Vismodegib for Preservation of Visual Function in Patients with Advanced Periocular Basal Cell Carcinoma: The VISORB Trial
| Autor: | Alon Kahana, Shelby P. Unsworth, Victor M. Elner, Alison B. Durham, Denise S. Kim, Chris Andrews, Paul L. Swiecicki, Christopher K. Bichakjian, May P. Chan, Francis P. Worden, Hakan Demirci, Scott C. Bresler, Christine C. Nelson, Shannon S. Joseph |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
Cancer Research
medicine.medical_specialty Skin Neoplasms Pyridines Patched PTCH Visual function Vismodegib Context (language use) Physical examination Antineoplastic Agents Lacrimal apparatus Orbital Epiphora medicine Clinical endpoint Humans Basal cell carcinoma Anilides Hedgehog Proteins Prospective Studies Cancer Smoothened Tumor medicine.diagnostic_test business.industry SMO medicine.disease Clinical trial medicine.anatomical_structure Treatment Outcome Lacrimal Oncology Carcinoma Basal Cell Basal cell Radiology Melanoma and Cutaneous Malignancies Skin cancer business Orbit Hedgehog medicine.drug |
| Zdroj: | The Oncologist |
| ISSN: | 1549-490X |
| Popis: | Background Basal cell carcinoma (BCC) is a common skin cancer often curable by excision; however, for patients with BCC around the eye, excision places visual organs and function at risk. In this article, we test the hypothesis that use of the hedgehog inhibitor vismodegib will improve vision‐related outcomes in patients with orbital and extensive periocular BCC (opBCC). Materials and Methods In this open‐label, nonrandomized phase IV trial, we enrolled patients with globe‐ and lacrimal drainage system–threatening opBCC. To assess visual function in the context of invasive periorbital and lacrimal disease, we used a novel Visual Assessment Weighted Score (VAWS) in addition to standard ophthalmic exams. Primary endpoint was VAWS with a score of 21/50 (or greater) considered successful, signifying globe preservation. Tumor response was evaluated using RECIST v1.1. Surgical specimens were examined histologically by dermatopathologists. Results In 34 patients with opBCC, mean VAWS was 44/50 at baseline, 46/50 at 3 months, and 47/50 at 12 months or postsurgery. In total, 100% of patients maintained successful VAWS outcome at study endpoint. Compared with baseline, 3% (95% confidence interval [CI], 0.1–15.3) experienced major score decline (5+ points), 14.7% (95% CI, 5 to 31.1) experienced a minor decline (2–4 points), and 79.4% experienced a stable or improved score (95% CI, 62.1–91.3). A total of 56% (19) of patients demonstrated complete tumor regression by physical examination, and 47% (16) had complete regression by MRI/CT. A total of 79.4% (27) of patients underwent surgery, of which 67% (18) had no histologic evidence of disease, 22% (6) had residual disease with clear margins, and 11% (3) had residual disease extending to margins. Conclusion Vismodegib treatment, primary or neoadjuvant, preserves globe and visual function in patients with opBCC. Clinical trail identification number.NCT02436408. Implications for Practice Use of the antihedgehog inhibitor vismodegib resulted in preservation of end‐organ function, specifically with regard to preservation of the eye and lacrimal apparatus when treating extensive periocular basal cell carcinoma. Vismodegib as a neoadjuvant also maximized clinical benefit while minimizing toxic side effects. This is the first prospective clinical trial to demonstrate efficacy of neoadjuvant antihedgehog therapy for locally advanced periocular basal cell carcinoma, and the first such trial to demonstrate end‐organ preservation. This article reports the results of a prospective clinical trial of vismodegib for patients with basal cell carcinoma occurring in the orbital and periocular regions to assess whether such treatment helps to preserve visual organs and function. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|
Plný text