Novel therapy for transthyretin-related ocular amyloidosis: a pilot study of retinal laser photocoagulation.

OBJECTIVE

The occurrence of ocular complications associated with transthyretin-related familial amyloidotic polyneuropathy increases with time, even after liver transplantation, which leads to a halt in the progression of systemic neurologic complications. This study investigated a new strategy to prevent ocular involvements.

DESIGN

Two interventional case reports.

PARTICIPANTS

Two patients with familial amyloidotic polyneuropathy who had progressive ocular manifestations.

METHODS

We used panretinal laser photocoagulation, which damages the retinal pigment epithelium, the main location for synthesis of amyloidogenic transthyretin in ocular tissues, to treat 1 eye of each patient. After laser photocoagulation, we performed general ophthalmic examinations every 3 months for 3 years.

MAIN OUTCOME MEASURES

Fundus photography, visual acuity, and intraocular pressure.

RESULTS

Panretinal laser photocoagulation clearly prevented progression of amyloid deposition in the vitreous and on the retinal surface in both cases during 3 years of follow-up. No serious complications occurred.

CONCLUSIONS

Panretinal laser photocoagulation is a safe and well-known procedure that offers a new treatment option to mitigate ocular manifestations in patients with transthyretin-related familial amyloidotic polyneuropathy.