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Novel therapy for transthyretin-related ocular amyloidosis: a pilot study of retinal laser photocoagulation
Takahiro Kawaji1, Yukio Ando, Ryuhei Hara
1Department of Ophthalmology and Visual Science, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan. kawag@white.plala.or.jp
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.
Insights
Panretinal laser photocoagulation can prevent amyloid deposition in the eyes of patients with transthyretin-related familial amyloidotic polyneuropathy. This safe procedure offers a new option for mitigating ocular complications.
Area of Science:
- Ophthalmology
- Neurology
- Genetics
Background:
- Ocular complications are common in transthyretin-related familial amyloidotic polyneuropathy (hATTR amyloidosis).
- These complications can progress even after liver transplantation, which halts systemic disease progression.
Observation:
- This study explored panretinal laser photocoagulation as a novel strategy to prevent ocular involvement in hATTR amyloidosis.
- The procedure targets the retinal pigment epithelium, a key site for amyloidogenic transthyretin production in the eye.
Findings:
- Panretinal laser photocoagulation successfully prevented the progression of amyloid deposition in the vitreous and on the retinal surface in two patients over three years.
- No serious adverse events were reported during the follow-up period.
Implications:
- Panretinal laser photocoagulation presents a safe and viable treatment option for managing ocular manifestations of hATTR amyloidosis.
- This intervention may help preserve vision and reduce the burden of ocular disease in affected individuals.
