Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, Ontario, Canada.
Division of Oculofacial Plastic and Orbital Surgery, California Pacific Medical Center, San Francisco, California.
JAMA Ophthalmol. 2013 Dec;131(12):1591-4. doi: 10.1001/jamaophthalmol.2013.5018.
Basal cell carcinoma (BCC) represents 90% of malignant eyelid tumors and is locally invasive and destructive, if left untreated.
To assess the feasibility of using vismodegib for periocular and orbital BCC based on its efficacy and tolerability.
DESIGN, SETTING, AND PARTICIPANTS: In this prospective observational case series, consecutive patients with periocular or orbital BCC who met criteria for treatment with vismodegib were recruited prospectively during an 8-month period from February through September 2012 from 2 academic hospitals. Seven patients received oral vismodegib, 150 mg daily, until maximum clinical response was achieved, the tumor progressed, or the patient could no longer tolerate adverse effects. Clinical response and adverse effects related to treatment were recorded. The primary endpoint was reduction in lesion size, measured as percentage change in the externally visible dimension.
Oral vismodegib.
All 7 patients had locally advanced, biopsy-proven, infiltrative BCC that was not amenable to surgical resection or radiation. No patients had metastatic disease at presentation. The mean patient age was 71 years (range, 43-100 years), and 4 patients (57%) had secondary orbital involvement. The mean lesion size was 3.4 cm (range, 1.0-6.0 cm), and all 7 cases (100%) represented recurrent tumors excised previously with controlled margins by frozen section or Mohs micrographic surgery. The mean treatment duration was 11 weeks (range, 4-16 weeks), and the mean duration of follow-up was 7.3 months (range, 5-10 months). Two patients (29%) demonstrated complete clinical regression, 2 (29%) demonstrated greater than 80% partial clinical regression, 2 (29%) demonstrated less than 35% partial clinical regression, and 1 (14%) progressed. Adverse reactions occurred in 6 patients (86%) and included alopecia (29%), dysgeusia (29%), muscle cramps (29%), and anorexia (14%). Two patients (29%) developed new squamous cell carcinomas (well-differentiated, keratoacanthoma type) at uninvolved sites including the eyebrow and forearm.
Vismodegib seems to be well-tolerated and effective for treating periocular and orbital BCC in about half of all cases. Patients receiving treatment should be monitored for new squamous cell carcinomas at uninvolved sites.
基底细胞癌（BCC）占恶性眼睑肿瘤的 90%，如果不治疗，它具有局部侵袭性和破坏性。
评估维莫德吉治疗眼周和眼眶 BCC 的疗效和耐受性。
设计、地点和参与者：这是一项前瞻性观察性病例系列研究，连续招募了 2012 年 2 月至 9 月在 2 所学术医院接受治疗的符合维莫德吉治疗标准的眼周或眼眶 BCC 患者。7 例患者接受了口服维莫德吉，每天 150mg，直至达到最大临床反应、肿瘤进展或患者无法耐受不良反应为止。记录临床反应和与治疗相关的不良反应。主要终点是病变大小的减少，以外部可见尺寸的百分比变化来衡量。
口服维莫德吉。
所有 7 例患者均患有局部晚期、经活检证实、浸润性 BCC，不适合手术切除或放疗。无患者在就诊时发生转移性疾病。患者的平均年龄为 71 岁（范围为 43-100 岁），4 例（57%）有继发性眼眶受累。平均病变大小为 3.4cm（范围为 1.0-6.0cm），所有 7 例（100%）均为先前经冷冻切片或 Mohs 显微外科手术切除的复发性肿瘤，切缘控制良好。平均治疗持续时间为 11 周（范围为 4-16 周），平均随访时间为 7.3 个月（范围为 5-10 个月）。2 例（29%）患者完全临床缓解，2 例（29%）患者部分临床缓解大于 80%，2 例（29%）患者部分临床缓解小于 35%，1 例（14%）患者进展。6 例（86%）患者出现不良反应，包括脱发（29%）、味觉障碍（29%）、肌肉痉挛（29%）和食欲不振（14%）。2 例（29%）患者在未受累部位（包括眉毛和前臂）发生新的鳞状细胞癌（分化良好，角化棘皮瘤型）。
维莫德吉治疗眼周和眼眶 BCC 的疗效似乎较好，约半数患者有效。接受治疗的患者应在未受累部位监测新的鳞状细胞癌。
