A randomized placebo-controlled trial of cysteamine eye drops in nephropathic cystinosis
M. I. Kaiser-Kupfer, M. A. Gazzo, M. B. Datiles, R. C. Caruso, E. M. Kuehl and W. A. Gahl
Ophthalmic Genetics and Clinical Services Branch, National Eye Institute, National Institutes of Health, Bethesda, Md 20892.
Eighteen patients with nephropathic cystinosis who were younger than 42
months and 11 patients 4 to 31 years of age were entered into a
double-masked, randomized, placebo-controlled trial of topical cysteamine
eye drops between November 1985 and September 1989. Eight of the younger
patients and 2 of the older patients showed marked clearing of corneal
crystals in one eye compared with the fellow eye. When the code was broken,
all 10 patients were found to have received cysteamine eye drops in the
improved eye. Of the remaining 19 patients 4 were unavailable for
follow-up. In 15 patients no marked difference was noted between the two
eyes. Eight have presumably been in the protocol for too short a time and
several have been poor compliers with the therapy. These results not only
demonstrate the potential for primary prevention of corneal crystal
deposition but also, for the first time, offer the possibility of reversing
the corneal complications of cystinosis in older patients.
