Abstract |
Fifty-nine patients with cystinosis were treated with cysteamine or phosphocysteamine in the United Kingdom up to May 1990. Treatment was started at a median age of 3.2 years (range 0.6-24.8 years) and continued for a median duration of 3.0 years (range 0.01-1.2 years). At the end of the study, 46 (78%) patients remained on treatment. One patient developed end-stage renal failure and 6 died. Efficacy was assessed in the 44 pre-transplant patients. The United Kingdom pre-transplant patients had significantly lower plasma creatinine concentrations at 6 and 8 years than a historical group of patients who did not receive cysteamine (P < 0.0001 and P < 0.0003, respectively). There was no significant difference between pretreatment and final post-treatment height standard deviation scores, suggesting maintenance of growth rate. The leucocyte cystine concentration was less than the accepted upper limit of the treatment range (1 nmol 1/2 cystine/mg protein) in only 21% of determinations. There was no significant difference between the mean pre-treatment and final values of leucocyte cystine concentration. The mean final doses of cysteamine (33 mg/kg per day) and phosphocysteamine (37 mg/kg per day base equivalent) were less than the mean dose (51 mg/kg per day) used in a United States multicentre trial. We conclude that cysteamine treatment was beneficial, but further improvements might be achieved by an improvement in monitoring of therapy.
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Authors | W G van't Hoff, N Gretz |
Journal | Pediatric nephrology (Berlin, Germany)
(Pediatr Nephrol)
Vol. 9
Issue 6
Pg. 685-9
(Dec 1995)
ISSN: 0931-041X [Print] Germany |
PMID | 8747105
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
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Chemical References |
- Cystine
- Cysteamine
- Cystaphos
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Topics |
- Adolescent
- Child
- Child, Preschool
- Cystaphos
(therapeutic use)
- Cysteamine
(therapeutic use)
- Cystine
(blood)
- Cystinosis
(drug therapy, mortality, physiopathology)
- Female
- Growth
(drug effects)
- Humans
- Infant
- Ireland
- Kidney Function Tests
- Leukocytes
(metabolism)
- Male
- Retrospective Studies
- United Kingdom
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