Immune Checkpoint Inhibitors in the Treatment of Ocular Surface Cancers: A Review

Wendy J Li1, Wisam Najdawi1, Omar Badla1

  • 1Department of Ophthalmology, Bascom Palmer Eye Institute, Miami, FL, USA.

Seminars in Ophthalmology
|February 9, 2025
PubMed
Abstract

Insights

Immune checkpoint inhibitors (ICIs) show promise for ocular surface tumors like conjunctival melanoma and OSSN, offering new hope where traditional treatments fail. Further research is needed to overcome challenges and optimize their use in these rare cancers.

Area of Science:

  • Oncology
  • Immunology
  • Ophthalmology

Background:

  • Immune checkpoint inhibitors (ICIs) target immune pathways (PD-1, PD-L1, CTLA-4, LAG-3) to enhance anti-cancer immunity.
  • Emerging research explores ICI efficacy in ocular surface tumors, including conjunctival melanoma (CM) and ocular surface squamous neoplasia (OSSN).
  • Potential roles for ICIs are also suggested in sebaceous gland carcinoma (SeC), conjunctival lymphoma, and Kaposi sarcoma of the eye.

Purpose of the Study:

  • To review the role of immune checkpoint inhibitors (ICIs) in treating ocular surface tumors.
  • To analyze the mechanisms of action, clinical outcomes, and therapeutic potential of ICIs in this context.

Main Methods:

  • Conducted a literature review of studies published between January 2014 and October 2024.
  • Searched the PubMed database for relevant original research, clinical trials, case reports, and reviews.

Main Results:

  • Pembrolizumab and nivolumab demonstrated significant efficacy in conjunctival melanoma (CM), inducing tumor regression and stabilization in advanced cases.
  • Immune checkpoint inhibitors (ICIs) showed effectiveness in ocular surface squamous neoplasia (OSSN), particularly those with high tumor mutational burden, leading to partial or complete resolution.
  • Limited but suggestive data indicate potential for ICIs in refractory or advanced sebaceous gland carcinoma (SeC) and conjunctival lymphoma.

Conclusions:

  • Immune checkpoint inhibitors (ICIs) offer transformative potential for ocular surface malignancies, especially in treatment-resistant or advanced disease.
  • Challenges include variable response rates, immune-related adverse events, and the need for predictive biomarkers.
  • Prospective studies are essential to refine ICI application, optimize strategies, and improve outcomes for these rare eye cancers.

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