Supplementary Material for: Real-life experience with sonidegib for locally advanced basosquamous carcinoma: a case series

Introduction: Basosquamous carcinoma is an uncommon subtype of basal cell carcinoma, characterized by aggressive local growth and metastatic potential, that mainly develops on the nose, perinasal area and ears, representing 1.2%-2.7% of all head-neck keratinocyte carcinomas. Although systemic therapy with hedgehog inhibitors represents the first line medical treatment in advanced BCC, to date, no standard therapy for advanced basosquamous carcinoma has been established. Herein we reported a case series of patients affected by locally advanced basosquamous carcinomas s, who were treated with HHIs.
Case presentation: Data of five patients receiving hedgehog inhibitors for locally advanced basosquamous carcinomas were retrieved (2 women and 3 males, age range: 63 - 89 years, average age of 77 years old). Skin lesions were located on the head-neck area, in particular 4 tumors involved orbital and periorbital area and 1 tumor developed in the retro-auricular region. A clinical response was obtained in 3 out of 5 patients (2 partial responses and 1 complete response), while disease progression was observed in the remaining 2 patients, hence therapy was interrupted, switching to surgery or immunotherapy.
Conclusion: Increasing evidence suggest considering hedgehog inhibitors for large skin tumors developing in functionally and cosmetically sensitive areas, in patients with multiple comorbidities, although their use for basosquamous carcinoma require more exploration, large cohort populations and long follow-up assessment.

引言：基底鳞状细胞癌（basosquamous carcinoma）是一种少见的基底细胞癌（basal cell carcinoma，BCC）亚型，以侵袭性局部生长及转移潜能为特征，好发于鼻部、鼻周区域及耳部，约占所有头颈部角质形成细胞癌（keratinocyte carcinomas）的1.2%~2.7%。尽管Hedgehog信号通路抑制剂（hedgehog inhibitors）全身治疗是晚期基底细胞癌（BCC）的一线内科治疗方案，但截至目前，尚无针对晚期基底鳞状细胞癌的标准治疗方案。本研究报告了一组接受Hedgehog信号通路抑制剂治疗的局部晚期基底鳞状细胞癌患者的病例系列。

病例报告：本研究检索获取了5例因局部晚期基底鳞状细胞癌接受Hedgehog信号通路抑制剂治疗的患者资料（2例女性，3例男性，年龄63~89岁，平均年龄77岁）。皮肤病变均位于头颈部区域，其中4例肿瘤累及眼眶及眶周区域，1例肿瘤发生于耳后区域。5例患者中3例获得临床应答，其中2例部分缓解（partial response）、1例完全缓解（complete response）；剩余2例患者出现疾病进展，遂中断治疗，改用手术或免疫治疗。

结论：越来越多的证据表明，对于合并多种基础疾病、且肿瘤位于功能及外观敏感区域的大型皮肤肿瘤患者，可考虑采用Hedgehog信号通路抑制剂治疗；尽管将其用于基底鳞状细胞癌仍需更多探索、大样本队列研究及长期随访评估。