 |
|
A Randomized Placebo-Controlled Trial of Cysteamine Eye Drops in Nephropathic Cystinosis
Muriel I. Kaiser-Kupfer, MD;
Maria A. Gazzo, MD;
Manuel B. Datiles, MD;
Rafael C. Caruso, MD;
Ernest M. Kuehl;
William A. Gahl, MD, PhD
Arch Ophthalmol. 1990;108(5):689-693.
Abstract
| |
• 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.
Author Affiliations
From the Ophthalmic Genetics and Clinical Services Branch, National Eye Institute (Drs Kaiser-Kupfer, Gazzo, Datiles, and Caruso and Mr Kuehl), and the Section on Human Biochemical Genetics, Human Genetics Branch, National Institute of Child Health and Human Development (Dr Gahl), the National Institutes of Health, Bethesda, Md.
Footnotes
Accepted for publication January 13, 1990.
Reprint requests to NIH-NEI, 9000 Rockville Pike, Bldg 10, Room 10N226, Bethesda, MD 20892 (Dr Kaiser-Kupfer).
 CiteULike  Connotea  Del.icio.us  Digg  Reddit  Technorati  Twitter
What's this?
THIS ARTICLE HAS BEEN CITED BY OTHER ARTICLES
|
Nephropathic Cystinosis in Adults: Natural History and Effects of Oral Cysteamine Therapy
Gahl et al.
ANN INTERN MED 2007;147:242-250.
ABSTRACT
| FULL TEXT
A multicentre randomised double masked clinical trial of a new formulation of topical cysteamine for the treatment of corneal cystine crystals in cystinosis
Tsilou et al.
Br J Ophthalmol 2003;87:28-31.
ABSTRACT
| FULL TEXT
Cystinosis: Antibodies and Healthy Bodies
Kleta and Gahl
J. Am. Soc. Nephrol. 2002;13:2189-2191.
FULL TEXT
Cystinosis
Gahl et al.
NEJM 2002;347:111-121.
FULL TEXT
Pulmonary Dysfunction in Adults With Nephropathic Cystinosis
Anikster et al.
Chest 2001;119:394-401.
ABSTRACT
| FULL TEXT
Ultrasound Biomicroscopy of the Eye in Cystinosis
Mungan et al.
Arch Ophthalmol 2000;118:1329-1333.
ABSTRACT
| FULL TEXT
Classic Nephropathic Cystinosis as an Adult Disease
Theodoropoulos et al.
JAMA 1993;270:2200-2204.
ABSTRACT
Improved Renal Function in Children with Cystinosis Treated with Cysteamine
Markello et al.
NEJM 1993;328:1157-1162.
ABSTRACT
| FULL TEXT
Ocular Manifestations of Nephropathic Cystinosis: The French-Canadian Experience in a Genetically Homogeneous Population
Richler et al.
Arch Ophthalmol 1991;109:359-362.
ABSTRACT
|
