One-year results of a pilot study using oral 13-cis retinoic acid as a treatment for subfoveal predominantly occult choroidal neovascularization in patients with age-related macular degeneration.
OBJECTIVE
To evaluate the safety and evidence of efficacy for oral 13-cis retinoic acid as a treatment for patients with subfoveal occult choroidal neovascularization (CNV) due to age-related macular degeneration (ARMD).


METHODS
Patients with active, subfoveal occult CNV with no prior treatment of the subfoveal component were eligible for inclusion. Patients received 40 mg of 13-cis retinoic acid twice daily for 5 months, stopped treatment for 2 months, and then resumed treatment for 5 months. Patients were observed monthly with Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity (VA), clinical examination, fluorescein angiography, and laboratory testing.


RESULTS
Eleven patients, aged 64 to 88 years, were enrolled and followed for 1 year. Initial VA ranged from 55 (20/40) to 5 (20/400) ETDRS letters (median 48 letters). Mild drug-related side effects (dry skin, chapped lips) occurred in all 11 patients. Three patients experienced more severe side effects (muscle aches, mood swings) and did not resume treatment after the drug holiday. Moderate VA loss occurred in 36% at both 6 and 12 months.


CONCLUSIONS
Oral 13-cis retinoic acid is too toxic to be useful in patients with ARMD. Oral 13-cis retinoic acid did not improve vision although it may have slowed visual acuity loss in patients with ARMD with occult subfoveal CNV.
Lim
Jennifer I
JI
Doheny Retina Institute of the Doheny Eye Institute, Los Angeles, CA, USA. jennifil@usc.edu
Walonker
A Frances
AF
Levin
Lori
L
Mahmoud
Mahmoud
M
Sadda
Srinivas
S
Flaxel
Christina J
CJ
Humayun
Mark
M
deJuan
Eugene
E
Labree
Laurie
L
D000284:Administration, Oral; D000368:Aged; D000369:Aged, 80 and over; D020256:Choroidal Neovascularization / Q000188:drug therapy* / Q000209:etiology; D005260:Female; D005451:Fluorescein Angiography; D005500:Follow-Up Studies; D005584:Fovea Centralis; D006801:Humans; D015474:Isotretinoin / Q000009:adverse effects / Q000627:therapeutic use*; D008268:Macular Degeneration / Q000150:complications / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D016896:Treatment Outcome; D014792:Visual Acuity / Q000187:drug effects
D015474:Isotretinoin