Purpose: To report the successful use of topical cysteamine formulated from the oral preparation in the treatment of severe photophobia from corneal crystal deposition in cystinosis.
Design: Interventional case report.
Methods: Retrospective chart review.
Results: An 8-year-old boy with nephropathic cystinosis was experiencing debilitating and worsening photophobia from corneal crystal deposition. Because no parenteral cysteamine was available nationally, the oral capsule was used to formulate an ophthalmic preparation for compassionate use. After 8 months of topical application, the patient has marked improvement of his corneal disease, both subjectively and objectively.
Conclusions: In situations of need, there is a role for the formulation of ophthalmic cysteamine from its oral preparation.