review and pooled analysis of interventional studies

JAMA Dermatology

Volumn 152, Issue 7, 2016, Pages 816-824

  Jacobsen, Audrey A ^a   Aldahan, Adam S ^a   Hughes, Olivia B ^a   Shah, Vidhi V ^a   Strasswimmer, John ^a  

^a UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PLACEBO; SONIDEGIB; VISMODEGIB; ANILIDE; ANTINEOPLASTIC AGENT; BIPHENYL DERIVATIVE; PYRIDINE DERIVATIVE; SMOOTHENED PROTEIN; SONIC HEDGEHOG PROTEIN;

ALOPECIA; AMENORRHEA; BASAL CELL CARCINOMA; CLINICAL TRIAL (TOPIC); DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; DYSGEUSIA; FATIGUE; HUMAN; LOCALLY ADVANCED BASAL CELL CARCINOMA; MEDICAL RECORD; METASTATIC BASAL CELL CARCINOMA; MUSCLE SPASM; PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); RETROSPECTIVE STUDY; REVIEW; SIDE EFFECT; SQUAMOUS CELL CARCINOMA; SYSTEMATIC REVIEW; TREATMENT DURATION; WEIGHT REDUCTION; ANTAGONISTS AND INHIBITORS; CARCINOMA, BASAL CELL; META ANALYSIS; PATHOLOGY; SECONDARY; SKIN NEOPLASMS;

ANILIDES; ANTINEOPLASTIC AGENTS; BIPHENYL COMPOUNDS; CARCINOMA, BASAL CELL; HEDGEHOG PROTEINS; HUMANS; PYRIDINES; SKIN NEOPLASMS; SMOOTHENED RECEPTOR;

EID: 84979708928     PISSN: 21686068     EISSN: None     Source Type: Journal    
DOI: 10.1001/jamadermatol.2016.0780     Document Type: Review

References (46)

1. Asgari MM, Moffet HH, Ray GT, Quesenberry CP. Trends in basal cell carcinoma incidence and identification of high-risk subgroups, 1998-2012. JAMA Dermatol. 2015;151(9):976-981.
2. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. N Engl J Med. 2009;361(12): 1164-1172.
3. Tang JY, Mackay-Wiggan JM, Aszterbaum M, et al. Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndrome. N Engl J Med. 2012;366(23):2180-2188.
4. McCusker M, Basset-Seguin N, Dummer R, et al. Metastatic basal cell carcinoma: prognosis dependent on anatomic site and spread of disease. Eur J Cancer. 2014;50(4):774-783.
5. Danial C, Sarin KY, Oro AE, Chang AL. An investigator-initiated open-label trial of sonidegib in advanced basal cell carcinoma patients resistant to vismodegib [published online November 6, 2015]. Clin Cancer Res. 2015. doi:10.1158/1078-0432.CCR-15-1588.
6. Chang AL, Solomon JA, Hainsworth JD, et al. Expanded access study of patients with advanced basal cell carcinoma treated with the Hedgehog pathway inhibitor, vismodegib. J AmAcad Dermatol. 2014;70(1):60-69.
7. LoRusso PM, Rudin CM, Reddy JC, et al. Phase I trial of hedgehog pathway inhibitor vismodegib (GDC-0449) in patients with refractory, locally advanced or metastatic solid tumors. Clin Cancer Res. 2011;17(8):2502-2511.
8. Basset-Seguin N, Hauschild A, Grob JJ, et al. Vismodegib in patients with advanced basal cell carcinoma (STEVIE): A pre-planned interim analysis of an international, open-label trial. Lancet Oncol. 2015;16(6):729-736.
9. US Food and Drug Administration. Prescribing Information for Erivedge. 2012; http://www.accessdata.fda.gov/drugsatfda-docs/label/2012/203388lbl.pdf. Accessed October 17, 2015.
10. Peris K, Licitra L, Ascierto PA, et al. Identifying locally advanced basal cell carcinoma eligible for treatment with vismodegib: An expert panel consensus. Future Oncol. 2015;11(4):703-712.
11. Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med. 2012;366(23):2171-2179.
12. Sekulic A, Migden MR, Lewis K, et al; ERIVANCE BCC investigators. Pivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC. J AmAcad Dermatol. 2015;72(6):1021-6.e8.
13. Moher D, Liberati A, Tetzlaff J, Altman DG; PRISMA Group. Preferred reporting items for systematic reviews and meta-Analyses: The PRISMA statement. BMJ. 2009;339:b2535.
14. Simone PSJ, Strasswimmer J. Four year experience of vismodegib hedgehog inhibitor therapy. J AmAcad Dermatol. In press.
15. Viscusi KS, Hanke CW. Vismodegib for locally advanced basal cell carcinoma: descriptive analysis of a case series and comparison to the literature. J Drugs Dermatol. 2015;14(9):956-962.
16. Gill HS, Moscato EE, Chang AL, Soon S, Silkiss RZ. Vismodegib for periocular and orbital basal cell carcinoma. JAMA Ophthalmol. 2013;131(12):1591-1594.
17. Ozgur OK, Yin V, Chou E, et al. Hedgehog pathway inhibition for locally advanced periocular basal cell carcinoma and basal cell nevus syndrome. Am J Ophthalmol. 2015;160(2):220-227.e2.
18. Demirci H, Worden F, Nelson CC, Elner VM, Kahana A. Efficacy of vismodegib (erivedge) for basal cell carcinoma involving the orbit and periocular area. Ophthal Plast Reconstr Surg. 2015;31(6):463-466.
19. Rodon J, Tawbi HA, Thomas AL, et al. A phase I, multicenter, open-label, first-in-human, dose-escalation study of the oral smoothened inhibitor Sonidegib (LDE225) in patients with advanced solid tumors. Clin Cancer Res. 2014;20 (7):1900-1909.
20. Migden MR, Guminski A, Gutzmer R, et al. Treatment with two different doses of sonidegib in patients with locally advanced or metastatic basal cell carcinoma (BOLT): Amulticentre, randomised, double-blind phase 2 trial. Lancet Oncol. 2015;16(6): 716-728.
21. Robinson JK, Dellavalle RP, Bigby M, Callen JP. Systematic reviews: grading recommendations and evidence quality. Arch Dermatol. 2008;144(1):97-99.
22. Dummer Rea. Impact of treatment breaks on vismodegib patient outcomes: exploratory analysis of the STEVIE study. Paper presented at: 51st American Society of Clinical Oncology Annual Meeting; May 29-June 2, 2015; Chicago, Illinois.
23. Yang X, Dinehart SM. intermittent vismodegib therapy in basal cell nevus syndrome. JAMA Dermatol. 2016;152(2):223-224.
24. Ally MS, Aasi S, Wysong A, et al. An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. J AmAcad Dermatol. 2014;71(5):904-911.e1.
25. Ash MM, Jolly PS. Cholestatic hepatic injury associated with vismodegib, aspirin, and naproxen use: A case study and review of vismodegib safety. Int J Dermatol. 2015;54(3):370-374.
26. Ventarola DJ, Silverstein DI. Vismodegib-Associated hepatotoxicity: A potential side effect detected in postmarketing surveillance. J AmAcad Dermatol. 2014;71(2):397-398.
27. Strasswimmer J, Latimer B, Ory S. Amenorrhea secondary to a vismodegib-induced blockade of follicle-stimulating hormone-receptor activation. Fertil Steril. 2014;102(2):555-557.
28. Kimura H, Ng JM, Curran T. Transient inhibition of the Hedgehog pathway in youngmice causes permanent defects in bone structure. Cancer Cell. 2008;13(3):249-260.
29. Lipinski RJ, Hutson PR, Hannam PW, et al. Dose-And route-dependent teratogenicity, toxicity, and pharmacokinetic profiles of the hedgehog signaling antagonist cyclopamine in the mouse. Toxicol Sci. 2008;104(1):189-197.
30. Lipinski RJ, Song C, Sulik KK, et al. Cleft lip and palate results from Hedgehog signaling antagonism in the mouse: Phenotypic characterization and clinical implications. Birth Defects Res A Clin Mol Teratol. 2010;88(4):232-240.
31. Huizenga T, Newsom E, Fakhouri T, Kado J. New-onset congestive heart failure in a patient on vismodegib. Dermatol Surg. 2015;41(11):1329-1332.
32. Hernandez C, Kim H, Mauleon G, Ruiz A, Robinson JK, Mermelstein RJ. A pilot program in collaboration with community centers to increase awareness and participation in skin cancer screening among Latinos in Chicago. J Cancer Educ. 2013;28(2):342-345.
33. US Food and Drug Administration. Prescribing Information for ODOMZO (sonidegib) 2015; http://www.accessdata.fda.gov/drugsatfda-docs/label/2015/205266s000lbl.pdf. Accessed January 22, 2016.
34. Dreno B, Basset-Seguin N, Caro I, Yue H, Schadendorf D. Clinical benefit assessment of vismodegib therapy in patients with advanced basal cell carcinoma. Oncologist. 2014;19(8):790-796.
35. Pollom EL, Bui TT, Chang AL, Colevas AD, Hara WY. concurrent vismodegib and radiotherapy for recurrent, advanced basal cell carcinoma. JAMA Dermatol. 2015;151(9):998-1001.
36. Strasswimmer JM. Potential synergy of radiation therapy with vismodegib for basal cell carcinoma. JAMA Dermatol. 2015;151(9):925-926.
37. Schulze B, Meissner M, Ghanaati S, Burck I, Rödel C, Balermpas P. Hedgehog pathway inhibitor in combination with radiation therapy for basal cell carcinomas of the head and neck: first clinical experience with vismodegib for locally advanced disease. Strahlenther Onkol. 2016;192(1):25-31. Published online 2015.
38. Alcalay J, Tauber G, Fenig E, Hodak E. Vismodegib as a neoadjuvant treatment to Mohs surgery for aggressive basal cell carcinoma. J Drugs Dermatol. 2015;14(3):219-223.
39. Efficacy NIH; Safety and pharmacokinetics of oral LDE225 in treatment of patients with nevoid basal cell carcinoma syndrome (NBCCS). 2015; https://clinicaltrials.gov/ct2/show/results/NCT01350115?term=gorlin&rank=2§=X970156. Accessed November 23, 2015.
40. Belum VR, Marulanda K, Ensslin C, et al. Alopecia in patients treated with molecularly targeted anticancer therapies. Ann Oncol. 2015;26 (12):2496-2502.
41. Alkeraye S, Maire C, Desmedt E, Templier C, Mortier L. Persistent alopecia induced by vismodegib. Br J Dermatol. 2015;172(6):1671-1672.
42. Wang C, Wu H, Katritch V, et al. Structure of the human smoothened receptor bound to an antitumour agent. Nature. 2013;497(7449):338-343.
43. Dummer R, Sondak VK, Grichnik JM, et al Composite assessment of treatment response in patients with locally advanced basal cell carcinoma (laBCC): sonidegib efficacy using two sets of response criteria. J Clin Oncol. 2015;33(suppl; abstr e20055).
44. Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45(2):228-247.
45. Maly TJ, Sligh JE. Defining locally advanced Basal cell carcinoma. J Drugs Dermatol. 2014;13(5): 528-529.
46. Robinson JK, Dellavalle RP, Bigby M, Callen JP. Systematic reviews: grading recommendations and evidence quality. Arch Dermatol. 2008;144(1):97-99.