A Matching-Adjusted Indirect Comparison of Sonidegib and Vismodegib in Advanced Basal Cell Carcinoma.

IF 1.2 Q3 DERMATOLOGY

Journal of Skin Cancer Pub Date : 2017-01-01 Epub Date: 2017-05-21 DOI:10.1155/2017/6121760

Dawn Odom, Deirdre Mladsi, Molly Purser, James A Kaye, Eirini Palaka, Alina Charter, Jo Annah Jensen, Dalila Sellami

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引用次数: 26

Abstract

Objectives: Based on single-arm trial data (BOLT), sonidegib was approved in the US and EU to treat locally advanced basal cell carcinomas (BCCs) ineligible for curative surgery or radiotherapy. Vismodegib, the other approved targeted therapy, also was assessed in a single-arm trial (ERIVANCE). We examined the comparative effectiveness of the two drugs using a matching-adjusted indirect comparison (MAIC) versus an unadjusted indirect comparison.

Methods: After comparing trials and identifying potential prognostic factors, an MAIC was conducted to adjust for differences in key patient baseline characteristics. Due to BOLT's small sample size, the number of matching variables was restricted to two. Efficacy results for sonidegib were generated so that selected baseline characteristics matched those from ERIVANCE and were compared with published ERIVANCE results.

Results: Matching variables were baseline percentages of patients receiving prior radiotherapy and surgery. After weighting, sonidegib objective response rate (ORR) and median progression-free survival (PFS) were effectively unchanged (prematched versus postmatched ORR and PFS, 56.1% versus 56.7% and 22.1 versus 22.1 months, resp.). Vismodegib's ORR and PFS were 47.6% and 9.5 months.

Conclusions: Comparative effectiveness of sonidegib versus vismodegib remains unchanged after adjusting BOLT patient-level data to match published ERIVANCE baseline percentages of patients receiving prior surgery and radiotherapy.

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Sonidegib和Vismodegib治疗晚期基底细胞癌的间接比较。

目的:基于单臂试验数据(BOLT)， sonidegib在美国和欧盟被批准用于治疗局部晚期基底细胞癌(bcc)，不适合手术或放疗治疗。另一种获批的靶向治疗药物Vismodegib也在一项单臂试验(ERIVANCE)中进行了评估。我们使用匹配调整间接比较(MAIC)和未调整间接比较来检查两种药物的比较有效性。方法:在比较试验并确定潜在预后因素后，进行MAIC以调整关键患者基线特征的差异。由于BOLT的样本量较小，匹配变量的数量限制为两个。生成sonidegib的疗效结果，以便选定的基线特征与ERIVANCE的结果相匹配，并与ERIVANCE公布的结果进行比较。结果:匹配变量为既往放疗和手术患者的基线百分比。加权后，sonidegib客观缓解率(ORR)和中位无进展生存期(PFS)有效不变(匹配前与匹配后ORR和PFS, 56.1%对56.7%，22.1个月对22.1个月，分别)。Vismodegib的ORR和PFS分别为47.6%和9.5个月。结论:在调整BOLT患者水平数据以匹配已公布的ERIVANCE基线百分比接受术前手术和放疗的患者后，sonidegib与vismodegib的比较有效性保持不变。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Skin Cancer DERMATOLOGY-

CiteScore

2.30

自引率

18.20%

发文量

审稿时长

21 weeks

期刊介绍： Journal of Skin Cancer is a peer-reviewed, Open Access journal that publishes clinical and translational research on the detection, diagnosis, prevention, and treatment of skin malignancies. The journal encourages the submission of original research articles, review articles, and clinical studies related to pathology, prognostic indicators and biomarkers, novel therapies, as well as drug sensitivity and resistance.