Pembrolizumab and all-trans retinoic acid combination treatment of advanced melanoma: long-term survival update

Background: Primary analysis of the phase I/II clinical trial combining pembrolizumab with all-trans retinoic acid (ATRA) for patients with metastatic melanoma showed a median progression-free survival (PFS) of 20.3 months and median overall survival (OS) not reached. Objective: Report 5-year OS and...

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Main Authors: Jessica S. W. Borgers, Theresa M. Medina, William A. Robinson, Kasey L. Couts, Elizabeth N. Katsnelson, Claire M. Muckle, Eduardo Davilla, Sapna P. Patel, Dexiang Gao, Richard P. Tobin, Martin D. McCarter
Format: Article
Language:English
Published: SAGE Publishing 2025-06-01
Series:Therapeutic Advances in Medical Oncology
Online Access:https://doi.org/10.1177/17588359251349321
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Summary:Background: Primary analysis of the phase I/II clinical trial combining pembrolizumab with all-trans retinoic acid (ATRA) for patients with metastatic melanoma showed a median progression-free survival (PFS) of 20.3 months and median overall survival (OS) not reached. Objective: Report 5-year OS and PFS rates. Design: In this single-center, single-arm trial (NCT03200847), 24 anti-PD-1 naïve patients with metastatic melanoma were enrolled between 10/2017 and 07/2020. Methods: The Kaplan–Meier method was used to estimate OS and PFS. Results: At data cutoff (May 14, 2024), all patients had completed treatment, and 58% ( n  = 14) remained alive with a median follow-up of 48 months. The updated 5-year OS rate is 54.7% (95% confidence interval (CI): 36.4–82.1), and the 5-year PFS is 36.1% (95% CI: 21.0–62.2), with 33% ( n  = 8) having an ongoing complete response. Conclusion: These results underscore the long-term clinical benefit of combining anti-PD-1 with ATRA, with survival rates comparable to anti-PD-1/combinations but with lower toxicity.
ISSN:1758-8359