Low Occurrence of Ocular Adverse Events after CAR-T Cell Therapy.

Introduction: Chimeric antigen receptor (CAR)-T cell therapies have demonstrated remarkable therapeutic efficacy in leukemias and lymphomas that were previously considered incurable. However, concerns persist over potential risks related to toxicities, including those secondary to activation of the patient's immune system.

Methods: To investigate ocular adverse effects (o-AEs) associated with CAR-T cell therapy, a retrospective cohort study was designed in which data were obtained from the TriNetX aggregated electronic health records database through August 2024, with data analysis performed in August 2024. Billing codes were used to identify patients receiving autologous CAR-T therapy approved by the US Food and Drug Administration (FDA) for the treatment of a hematological malignancy: tisagenlecleucel, brexucabtagene autoleucel, lisocabtagene maraleucel, ciltacabtagene autoleucel, idecabtagene vicleucel, or axicabtagene ciloleucel.

Results: In a cohort of 684 patients on CAR-T therapy with at least 6 months of follow-up, the most prevalent o-AEs were related to vision changes (1.9%), which included vitreous opacities, visual disturbances, diplopia, and visual discomfort; inflammation (1.8%), which included optic neuritis, conjunctivitis, optic papillitis, chorioretinal inflammation, iridocyclitis, zoster ocular disease; and dry eyes (1.6%), which included dry eye syndrome, keratitis, and ocular manifestations of Vitamin A deficiency.

Conclusion: In the period of 6 months following CAR-T therapy infusion, o-AEs were rare in patients receiving CAR-T cell therapy, indicating that patients without existing eye conditions do not need routine prescreening or directed follow-up after treatment, unless symptomatic. Ongoing monitoring and reporting of ocular adverse events will be important given the durable effects of CAR-T therapy in the treatment of hematologic cancers as well as increasing indications for CAR-T therapy in malignant and nonmalignant disease.