Treatment of cystinosis with delayed-release cysteamine: 6-year follow-up.
Pediatr Nephrol
; 28(3): 507-10, 2013 Mar.
Article
em En
| MEDLINE
| ID: mdl-23001048
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.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Cisteamina
/
Cistinose
/
Síndrome de Fanconi
/
Rim
/
Síndrome Nefrótica
Tipo de estudo:
Diagnostic_studies
Limite:
Adolescent
/
Adult
/
Female
/
Humans
/
Male
Idioma:
En
Revista:
Pediatr Nephrol
Assunto da revista:
NEFROLOGIA
/
PEDIATRIA
Ano de publicação:
2013
Tipo de documento:
Article
País de afiliação:
Estados Unidos