Puig Susana, Serra-Guillén Carlos, Pérez-Pastor Gemma, Martínez-Domenech Álvaro, Fernández-de-Misa Cabrera Ricardo
Dermatology Department, Hospital Clínic Barcelona, University of Barcelona, Barcelona, Spain.
CIBERER, Instituto de salud Carlos III, Barcelona, Spain.
Drugs Context. 2022 May 23;11. doi: 10.7573/dic.2022-3-8. eCollection 2022.
Sonidegib is a Hedgehog signalling pathway inhibitor approved for use in patients with advanced basal cell carcinoma (BCC) not eligible for surgery or radiotherapy. This report describes clinical experience with sonidegib in two patients with locally advanced BCC (one with a tumour adjacent to the right eye and the other with a tumour associated with the left ear) and in one patient with Gorlin syndrome. Two of the patients had recurrent and intractable tumours. Treatment with sonidegib 200 mg/day led to remission in both patients with locally advanced BCC within 7 months and to a reduction in the size and number of lesions after 4 months in the patient with Gorlin syndrome. Adverse effects reported in these patients were cramps, alopecia, ageusia and weight loss, all of which were mild and consistent with the known toxicity profile for sonidegib. Sonidegib has an important role to play in the effective treatment of challenging cases of advanced BCC. In parallel, a need remains to improve management protocols for patients with advanced BCC, particularly through earlier intervention and a multidisciplinary team approach.
索尼德吉是一种获批用于治疗不适合手术或放疗的晚期基底细胞癌(BCC)患者的刺猬信号通路抑制剂。本报告描述了索尼德吉在两名局部晚期BCC患者(一名患者的肿瘤毗邻右眼,另一名患者的肿瘤与左耳相关)以及一名戈林综合征患者中的临床应用经验。其中两名患者患有复发性和难治性肿瘤。每天服用200毫克索尼德吉治疗,使得两名局部晚期BCC患者在7个月内病情缓解,戈林综合征患者在4个月后病灶大小和数量减少。这些患者报告的不良反应有痉挛、脱发、味觉丧失和体重减轻,所有这些不良反应均较轻微,且与索尼德吉已知的毒性特征相符。索尼德吉在有效治疗具有挑战性的晚期BCC病例中发挥着重要作用。与此同时,仍有必要改进晚期BCC患者的管理方案,特别是通过早期干预和多学科团队方法。