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Resumen: Antecedentes: El carcinoma de células basales (CBC) es el cáncer más prevalente. Una minoría de CBC tiene un comportamiento agresivo (CBCla) y puede requerir inhibidores de la vía hedgehog, como sonidegib como tratamiento. Objetivo: Describir el uso de sonidegib en un gran número de pacientes y aportar más datos sobre su perfil de eficacia y seguridad en la vida real. Métodos: Realizamos un estudio retrospectivo y multicéntrico que incluyó pacientes tratados con sonidegib. Se recogieron datos epidemiológicos, de eficacia y de seguridad. Resultados: Se incluyeron un total de 82 pacientes con una edad media de 73,9 años. Diez pacientes tenían síndrome de Gorlin. La mediana de duración del tratamiento fue de seis meses. La mediana de duración del seguimiento fue de 34,2 meses. Globalmente, 81,7% de los pacientes mostró mejoría clínica (52,4% respuesta parcial y 29,3% respuesta completa), 12,2% estabilidad clínica y 6,1% progresión de la enfermedad. No hubo diferencias estadísticamente significativas en la mejoría clínica entre la posología de sonidegib de 24 h y de 48 h. Después de seis meses de tratamiento, 48,8% de los pacientes suspendió sonidegib. El tratamiento previo con vismodegib y el CBC primario recurrente se asociaron con una peor respuesta a sonidegib. A los seis meses de tratamiento, 68,3% de los pacientes experimentó al menos un efecto adverso. Conclusión: Sonidegib muestra un perfil de eficacia y seguridad mejor de lo esperado en la práctica clínica habitual. Abstract: Background: Basal cell carcinoma (BCC) is the most prevalent cancer. A minority of BCCs have an aggressive behaviour (laBCC) and may require hedgehog pathway inhibitors such as sonidegib as its treatment. Objective: To describe the use of sonidegib in a large number of patients and provide more data on its real-life efficacy and safety profile. Methods: We conducted a retrospective and multicentric study that included patients treated with sonidegib. Epidemiological, effectiveness and safety data were collected. Results: A total of 82 patients with a mean age of 73.9 years were included. Ten patients had Gorlin syndrome. Median treatment duration was 6 months. Median follow-up duration was 34.2 months. Globally, 81.7% of the patients showed clinical improvement (52.4% partial response and 29.3% complete response), 12.2% clinical stability and 6.1% disease progression. There was no statistically significant difference in clinical improvement between the 24 h and 48 h sonidegib posology. After 6 months of treatment, 48.8% of the patients discontinued sonidegib. Prior vismodegib treatment and recurrent primary BCC were associated with a poorer response to sonidegib. At 6 months of treatment, 68.3% of the patients experienced at least one adverse effect. Conclusion: Sonidegib shows good effectiveness and acceptable safety profile in usual clinical practice.

Background: Basal cell carcinoma (BCC) is the most prevalent cancer. A minority of BCCs have an aggressive behaviour (laBCC) and may require hedgehog pathway inhibitors such as sonidegib as its treatment., Objective: To describe the use of sonidegib in a large number of patients and provide more data on its real-life efficacy and safety profile., Methods: We conducted a retrospective and multicentric study that included patients treated with sonidegib. Epidemiological, effectiveness and safety data were collected., Results: A total of 82 patients with a mean age of 73.9 years were included. Ten patients had Gorlin syndrome. Median treatment duration was 6 months. Median follow-up duration was 34.2 months. Globally, 81.7% of the patients showed clinical improvement (52.4% partial response and 29.3% complete response), 12.2% clinical stability and 6.1% disease progression. There was no statistically significant difference in clinical improvement between the 24h and 48h sonidegib posology. After 6 months of treatment, 48.8% of the patients discontinued sonidegib. Prior vismodegib treatment and recurrent primary BCC were associated with a poorer response to sonidegib. At 6 months of treatment, 68.3% of the patients experienced at least one adverse effect., Conclusion: Sonidegib shows good effectiveness and acceptable safety profile in usual clinical practice., (Copyright © 2023 AEDV. Publicado por Elsevier España, S.L.U. All rights reserved.)