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Visual acuity in patients with Leber's congenital amaurosis and early childhood-onset retinitis pigmentosa
Journal article   Peer reviewed

Visual acuity in patients with Leber's congenital amaurosis and early childhood-onset retinitis pigmentosa

Saloni Walia, Gerald A Fishman, Samuel G Jacobson, Tomas S Aleman, Robert K Koenekoop, Elias I Traboulsi, Richard G Weleber, Mark E Pennesi, Elise Heon, Arlene Drack, …
Ophthalmology (Rochester, Minn.), Vol.117(6), pp.1190-1198
06/2010
DOI: 10.1016/j.ophtha.2009.09.056
PMID: 20079931

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Abstract

To correlate visual acuity of patients with Leber's congenital amaurosis (LCA) and early childhood-onset retinitis pigmentosa (RP) with mutations in underlying LCA genes. Multicentered retrospective observational study. After exclusion of 28 subjects, 169 patients with the diagnosis of LCA and 27 patients with early childhood-onset RP were included in the study because the underlying mutations in AIPL1, GUCY2D, RDH12, RPE65, CRX, CRB1, RPGRIP1, CEP290, LCA5, and TULP1 genes could be identified in this cohort of patients. We collected data on best-corrected visual acuity as recorded at the time of the patient's most recent visit to one of the participating ophthalmology departments. The median and range of visual acuities for each genetic subtype were calculated separately for the LCA and early childhood-onset RP groups. The range and median best-corrected visual acuities for each genetic subtype and age-related mean visual acuities for each genetic subtype. A wide variation in visual acuity was observed in patients with LCA and RPE65, RDH12, and CRB1 mutations, whereas AIPL1, GUCY2D, CRX, and RPGRIP1 gene mutations were associated with severely decreased visual acuities beginning within the first year of life. It was also noted that patients with either an RPE65 or CRB1 mutation have progressive visual loss with advancing age. Onset of visual symptoms after infancy was associated with a relatively better visual prognosis. The data obtained from this study will help clinicians provide counseling on visual prognosis to patients with known mutations in LCA genes and be of value in future studies aimed at the treatment of LCA and early childhood-onset RP.
Microtubule-Associated Proteins - genetics Humans Middle Aged Child, Preschool Infant Male Light Signal Transduction - genetics Alcohol Oxidoreductases - genetics Trans-Activators - genetics Aged, 80 and over Adult Female Retrospective Studies Eye Proteins - genetics Neoplasm Proteins - genetics Child Retinitis Pigmentosa - physiopathology Visual Acuity - physiology cis-trans-Isomerases Antigens, Neoplasm - genetics Leber Congenital Amaurosis - physiopathology Membrane Proteins - genetics Retinitis Pigmentosa - genetics Genotype Leber Congenital Amaurosis - genetics Nerve Tissue Proteins - genetics Homeodomain Proteins - genetics Proteins - genetics Carrier Proteins - genetics Adolescent Age of Onset Guanylate Cyclase - genetics Aged Mutation Receptors, Cell Surface - genetics

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