Risk Factors and Disease Course for Blood-Brain Barrier Disruption-Associated Maculopathy

Area of Science:

• Ophthalmology
• Neuro-oncology
• Medical Imaging

Background:

• Blood-brain barrier disruption (BBBD) is a therapeutic strategy for malignant central nervous system (CNS) tumors.
• BBBD has been associated with pigmentary maculopathy, but the underlying mechanisms and clinical implications remain poorly understood.

Purpose of the Study:

• To determine the incidence of BBBD-associated maculopathy.
• To identify risk factors contributing to its development.
• To assess the potential for visual progression after BBBD therapy completion.

Main Methods:

• Retrospective case series analysis of patients treated with osmotic BBBD for CNS tumors between February 2006 and December 2019.
• Ophthalmic evaluations were conducted post-treatment to assess maculopathy and visual acuity.
• Statistical analysis to identify associations between BBBD sessions, patient demographics, chemotherapy, and maculopathy development.

Main Results:

• Of 65 evaluable patients, 32 (49.2%) developed pigmentary maculopathy.
• The number of BBBD treatment sessions was significantly associated with maculopathy development (OR, 1.30; 95% CI, 1.12-1.50; P=.001).
• Progressive geographic atrophy and choroidal neovascularization were observed in a subset of patients after therapy completion.

Conclusions:

• BBBD-associated maculopathy is common and appears to be dose-dependent on the number of BBBD treatment sessions.
• Ocular changes, including geographic atrophy, can progress years after cessation of systemic therapy.
• Findings underscore the need for patient education and regular ophthalmic monitoring in patients undergoing BBBD therapy.