A major advance in treating cystinosis

Abstract

Although many pediatricians have not heard of it, cysteamine bitartrate (Cystagon) can be considered one of pediatrics' “miracle drugs.” By promoting the movement of cystine out of the cell, the drug can arrest the ongoing organ damage in cystinosis. Prior to the development of this drug, renal failure in cystinosis was inevitable; today, with early introduction of cysteamine treatment, it can be postponed indefinitely. Unfortunately, the drug has a number of side effects that make adherence with therapy quite difficult. The recent development of an enteric coated product had been hoped to increase compliance by virtue of less frequent dosing (twice daily, instead of every 6 hours) and less troublesome gastrointestinal distress. The current issue of The Journal reports the long term (10 to 27 months) experience with enteric coated cysteamine in 5 children with cystinosis. Depletion of intracellular cystine was equivalent with this regimen to that achieved with every 6 hour dosing of the current product, and there was a trend toward less gastrointestinal side effects. Cystinosis may not be a common disease, but the development of a simple therapy that can promote growth and prevent kidney transplantation is a tribute to the efforts of basic scientists. This paper also provides the generalist with a nice review of the basic clinical chemistry involved. Long-Term Treatment of Cystinosis in Children with Twice-Daily CysteamineThe Journal of PediatricsVol. 156Issue 5PreviewCystinosis causes renal and other organ failure. Treatment with 6-hourly cysteamine bitartrate (Cystagon, Mylan, Morgantown, West Virginia) reduces intracellular cystine and the rate of organ deterioration. A recent study showed that an enteric-release cysteamine required less frequent daily dosing. This report describes the long-term use of enteric-coated (EC) cysteamine bitartrate (Cystagon) in children with cystinosis. Full-Text PDF