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Updated: Aug 28, 2025

Retinal Pigment Epithelium Transplantation in a Non-human Primate Model for Degenerative Retinal Diseases
Published on: June 14, 2021
IRREVERSIBLE RETINAL PIGMENT EPITHELIUM TOXICITY ASSOCIATED WITH FIBROBLAST GROWTH FACTOR RECEPTOR INHIBITOR THERAPY
1Department of Ophthalmology, Mayo Clinic, Scottsdale, Arizona.
Purpose:
Erdafitinib is a fibroblast growth factor receptor inhibitor indicated for the treatment of cancer. A case of fibroblast growth factor receptor inhibitor-associated retinopathy that resulted in significant visual symptoms and chronic subretinal abnormalities is reported.
Methods:
A 73-year-old man with a history of relapsed multiple myeloma was treated with erdafitinib. Soon after his fourth treatment cycle, he developed blurred vision in both eyes. Therapy with erdafitinib was subsequently discontinued.
Results:
Funduscopic examination and optical coherence tomography of both eyes revealed multifocal subretinal fluid in the macula of both eyes. Eleven weeks after cessation of the erdafitinib, the visual acuity improved but the patient reported bilateral annular scotomas. Evaluation was notable for resolution of the subretinal fluid with the development irregular subfoveal thickening in both eyes. The patient's symptoms improved, but the subfoveal abnormalities were persistent at 14 months follow-up.
Conclusion:
Erdafitinib may be associated with permanent retinal pigment epithelium toxicity.
Insights
Erdafitinib treatment for cancer can cause retinopathy, leading to vision loss and persistent retinal abnormalities. This case highlights potential permanent retinal pigment epithelium toxicity from fibroblast growth factor receptor inhibitors.
Area of Science:
- Ophthalmology
- Oncology
- Pharmacology
Background:
- Erdafitinib is a targeted therapy for cancer.
- Fibroblast growth factor receptor (FGFR) inhibitors are used in cancer treatment.
- Ocular side effects can occur with FGFR inhibitors.
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