Topical Cysteamine to Tx Corneal Cystine Crystal Accumulation in Cystinosis

Bishop, Rachel

Abstract

This study was originally designed to test the efficacy of topical cysteamine for the treatment of corneal cystine crystal accumulation in patients with cystinosis. It was started in 1986, and by 1993 the efficacy of cysteamine drops had been established, at which time benzalkonium was added to the formulation as a preservative. In the years that followed, two variations of the formula were studied, neither proving as efficacious as the original. That formula, consisting of cysteamine 0.5% plus benzalkonium, has been used since 2001. It received FDA approval in 2012, and was available commercially through Sigma-Tau Pharmaceuticals as of May 2013.

Funding Agency

• Agency: National Institute of Health (NIH)
• Institute: National Eye Institute (NEI)
• Type: Investigator-Initiated Intramural Research Projects (ZIA)
• Project #: 1ZIAEY000519-02
• Application #: 8737678
• Support Year: 2
• Fiscal Year: 2013
• Total Cost: $94,557

Institution

• Name: U.S. National Eye Institute

Publications

Brooks, Brian P; Thompson, Amy H; Bishop, Rachel J et al. (2013) Ocular manifestations of xeroderma pigmentosum: long-term follow-up highlights the role of DNA repair in protection from sun damage. Ophthalmology 120:1324-36

Baird, Kristin; Steinberg, Seth M; Grkovic, Lana et al. (2013) National Institutes of Health chronic graft-versus-host disease staging in severely affected patients: organ and global scoring correlate with established indicators of disease severity and prognosis. Biol Blood Marrow Transplant 19:632-9

Wang, Yujuan; Marciano, Beatriz E; Shen, Defen et al. (2013) Molecular identification of bacterial DNA in the chorioretinal scars of chronic granulomatous disease. J Clin Immunol 33:917-24

Smith, Wendy M; Reddy, Madhu G; Hutcheson, Kelly A et al. (2012) Rifabutin-associated hypopyon uveitis and retinal vasculitis with a history of acute myeloid leukemia. J Ophthalmic Inflamm Infect 2:149-52

Sibley, Cailin H; Plass, Nikki; Snow, Joseph et al. (2012) Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: a cohort study to determine three- and five-year outcomes. Arthritis Rheum 64:2375-86