Vismodegib: therapeutic application in advanced basal cell carcinoma and special groups. Experience at the Prof. A. Posadas Hospital

Authors

  • Agustina Ragalli National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina
  • Agustina María Stringa National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina
  • María Laura Mauri National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina
  • Roxana Di Gaeta National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina
  • Roberta Pedevilla National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina
  • Patricia Silvia Della Giovanna National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

DOI:

https://doi.org/10.47196/da.v31i3.2957

Keywords:

basal cell carcinoma, Hedgehog, vismodegib

Abstract

Introduction: basal cell carcinoma (BCC) accounts for 75% of non-melanoma skin cancers. Vismodegib is a Hedgehog signaling pathway inhibitor approved by the FDA (Food and Drug Administration) in 2012 for the treatment of locally advanced BCC (LA-BCC). Regarding the response to treatment, a partial or complete decrease in tumor mass is evident. Adverse effects are generally mild. The most frequent are: cramps, alopecia, dysgeusia, diarrhea, fatigue, asthenia, among others.

Objectives: demonstrate the response to treatment with vismodegib in patients with BCC-LA, metastatic and special groups, evaluated from 2/1/2018 to 29/12/202, at Posadas Hospital. Describe the variables: sex, age, number of lesions, location, size, histological subtype, time of evolution, primary/recurrence, treatment duration, response to treatment, adverse effects, medical coverage, discontinuation due to toxicity and discontinuation due to lack of access to medication.

Materials and methods: descriptive and retrospective study. A search was conducted of photographic archives and medical records of patients with BCC-LA who received vismodegib.

Results: 31 patients with BCC-LA, including 2 metastatic, 1 Gorlin syndrome and 1 chronic endemic hydroarsenicism (CEHA). Complete response was observed in 11 (35.5%) patients and partial response in 13 (41.9%). Regarding toxicity, 18 (58%) patients presented adverse effects and 7 (22.6%) suspended treatment due to intolerance.

Conclusions: Hedgehog pathway inhibitors are novel treatments for LA-CBC. They represent a great therapeutic alternative, thus avoiding mutilating surgeries. In our population, response rates like those described in the literature were obtained. Although adverse effects are common, the drug has a mild toxicity profile.

Author Biographies

Agustina Ragalli, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Resident Physician, Dermatology Service

Agustina María Stringa, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Resident Physician, Dermatology Service

María Laura Mauri, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Dermatologist, Dermatology Service

Roxana Di Gaeta, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Dermatologist, Dermatology Service

Roberta Pedevilla, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Medical Oncologist, Oncology Service

Patricia Silvia Della Giovanna, National Hospital Prof. A. Posadas, Province of Buenos Aires, Argentina

Medical Dermatologist, Head of Dermatology

References

I. Pulido-Prieto L, Esguerra-Cantillo JA, Toquica-Díaz NA, Ospina-Delgado MA. Terapia multimodal con vismodegib y radioterapia en el tratamiento del carcinoma basocelular localmente avanzado: reporte de cuatro casos. Actas Dermosifiliogr. 2023;114:264-267.

II. Pulido L, Ospina M, Jaime AO, Carreño J. Terapia intermitente con vismodegib para el CBC avanzado: experiencia en un centro de referencia oncológico en Colombia. Rev Colomb Cancerol. 2022;26:403-411.

III. Loizate-Sarrionandia I, Hernández-González R, Suárez-Hernández J, Fernández de Misa Cabrera R. Combinación de terapias en el carcinoma basocelular localmente avanzado. De la teoría a la práctica clínica. Actas Dermosifiliogr. 2024;115:508- 510.

IV. Lear JT, Corner C, Dziewulski P, Fife K, et ál. Challenges and new horizons in the management of advanced basal cell carcinoma: a UK perspective. Br J Cancer. 2014;111: 1476-1481.

V. Khoo ABS, Ali FR, Lear JT. Defining locally advanced basal cell carcinoma and integrating smoothened inhibitors into clinical practice. Curr Opin Oncol. 2016; 28: 180-184.

VI. Schmults CD, Blitzblau R, Aasi SZ, Alam M, et ál. Basal cell skin cancer. J Natl Compr Canc Netw. 2023;21:1181-203.

VII. LoRusso PM, Rudin CM, Reddy JC, Tibes R, et ál. Phase I trial of Hedgehog pathway inhibitor vismodegib (GDC-0449) in patients with refractory, locally advanced or metastatic solid tumors. Clin Cancer Res. 2011;17:2502- 2511

VIII. Kurnia-Wijaya J, Wahab S, Nurdin A, Iran Anwae A. Vismodegib y sonidegib en el carcinoma de células basales localmente avanzado y metastásico: actualización acerca de los inhibidores de la vía de Hedgehog. Actas Dermosifiliogr. 2022;113:443- 450.

IX. Ruiz-Salas V, Alegre M, López-Ferrer A, Garcés JR. Vismodegib: revisión. Actas Dermosifiliogr. 2014;105:744-751.

X. Axelson M, Liu K, Jiang X, He K, et ál. U.S. Food and Drug Administrarion approval: vismodegib for recurrent, locally advanced, or metastatic basal cell carcinoma. Clin Cancer Res. 2013;19:2289-2293.

XI. Passarelli A, Galdo G, Aieta M, Fabrizio T, et ál. Vismodegib experience in elderly patients with basal cell carcinoma: case reports and review of the literature. Int J Mol Sci. 2020;21:8596.

XII. Campastri A, Bordón MP, Cilio AM, Foenquinos R, et ál. Experiencia del uso de vismodegib para el tratamiento del carcinoma basocelular. Dermatol Argent. 2022;28: 119-124.

XIII. Bernia E, Llombart B, Serra- Guillén C, Bancalari B. Experiencia con vismodegib en carcinoma basocelular avanzado en un centro oncológico. Actas Dermosifiliogr. 2018; 109:813-820

XIV. Sekulic A, Migden MR, Basset- Seguin N, Garbe C, et ál. 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.

XV. Basset-Seguin N, Hauschild A, Grob JJ, Kunstfeld R, et ál. Vismodegib in patients with advanced basal cell carcinoma (STEVIE): a pre-planned interim analysis of an international, open-label trial. Lancet Oncol. 2015;16:729-736.

XVI. Tang JY, Mackay Wiggan JM, Aszterbaum M, Yauch RL, et ál. Inhibiting the Hedgehog pathway in patients with the basal-cell nevus syndrome. N Engl J Med. 2012;366:2180-2188.

Published

2025-12-01

Issue

Section

Original Articles