ArticleOphthalmology and therapy2019
The Ocular Status of Cystinosis Patients Receiving a Hospital Pharmacy-Made Preparation of Cysteamine Eye Drops: A Case Series.
Article in Ophthalmology and therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed, 15 citations in OpenAlex.
- The Use of a Viscous Cysteamine Eyedrop in a Belgian Cohort: Early 3-Year Results.Ophthalmology and therapy · 2025Article
- A Comparative Pharmacokinetic Study for Cysteamine-Containing Eye Drops as an Orphan Topical Therapy in Cystinosis.International journal of molecular sciences · 2024Article
- Cysteamine Eye Drops in Hyaluronic Acid Packaged in Innovative Single-Dose Systems, Part II: Long-Term Stability and Clinical Ocular Biopermanence.Pharmaceutics · 2023Article
- Spectral domain optical coherence tomography-based retinochoroidal cystine crystal score: a window into infantile nephropathic cystinosis.The British journal of ophthalmology · 2023Article
- Development of a fibrin-mediated gene delivery system for the treatment of cystinosis via design of experiment.Scientific reports · 2022Article
- Limitations and Challenges in the Stability of Cysteamine Eye Drop Compounded Formulations.Pharmaceuticals (Basel, Switzerland) · 2021Article
- Long-term follow-up of cystinosis patients treated with 0.55% cysteamine hydrochloride.The British journal of ophthalmology · 2021Observational
- Ophthalmic Evaluation of Diagnosed Cases of Eye Cystinosis: A Tertiary Care Center's Experience.Diagnostics (Basel, Switzerland) · 2020Article
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Authors and funding
2 authors at 2 institutions in 1 country.
Funding
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Abstract
introductionInfantile nephropathic cystinosis (INC) is an autosomal recessive lysosomal disorder in which patients develop deposits of cystine crystals in their kidneys and corneas from a young age.
methodsWe conducted a retrospective analysis of children with INC seen by ophthalmologists at the Manchester Royal Eye Hospital between 2002 and 2018, to evaluate clinical findings, symptoms and treatment.
resultsTwenty-two children diagnosed with INC from age 0 (prenatally) to 11 years were assessed. All evaluable patients had corneal cystine crystal deposits, and 15 had mild to moderate photophobia. Ten patients had other ocular conditions including blepharitis/chalzion (n = 6), swollen optic nerve (n = 3), punctate epitheliopathy (n = 3), corneal scarring (n = 1),and elevated intraocular pressure (n = 2). Confocal imaging identified nerve abnormalities in two patients (enlarged corneal nerve + abnormal-looking tortuous nerves in one patient and beaded nerves in the sub-basal plexus in the other), both of whom had significant crystal deposition in the anterior stroma. Visual acuity was relatively unaffected. All 22 patients were receiving oral cysteamine, and 21 were applying cysteamine eye drops (galenic preparation of 0.55% concentration, compounded by a hospital pharmacy). Recommended application frequency was at least eight times per day in all patients with dosing information available.
conclusionsThis case series of patients with INC highlights the consistent pattern of corneal cystine crystal deposition, which is universally present from a young age in this condition, and the high incidence of photophobia even in young children. Corneal manifestations of INC persisted despite frequent administration of the hospital pharmacy-made eye drop preparation. Reasons for this lack of efficacy may include the lag period between diagnosis and first prescription of cysteamine eye drops and the difficulty in maintaining rigorous compliance with this treatment. In addition, the challenge for patients of maintaining optimal storage conditions may adversely affect the stability and efficacy of cysteamine within this preparation.
fundingEditorial assistance was funded by Orphan Europe Ltd.
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