Background/Aims Cystinosis is a rare, autosomal recessive disorder causing defective transport of cystine out of lysosomes. Cystadrops (0.55% cysteamine hydrochloride in viscous solution) has been used on a named-patient basis to treat the accumulation of cystine crystals in the cornea in patients with cystinosis.
Methods Retrospective analysis of the Temporary Authorisation for Use cohort of 130 patients who received Cystadrops between 2013 and 2017 in France.
Results Patients received an average dosage of 3.3 (±0.94) instillations per eye per day. Over the duration of follow-up, of up to 45 months, patients maintained visual acuity scores of 0.0, which approximated normal. Corneal cystine crystal scores tended to decrease over time, stabilising after around 27 months between 1.22 and 1.87. Photophobia decreased within 3 months, stabilising on scores of around 1.5 and 1.7. 47 non-serious adverse reactions were reported, which were generally transient irritation, stinging or blurred vision. Four serious adverse events were reported, including keratitis and corneal ulcer, but these may have been caused by the underlying disease.
Conclusion This large safety cohort confirms the efficacy, safety and tolerability of Cystadrops in real-world clinical practice.
- Ocular surface
- Clinical Trial
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Correction notice This paper has been corrected since it was published online. Two small changes were made to table 2.
Contributors All listed authors contributed to the conception or design of this study; or the acquisition, analysis or interpretation of patient data; drafted or revised the work critically; and approved this version to be published. HL and VG are responsible for the overall content as guarantors. Guy Ramsay provided editorial assistance.
Funding No specific grant or funding from any source or agency was received for this work.
Competing interests HL has received speaker honoraria from Recordati Rare Diseases, HL has received research funding from October-1 and CHOC clinical trials honoraria. AL has received speaker honoraria from Recordati Rare Diseases, and research funding from October-1 honoraria. CB has received honoraria from Orphan Europe. CP and VG are both employees of Recordati Rare Diseases.
Patient or parental consent for publication Not required.
Data sharing statement Data are available in a public, open-access repository. Data are available upon reasonable request.
Data availability statement The data that supports the findings of this study are available from the corresponding author, HL, upon reasonable request and upon approval of Recordati Rare Diseases, CP.
Provenance and peer review Not commissioned.
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