AP-HP Department of Dermatology, Université de Paris, DMU ICARE, INSERM U976 HIPI, Team 1 Hôpital Saint-Louis, FR-75010 Paris, France.
Acta Derm Venereol. 2022 May 23;102:adv00740. doi: 10.2340/actadv.v102.1995.
Sonidegib, a hedgehog pathway inhibitor, is indicated for treatment of locally advanced basal cell carcinoma, based on the results of the BOLT study. However, to date, no real-world study of sonidegib has been reported. An observational, retrospective, single-centre study (PaSoS study) was conducted. The primary objective was to evaluate the efficacy of sonidegib for treatment of locally advanced basal cell carcinoma in a real-world setting. Secondary objectives included modalities of use, tolerability, tumour evolution, and management after discontinuation. A total of 21 patients treated with sonidegib were included from March 2018 to January 2021. The median follow-up was 18.7 months and median exposure 7.0 months. Objective response (OR) rate was 81.0% (n = 17) including 6 (29%) patients with a complete response (CR). Disease control rate was 100%. First tumour response was rapid, with a median time of 2.3 months. Nine (43%) patients underwent surgery after sonidegib discontinuation, and no relapse was observed. All the patients experienced at least 1 adverse event (AE). Muscle spasms were the most frequent AE (n = 14; 67%), followed by dysgeusia (n = 8; 38%) and alopecia (n = 12; 57%). The efficacy and safety profile of sonidegib in this first-to-date real-life trial are consistent with prior results. Overall, real-world evidence corroborated sonidegib efficacy and tolerability as a first-line treatment for locally advanced basal cell carcinoma.
索尼德吉是一种 Hedgehog 通路抑制剂，基于 BOLT 研究的结果，被批准用于治疗局部晚期基底细胞癌。然而，迄今为止，尚无关于索尼德吉的真实世界研究报告。本研究开展了一项观察性、回顾性、单中心研究（PaSoS 研究）。主要目的是评估索尼德吉治疗局部晚期基底细胞癌的真实世界疗效。次要目的包括使用方式、耐受性、肿瘤演变和停药后的管理。共纳入 2018 年 3 月至 2021 年 1 月期间接受索尼德吉治疗的 21 例患者。中位随访时间为 18.7 个月，中位暴露时间为 7.0 个月。客观缓解率（ORR）为 81.0%（n=17），包括 6 例（29%）完全缓解（CR）患者。疾病控制率为 100%。首次肿瘤反应迅速，中位时间为 2.3 个月。9 例（43%）患者在索尼德吉停药后接受了手术，未观察到复发。所有患者均至少发生 1 次不良事件（AE）。肌肉痉挛是最常见的 AE（n=14；67%），其次是味觉障碍（n=8；38%）和脱发（n=12；57%）。在这项首次真实世界试验中，索尼德吉的疗效和安全性与既往结果一致。总体而言，真实世界证据证实了索尼德吉作为局部晚期基底细胞癌一线治疗的疗效和耐受性。
