Mayo Clinic, Scottsdale, Arizona.
University of Texas MD Anderson Cancer Center, Houston, Texas.
J Am Acad Dermatol. 2015 Jun;72(6):1021-6.e8. doi: 10.1016/j.jaad.2015.03.021.
Primary analysis from the pivotal ERIVANCE BCC study resulted in approval of vismodegib, a Hedgehog pathway inhibitor indicated for treatment of adults with metastatic or locally advanced basal cell carcinoma (BCC) that has recurred after surgery or for patients who are not candidates for surgery or radiation.
An efficacy and safety analysis was conducted 12 months after primary analysis.
This was a multinational, multicenter, nonrandomized, 2-cohort study in patients with measurable and histologically confirmed locally advanced or metastatic BCC taking oral vismodegib (150 mg/d). Primary outcome measure was objective response rate (complete and partial responses) assessed by independent review facility.
After 12 months of additional follow-up, median duration of exposure to vismodegib was 12.9 months. Objective response rate increased from 30.3% to 33.3% in patients with metastatic disease, and from 42.9% to 47.6% in patients with the locally advanced form. Median duration of response in patients with locally advanced BCC increased from 7.6 to 9.5 months. No new safety signals emerged with extended treatment duration.
Limitations include low prevalence of advanced BCC and challenges of designing a study with heterogenous manifestations.
The 12-month update of the study confirms the efficacy and safety of vismodegib in management of advanced BCC.
关键性 ERIVANCE BCC 研究的主要分析导致了 vismodegib 的批准，Hedgehog 通路抑制剂用于治疗手术后复发或不适合手术或放疗的转移性或局部晚期基底细胞癌（BCC）的成人。
对主要分析后 12 个月进行了疗效和安全性分析。
这是一项在可测量和组织学确认的局部晚期或转移性 BCC 患者中进行的多中心、多国、非随机、2 队列研究，患者接受口服 vismodegib（150 mg/d）治疗。主要观察指标为独立审查机构评估的客观缓解率（完全和部分缓解）。
在额外随访 12 个月后，vismodegib 的中位暴露时间为 12.9 个月。转移性疾病患者的客观缓解率从 30.3%增加到 33.3%，局部晚期疾病患者从 42.9%增加到 47.6%。局部晚期 BCC 患者的中位缓解持续时间从 7.6 个月增加到 9.5 个月。随着治疗时间的延长，没有出现新的安全信号。
局限性包括晚期 BCC 的患病率低和设计具有异质性表现的研究的挑战。
该研究的 12 个月更新证实了 vismodegib 在管理晚期 BCC 方面的疗效和安全性。
