BACKGROUND Vismodegib demonstrated 60% response rates in the ERIVANCE trial. Basal cell carcinoma has various histopathologies. Their effect on response is unclear. OBJECTIVE The purpose of this study was to determine whether basal cell carcinoma histopathology affected vismodegib response. METHODS This phase 2b, single-center, prospective case series study compared the efficacy of vismodegib in infiltrative, nodular, and superficial basal cell carcinomas treated for 12 or 24 weeks in 27 patients. Patients had 1 target lesion and up to 3 nontarget lesions. RESULTS Twenty-seven patients were enrolled, with 65 tumors (27 target lesions/38 nontarget lesions). At 24 weeks, most basal cell carcinomas achieved histologic clearance, with positive biopsy results in 10.5% of target lesions, 30.4% of nontarget lesions, and 21.4% overall. No statistical differences were observed between histopathologic subtypes. One hundred percent of patients experienced an adverse event, 94% grade 1 or 2. The most common adverse events were dysgeusia/loss of taste (86%), muscle spasms (82%), and alopecia (71%). Clinically progressive disease during treatment was low (1.5%). Two patients had recurrence within 1 year of treatment. LIMITATIONS Limitations included sample size of basal cell carcinoma histopathologic subtypes, sampling punch biopsies, and short follow-up. CONCLUSIONS Basal cell histopathologic subtype did not significantly affect response to vismodegib. Each subtype was observed to completely respond at 12 weeks of therapy, 24 weeks, or both.

中文翻译：

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开放标签的各种组织学亚型（浸润性，结节性和浅表性）高危或局部晚期基底细胞癌的口服vismodegib短期疗效（12-24周）的疗效，肿瘤反应率和安全性，前瞻性病例系列临床试验。

背景技术Vismodegib在ERIVANCE试验中显示出60％的缓解率。基底细胞癌具有多种组织病理学。它们对反应的影响尚不清楚。目的本研究的目的是确定基底细胞癌的组织病理学是否影响vismodegib反应。方法该2b期，单中心，前瞻性病例系列研究比较了vismodegib在27例治疗12或24周的浸润性，结节性和浅表性基底细胞癌中的疗效。患者有1个目标病变，最多3个非目标病变。结果招募了27例患者，其中65例肿瘤（27个靶病变/ 38个非靶病变）出现。在第24周时，大多数基底细胞癌达到了组织学清除率，活检阳性结果为目标病灶的10.5％，非目标病灶的30.4％和总体21.4％。在组织病理亚型之间未观察到统计学差异。百分之一百的患者经历了不良事件，94％的是1级或2级。最常见的不良事件是味觉障碍/味觉减退（86％），肌肉痉挛（82％）和脱发（71％）。治疗期间临床进行性疾病的发生率很低（1.5％）。两名患者在治疗1年内复发。局限性局限性包括基底细胞癌组织病理学亚型的样本量，取样打孔活组织检查和短期随访。结论基底细胞组织病理学亚型并未显着影响对vismodegib的反应。观察到每种亚型在治疗12周，24周或两者同时完全反应。最常见的不良事件是味觉障碍/味觉减退（86％），肌肉痉挛（82％）和脱发（71％）。治疗期间临床进行性疾病的发生率很低（1.5％）。两名患者在治疗1年内复发。局限性局限性包括基底细胞癌组织病理学亚型的样本量，取样打孔活组织检查和短期随访。结论基底细胞组织病理学亚型并未显着影响对vismodegib的反应。观察到每种亚型在治疗12周，24周或两者同时完全反应。最常见的不良事件是味觉障碍/味觉减退（86％），肌肉痉挛（82％）和脱发（71％）。治疗期间临床进行性疾病的发生率很低（1.5％）。两名患者在治疗1年内复发。局限性局限性包括基底细胞癌组织病理学亚型的样本量，取样打孔活组织检查和短期随访。结论基底细胞组织病理学亚型并未显着影响对vismodegib的反应。观察到每种亚型在治疗12周，24周或两者同时完全反应。局限性局限性包括基底细胞癌组织病理学亚型的样本量，取样打孔活组织检查和短期随访。结论基底细胞组织病理学亚型并未显着影响对vismodegib的反应。观察到每种亚型在治疗12周，24周或两者同时完全反应。局限性包括基底细胞癌组织病理学亚型的样本量，打孔活检样本和短期随访。结论基底细胞组织病理学亚型并未显着影响对vismodegib的反应。观察到每种亚型在治疗12周，24周或两者同时完全反应。