We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Treatment of cystinosis with delayed-release cysteamine: 6-year follow-up.
- Authors
Dohil, Ranjan; Cabrera, Betty
- Abstract
Background: Patients with nephropathic cystinosis are required to take 6-hourly immediate-release cysteamine (Cystagon®) to reduce disease progression. This arduous regimen affects quality of life, disrupts sleep, and may result in non-compliance with therapy. Enteric-coated cysteamine bitartrate (EC-cysteamine) was developed as a 'proof-of-concept' formulation for twice-daily ingestion. Previous reports have shown this therapy to be effective up to a mean of 14 months. Case-Diagnosis/Treatment: Two subjects (aged 13 and 15 years) received EC-cysteamine for 5-6 years at 60-65 % of their previous total daily dose of immediate-release cysteamine given at 6-h intervals. White blood cell (WBC) cystine levels were monitored every 1-3 months. Conclusion: The administration of EC-cysteamine did not result in any change in mean trough WBC cystine levels or any deterioration in the estimated glomerular filtration rate, thyroid, or liver function, suggesting that delayed-release, twice-daily EC-cysteamine is an effective long-term treatment alternative to immediate-release cysteamine given at 6-h intervals.
- Subjects
AMINES; CONTROLLED release preparations; LONGITUDINAL method; INBORN errors of metabolism; QUALITY of life; RESEARCH funding; CYSTEINE; DESCRIPTIVE statistics
- Publication
Pediatric Nephrology, 2013, Vol 28, Issue 3, p507
- ISSN
0931-041X
- Publication type
Report
- DOI
10.1007/s00467-012-2315-5