Original Article

A Non-interventional Study on Vismodegib for Basal Cell Carcinoma With A Treat-To-Target Regimen In Swedish Patients

Author Affiliation(s)


Introduction: Real-life data on vismodegib in advanced basal cell carcinoma (aBCC) are limited. Optimal treatment duration is left to the discretion of the physician.

Objectives: To assess the effectiveness, safety and treatment pattern for vismodegib in aBCC in clinical practice.

Methods: In this multicenter, non-interventional, prospective study, 49 Swedish patients planned for vismodegib treatment were included. The treatment pattern observed was treatment until remission, allowing unlimited discontinuations/pauses.

Results: The majority of patients (93.8%), discontinued at least once during the study. Compared to earlier studies there was a decrease of more than 2 months with actual drug intake, reducing the patients burden and costs, at the same time as a high number of responses were seen (87.8%). Median progression-free-survival was 16.7 months, and 90% of the patients were alive at 13.3 months. Ten patients were re-challenged with vismodegib at recurrence or progression, resulting in five partial remissions and three complete remissions. 

Conclusions: Clinical response rates with vismodegib for aBCC were similar to those of similar trials despite a shorter and more intermittent treatment duration. The majority of re-challenges lead to partial or complete remissions.

Keywords : Non-interventional, prospective, effectiveness, safety, cohort-study


1. Stegmayer B. Basal cell carcinoma in Sweden 2004-2008. Socialstyrelsen; 2009 2009-12-12. 2. Aszterbaum M, Beech J, Epstein EH, Jr. Ultraviolet radiation mutagenesis of hedgehog pathway genes in basal cell carcinomas. The journal of investigative dermatology Symposium proceedings, 1999; 4: 41-45. doi:10.1038/sj.jidsp.5640179 3. Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nature reviews Cancer, 2008; 8: 743-754. doi:10.1038/nrc2503 4. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. The New England journal of medicine, 2009; 361: 1164-1172. doi:10.1056/NEJMoa0905360 5. Caro I, Low JA. The role of the hedgehog signaling pathway in the development of basal cell carcinoma and opportunities for treatment. Clinical cancer research : an official journal of the American Association for Cancer Research, 2010; 16: 3335-3339. doi:10.1158/1078-0432.ccr-09-2570 6. Lindelöf B, Lapins J, Dal H. Shift in Occupational Risk for Basal Cell Carcinoma from Outdoor to Indoor Workers: A Large Population-based Case-control Register Study from Sweden. Acta dermato-venereologica, 2017; 97: 830-833. doi:10.2340/00015555-2660 7. Gorlin RJ. Nevoid basal-cell carcinoma syndrome. Medicine, 1987; 66: 98-113. doi:10.1097/00005792-198703000-00002 8. Tang JY, Mackay-Wiggan JM, Aszterbaum M, et al. Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndrome. The New England journal of medicine, 2012; 366: 2180-2188. doi:10.1056/NEJMoa1113538 9. Spiker AM, Troxell T, Ramsey ML. Gorlin Syndrome. StatPearls. Treasure Island (FL): StatPearls Publishing Copyright © 2021, StatPearls Publishing LLC., 2021. 10. Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. The New England journal of medicine, 2012; 366: 2171-2179. doi:10.1056/NEJMoa1113713 11. Sekulic A, Migden MR, Basset-Seguin N, et al. Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study. BMC cancer, 2017; 17: 332. doi:10.1186/s12885-017-3286-5 12. Basset-Séguin N, Hauschild A, Kunstfeld R, et al. Vismodegib in patients with advanced basal cell carcinoma: Primary analysis of STEVIE, an international, open-label trial. European journal of cancer (Oxford, England : 1990), 2017; 86: 334-348. doi:10.1016/j.ejca.2017.08.022 13. Dréno B, Kunstfeld R, Hauschild A, et al. Two intermittent vismodegib dosing regimens in patients with multiple basal-cell carcinomas (MIKIE): a randomised, regimen-controlled, double-blind, phase 2 trial. The Lancet Oncology, 2017; 18: 404-412. doi:10.1016/s1470-2045(17)30072-4 14. Woltsche N, Pichler N, Wolf I, Di Meo N, Zalaudek I. Managing adverse effects by dose reduction during routine treatment of locally advanced basal cell carcinoma with the hedgehog inhibitor vismodegib: a single centre experience. Journal of the European Academy of Dermatology and Venereology : JEADV, 2019; 33: e144-e145. doi:10.1111/jdv.15367 15. Routt E, Ratner D. Outcomes for Basal Cell Carcinoma Treated With Vismodegib Extended Alternate Day Dosing. Dermatologic surgery : official publication for American Society for Dermatologic Surgery [et al], 2020; 46: 1109-1112. doi:10.1097/dss.0000000000001985 16. Wong C, Poblete-Lopez C, Vidimos A. Comparison of daily dosing versus Monday through Friday dosing of vismodegib for locally advanced basal cell carcinoma and basal cell nevus syndrome: A retrospective case series. Journal of the American Academy of Dermatology, 2020; 82: 1539-1542. doi:10.1016/j.jaad.2020.02.050 17. Apalla Z, Spyridis I, Kyrgidis A, et al. Vismodegib in real-life clinical settings: A multicenter, longitudinal cohort providing long-term data on efficacy and safety. Journal of the American Academy of Dermatology, 2021; 85: 1589-1592. doi:10.1016/j.jaad.2020.11.036 18. Gutzmer R, Schulze HJ, Hauschild A, et al. Effectiveness, safety and utilization of vismodegib in locally advanced basal cell carcinoma under real-world conditions in Germany - The non-interventional study NIELS. Journal of the European Academy of Dermatology and Venereology : JEADV, 2021; 35: 1678-1685. doi:10.1111/jdv.17332 19. Kaatz M, Mohr P, Livingstone E, et al. Effectiveness, Safety and Utilization of Vismodegib for Locally Advanced Basal Cell Carcinoma Under Real-world Conditions: Non-interventional Cohort Study JONAS. Acta dermato-venereologica, 2022; 102: adv00695. doi:10.2340/actadv.v102.293 20. Dummer R, Basset-Seguin N, Hansson J, et al. Impact of treatment breaks on vismodegib patient outcomes: Exploratory analysis of the STEVIE study. Journal of Clinical Oncology, 2015; 33: 9024-9024. doi:10.1200/jco.2015.33.15_suppl.9024 21. Frampton JE, Basset-Séguin N. Vismodegib: A Review in Advanced Basal Cell Carcinoma. Drugs, 2018; 78: 1145-1156. doi:10.1007/s40265-018-0948-9 22. Lacouture ME, Dréno B, Ascierto PA, et al. Characterization and Management of Hedgehog Pathway Inhibitor-Related Adverse Events in Patients With Advanced Basal Cell Carcinoma. The oncologist, 2016; 21: 1218-1229. doi:10.1634/theoncologist.2016-0186 23. Peris K, Fargnoli MC, Garbe C, et al. Diagnosis and treatment of basal cell carcinoma: European consensus-based interdisciplinary guidelines. European journal of cancer (Oxford, England : 1990), 2019; 118: 10-34. doi:10.1016/j.ejca.2019.06.003

Send mail to Author

Send Cancel