Bapna Monika, Ruiz Emily
Department of Dermatology, Georgetown University School of Medicine, Washington, DC, USA.
Department of Dermatology, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.
Future Oncol. 2025 Jul;21(16):1999-2005. doi: 10.1080/14796694.2025.2511568. Epub 2025 May 27.
Metastatic basal cell carcinoma (mBCC) is a rare but aggressive form of skin cancer that poses significant therapeutic challenges. Historically, systemic treatment options were limited, with hedgehog pathway inhibitors (HHIs) providing modest efficacy, but poor tolerability long term. This review explores cemiplimab, an immune checkpoint inhibitor targeting PD-1, as a novel therapeutic agent for mBCC. We examine its pharmacologic profile, clinical efficacy in clinical trials, safety and tolerability, and real-world performance. A comprehensive literature review was conducted using PubMed and clinical trial registries to assess cemiplimab's role in mBCC treatment, including its comparison to HHIs and potential in combination or neoadjuvant strategies. Cemiplimab has emerged as a transformative therapy for patients with mBCC who are intolerant to or have progressed on HHIs. Despite lower response rates compared to cutaneous squamous cell carcinoma, cemiplimab offers meaningful and durable disease control with a favorable safety profile and preservation of quality of life. Ongoing research into predictive biomarkers, neoadjuvant use, and combination regimens may further enhance its clinical utility.
转移性基底细胞癌(mBCC)是一种罕见但侵袭性强的皮肤癌,带来了重大的治疗挑战。从历史上看,全身治疗选择有限,刺猬通路抑制剂(HHIs)疗效一般,且长期耐受性差。本综述探讨了靶向程序性死亡受体1(PD-1)的免疫检查点抑制剂西米普利单抗作为mBCC的新型治疗药物。我们研究了其药理学特征、临床试验中的临床疗效、安全性和耐受性以及实际应用表现。通过使用PubMed和临床试验注册库进行了全面的文献综述,以评估西米普利单抗在mBCC治疗中的作用,包括其与HHIs的比较以及联合或新辅助治疗策略的潜力。对于不耐受HHIs或使用HHIs后病情进展的mBCC患者,西米普利单抗已成为一种变革性疗法。尽管与皮肤鳞状细胞癌相比缓解率较低,但西米普利单抗能提供有意义且持久的疾病控制,安全性良好,且能维持生活质量。对预测生物标志物、新辅助治疗用途和联合治疗方案的持续研究可能会进一步提高其临床效用。