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Original Investigation
May 14, 2020

Outcomes of Vismodegib for Periocular Locally Advanced Basal Cell Carcinoma From an Open-label Trial

Author Affiliations
  • 1Department of Ophthalmology, Rabin Medical Center, Beilinson Hospital, Petach Tikva, Israel
  • 2Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
  • 3Davidoff Center for Oncology, Rabin Medical Center, Beilinson Hospital, Petach Tikva, Israel
  • 4Goldscgleger Eye Institute, Sheba Medical Center, Tel Hashomer, Israel
JAMA Ophthalmol. Published online May 14, 2020. doi:10.1001/jamaophthalmol.2020.1539
Key Points

Question  What were the outcomes of vismodegib treatment for study participants with periocular locally advanced basal cell carcinoma?

Findings  The post hoc analysis of an open-label cohort study of 244 participants revealed that 22 (9.0%) died, 70 (28.7%) achieved complete response, and 94 (38.5%) achieved partial response. Two hundred thirty-two participants (95.1%) had more than 1 adverse event, and vismodegib was discontinued owing to an adverse event in 58 (23.8%).

Meaning  These data may be helpful when considering vismodegib treatment for patients with periocular locally advanced basal cell carcinoma.

Abstract

Importance  The outcomes of vismodegib treatment in a relatively large cohort of study participants with periocular locally advanced basal cell carcinoma (POLA-BCC) may guide physicians when considering this treatment.

Objective  To report the outcomes of vismodegib treatment in patients with POLA-BCC in the Safety Events in Vismodegib (STEVIE) study.

Design, Setting, and Participants  This post hoc subgroup analysis from the STEVIE single-arm, multicenter, open-label cohort study screened all 1215 participants for ocular or periocular involvement and identified 244 participants with POLA-BCC or metastatic BCC. Data for the first STEVIE trial were collected from 167 treatment locations in 36 countries from June 30, 2011, to June 14, 2017. This post hoc analysis was performed from April 1 to August 31, 2019.

Main Outcomes and Measures  Response to treatment and adverse events.

Results  Ocular or periocular involvement was found in 244 of 1215 STEVIE participants (20.1%), who constituted the analytic sample. The median age of the study participants was 72.0 (interquartile range [IQR], 60.0-82.0]) years, and they included 143 men (58.6%). Locally advanced BCC was diagnosed in 238 of the 244 participants (97.5%) and metastatic BCC, in 6 (2.5%). The median duration of exposure to vismodegib was 40.0 (IQR, 20.0-78.0) weeks, specifically 39.7 (IQR, 19.9-76.0) weeks for POLA-BCC and 92.4 (IQR, 53.2-163.0) weeks for metastatic BCC. Sixty-nine participants (28.3%) sustained serious adverse events (alopecia, muscle spasms, dysgeusia, weight loss, decreased appetite, asthenia, ageusia, nausea, fatigue, and diarrhea). Two hundred thirty-two study participants (95.1%) sustained more than 1 adverse effect. The overall mean (SD) number of drug-related adverse effects per study participant by first adverse event, regardless of the severity, was 5.48 (3.84). Discontinuation of vismodegib treatment owing to an adverse event was recorded in 58 participants (23.8%). During the study, 22 participants (9.0%) died, 70 (28.7%) achieved complete response, and 94 (38.5%) achieved partial response.

Conclusions and Relevance  Vismodegib was well tolerated by the study participants with POLA-BCC. The safety of vismodegib treatment according to the STEVIE trial findings is consistent with that reported in previous studies. These data may be helpful when considering vismodegib for patients with POLA-BCC.

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