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

Jennifer I. Lim, A. Frances Walonker, Lori Levin, Mahmoud Mahmoud, Srinivas Sadda, Christina Flaxel, Mark Humayun, Eugene Dejuan, Laurie Labree

Research output: Contribution to journalArticle

4 Citations (Scopus)

Abstract

PURPOSE: 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.

Original languageEnglish (US)
Pages (from-to)314-321
Number of pages8
JournalRetina
Volume26
Issue number3
DOIs
StatePublished - Mar 2006
Externally publishedYes

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Isotretinoin
Choroidal Neovascularization
Macular Degeneration
Visual Acuity
Therapeutics
Diabetic Retinopathy
Holidays
Fluorescein Angiography
Poisons
Lip
Drug-Related Side Effects and Adverse Reactions
Safety
Pain
Muscles
Skin

Keywords

  • 13-cis retinoic acid
  • Accutane
  • Age-related macular degeneration
  • Predominantly occult choroidal neovascularization

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems

Cite this

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. / Lim, Jennifer I.; Walonker, A. Frances; Levin, Lori; Mahmoud, Mahmoud; Sadda, Srinivas; Flaxel, Christina; Humayun, Mark; Dejuan, Eugene; Labree, Laurie.

In: Retina, Vol. 26, No. 3, 03.2006, p. 314-321.

Research output: Contribution to journalArticle

Lim, Jennifer I. ; Walonker, A. Frances ; Levin, Lori ; Mahmoud, Mahmoud ; Sadda, Srinivas ; Flaxel, Christina ; Humayun, Mark ; Dejuan, Eugene ; Labree, Laurie. / 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. In: Retina. 2006 ; Vol. 26, No. 3. pp. 314-321.
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AU - Lim, Jennifer I.

AU - Walonker, A. Frances

AU - Levin, Lori

AU - Mahmoud, Mahmoud

AU - Sadda, Srinivas

AU - Flaxel, Christina

AU - Humayun, Mark

AU - Dejuan, Eugene

AU - Labree, Laurie

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N2 - PURPOSE: 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.

AB - PURPOSE: 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.

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