Burness Celeste B, Scott Lesley J
Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand.
Target Oncol. 2016 Apr;11(2):239-46. doi: 10.1007/s11523-016-0418-9.
Sonidegib (Odomzo®), an oral smoothened (SMO) antagonist, is indicated for the treatment of adults with locally advanced basal cell carcinoma (laBCC) who are not candidates for surgery or radiation therapy, or adults with recurrent laBCC following surgery or radiation therapy. In the multicentre BOLT trial, the primary endpoint (i.e., an objective tumour response rate point estimate of ≥30 % and a 95 % confidence interval lower bound of >20 % in patients with fully assessable laBCC and all patients with metastatic BCC) was met at the primary analysis cut-off date (median follow-up 13.9 months) in the sonidegib 200 mg (36 % [95 % CI 24-50]) and 800 mg (34 % [95 % CI 25-43]) once-daily groups. Sonidegib 200 mg once daily (recommended dosage) had a better benefit-risk profile than the 800 mg dosage. Central review of the patients with laBCC in this population showed that 43 % achieved an objective response with sonidegib 200 mg once daily at the primary analysis date. Clinically meaningful responses were sustained in the sonidegib 200 mg group, based on an 18-month analysis. The majority of treatment-emergent adverse events were of mild to moderate severity and manageable with dosage adjustments, concomitant medications and/or non-drug therapies (e.g., adequate hydration). The acceptable benefit-risk profile of sonidegib, along with a paucity of treatment options and the seriousness of the condition, makes sonidegib an emerging option for the treatment of adults with laBCC that has recurred following surgery or radiation therapy, or in those who are not candidates for surgery or radiation therapy.
索尼德吉（Odomzo®）是一种口服的 smoothened（SMO）拮抗剂，适用于治疗不适合手术或放射治疗的局部晚期基底细胞癌（laBCC）成人患者，或手术或放射治疗后复发的 laBCC 成人患者。在多中心 BOLT 试验中，在主要分析截止日期（中位随访 13.9 个月）时，索尼德吉 200mg（36%[95%CI 24-50]）和 800mg（34%[95%CI 25-43]）每日一次组达到了主要终点（即，在完全可评估的 laBCC 患者和所有转移性 BCC 患者中，客观肿瘤缓解率点估计≥30%且 95%置信区间下限>20%）。索尼德吉 200mg 每日一次（推荐剂量）的获益-风险比优于 800mg 剂量。对该人群中 laBCC 患者的中心评估显示，在主要分析日期，每日一次服用 200mg 索尼德吉的患者中有 43%达到了客观缓解。根据 18 个月的分析，索尼德吉 200mg 组的临床有意义缓解得以持续。大多数治疗中出现的不良事件为轻度至中度，可通过调整剂量、联合用药和/或非药物治疗（如充分补水）进行管理。索尼德吉可接受的获益-风险比，加上治疗选择匮乏以及病情的严重性，使其成为治疗手术或放射治疗后复发的 laBCC 成人患者或不适合手术或放射治疗患者中的一种新兴选择。
