Abstract
Cystinosis is a hereditary genetic disease that results in the accumulation of cystine crystals in the lysosomes, leading to many clinical manifestations. One of these manifestations is the formation of corneal cystine crystals, which can cause serious ocular complications. The only available drug to treat cystinosis is cysteamine, which breaks cystine and depletes its accumulation in the lysosomes. However, the oral form of cysteamine is not effective in treating corneal manifestations. Thus, ophthalmic solutions of cysteamine are applied. Because the commercial cysteamine eye drops are not available in most countries, hospital pharmacies are responsible for preparing “homemade” drops usually without a control of stability of cysteamine in different storage conditions. Hence, we aimed in this study to investigate the effect of different storage conditions on the stability of a cysteamine ophthalmic compounded solution. Cysteamine ophthalmic solution was prepared in the hospital pharmacy and sterilized using a candle filter. The preparations are then stored either in the freezer at −20°C or in the refrigerator at +4°C for up to 52 weeks. The amount of cysteamine hydrochloride in the preparation at different time points was determined using capillary electrophoresis (CE). Storage of the cysteamine ophthalmic preparations at +4° resulted in significant loss of free cysteamine at all time points, from 1 to 52 weeks of storage, when compared with storage in the freezer (−20°C). We demonstrate that cysteamine 0.5% compounded eye drops are easily oxidized within the first week after storage at +4°C, rendering the preparation less effective. Storage at −20°C is recommended to prevent this process.
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Acknowledgment
A.R. and E.L. are supported by the Irish cystinosis foundation. M.A.E. is supported by ERA-Net, E-Rare2-JTC2014: Novel therapies for cystinosis. E.L. is supported by the Research Foundation – Flanders (F.W.O. Vlaanderen), grant 1801110N, and the Cystinosis Research Network (CRN).
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Communicated by: Bruce A Barshop, MD, PhD
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Synopsis
Cystinosis patients may get less effective ophthalmological treatment than what is expected.
Details of the Contributions of Individual Authors
Ahmed Reda, Lambertus van den Heuvel, and Elena Levtchenko contributed in planning. Ahmed Reda, Ann Van Schepdael, Erwin Adams, Prasanta Paul, David Devolder, and Ingele Casteels contributed in conducting. Ahmed Reda, Mohamed A. Elmonem, Koenraad Veys, Lambertus van den Heuvel, and Elena Levtchenko contributed in reporting.
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Prof. Elena Levtchenko.
A Competing Interest Statement
Ahmed Reda, Ann Van Schepdael, Erwin Adams, Prasanta Paul, David Devolder, Mohamed A. Elmonem, Koenraad Veys, Ingele Casteels, and Lambertus van den Heuvel declare that they have no conflict of interest.
Elena Levtchenko has a consultancy agreement with Horizon Pharma and Orphan Europe and a financial grant support from Horizon Pharma.
Details of Funding
Ahmed Reda and Elena Levtchenko are supported by the Irish cystinosis foundation. Mohamed A. Elmonem is supported by ERA-Net, E-Rare2-JTC2014: Novel therapies for cystinosis. Elena Levtchenko is supported by the Research Foundation – Flanders (F.W.O. Vlaanderen), grant 1801110N, and the Cystinosis Research Network (CRN).
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This article does not contain any studies with human or animal subjects performed by any of the authors.
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© 2017 Society for the Study of Inborn Errors of Metabolism (SSIEM)
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Reda, A. et al. (2017). Effect of Storage Conditions on Stability of Ophthalmological Compounded Cysteamine Eye Drops. In: Morava, E., Baumgartner, M., Patterson, M., Rahman, S., Zschocke, J., Peters, V. (eds) JIMD Reports, Volume 42. JIMD Reports, vol 42. Springer, Berlin, Heidelberg. https://doi.org/10.1007/8904_2017_77
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DOI: https://doi.org/10.1007/8904_2017_77
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