Effect of Age at Treatment on Cognitive Performance in Patients with Cystinosis

• Published in: The Journal of pediatrics Vol. 163; no. 2; pp. 489 - 492
• Main Authors: Viltz, Lisa, BS, Trauner, Doris A., MD
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-08-2013; Mosby, Inc
• Subjects: Adolescent; age; Age Factors; Child; Child, Preschool; Cognition; Cognition Disorders - etiology; Cognition Disorders - prevention & control; cysteamine; Cysteamine - therapeutic use; Fanconi Syndrome - complications; Fanconi Syndrome - drug therapy; Fanconi Syndrome - psychology; Humans; patients; Pediatrics; Young Adult; VMI; Beery-Buktenica Developmental Test of Visual-Motor Integration
• ISSN: 0022-3476; 1097-6833
• DOI: 10.1016/j.jpeds.2013.01.027

Objective To determine whether early treatment with cysteamine affects cognitive functioning in patients with nephropathic cystinosis. Study design Forty-six subjects aged 3-18 years with cystinosis underwent cognitive testing to determine intelligence, visual-spatial abilities, and visual-motor skills. An age-matched control group (n = 85; age 2-22 years) underwent the same tests. Age at diagnosis and age at initiation of treatment with cysteamine were recorded at the time of testing. Results Patients with cystinosis treated at or after age 2 years (late-treatment group) scored significantly lower on verbal, performance, and full-scale IQ measures, as well as on a test of visual-spatial skills, compared with patients treated before age 2 years (early-treatment group) and controls. Both groups of subjects with cystinosis demonstrated impaired visual-motor skills compared with controls, with no difference between the early-treatment and late-treatment groups. Conclusion Early treatment with cysteamine appears to improve intellectual function in patients with nephropathic cystinosis. However, the lack of improvement in visual-motor function with early cysteamine treatment suggests possibly different mechanisms underlying visual-motor performance compared with other areas of cognition in this disorder.