William William N, Feng Lei, Ferrarotto Renata, Ginsberg Lawrence, Kies Merrill, Lippman Scott, Glisson Bonnie, Kim Edward S
Department of Thoracic/Head and Neck Medical Oncology, The University of Texas M.D. Anderson Cancer Center, Houston, Texas.
Department of Biostatistics, The University of Texas M.D. Anderson Cancer Center, Houston, Texas.
J Am Acad Dermatol. 2017 Dec;77(6):1110-1113.e2. doi: 10.1016/j.jaad.2017.07.048. Epub 2017 Sep 28.
Preclinical data demonstrate a key role for the epidermal growth factor receptor (EGFR) in the carcinogenesis of cutaneous squamous cell carcinomas (CSCCs). There are, however, limited data on the efficacy of EGFR inhibitors in incurable, recurrent, and/or metastatic CSCC.
To determine the response rate to gefitinib in patients with CSCC not amenable to curative therapy including surgery or radiation.
This was a single-arm phase II study. A total of 40 patients were treated with gefitinib, 250 mg orally daily, until disease progression or intolerable toxicities. The prespecified target response rate of interest was 20%.
The overall response rate was 16% (95% confidence interval, 0.06-0.32; 6 partial responses in 37 evaluable patients). An additional 13 patients (35%) had stable disease at 8 weeks. The median durations of response and progression-free survival were 31.4 months (95% confidence interval, 3.91-not applicable) and 3.8 months (95% confidence interval, 2.2-5.7), respectively. The side effect profile was consistent with the previous experience with gefitinib in other tumor types.
This was a single-institution, single-arm study. The prespecified target response rate was not met.
Gefitinib demonstrated modest activity in incurable CSCC, with a favorable adverse event profile.
临床前数据表明表皮生长因子受体（EGFR）在皮肤鳞状细胞癌（CSCC）的致癌过程中起关键作用。然而，关于EGFR抑制剂在无法治愈、复发和/或转移性CSCC中的疗效数据有限。
确定吉非替尼对无法接受包括手术或放疗在内的根治性治疗的CSCC患者的缓解率。
这是一项单臂II期研究。共有40例患者接受吉非替尼治疗，每日口服250毫克，直至疾病进展或出现无法耐受的毒性。预先设定的目标缓解率为20%。
总缓解率为16%（95%置信区间，0.06 - 0.32；37例可评估患者中有6例部分缓解）。另外13例患者（35%）在8周时病情稳定。缓解持续时间和无进展生存期的中位数分别为31.4个月（95%置信区间，3.91 - 不适用）和3.8个月（95%置信区间，2.2 - 5.7）。副作用情况与吉非替尼在其他肿瘤类型中的既往经验一致。
这是一项单机构、单臂研究。未达到预先设定的目标缓解率。
吉非替尼在无法治愈的CSCC中显示出适度活性，不良事件情况良好。
