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用于治疗眼胱氨酸病的半胱胺眼用水凝胶

[Cysteamine ophthalmic hydrogel for the treatment of ocular cystinosis].

作者信息

Fernández-Ferreiro Anxo, Luaces-Rodríguez Andrea, Díaz-Tomé Victoria, Gil-Martínez María, Rodríguez Ares María Teresa, Touriño Peralba Rosario, Blanco-Méndez José, González-Barcia Miguel, Otero-Espinar Francisco Javier, Lamas María Jesús

机构信息

Servicio de Farmacia, Xerencia Xestión Integrada Santiago de Compostela (SERGAS) Grupo Farmacología Clínica, Instituto de Investigación Sanitaria, Santiago de Compostela (IDIS-ISCIII) Departamento de Farmacología, Farmacia y Tecnología Farmacéutica, Facultad de Farmacia, Universidad de Santiago de Compostela (USC).

Grupo Farmacología Clínica, Instituto de Investigación Sanitaria, Santiago de Compostela (IDIS-ISCIII) Departamento de Farmacología, Farmacia y Tecnología Farmacéutica, Facultad de Farmacia, Universidad de Santiago de Compostela (USC).

出版信息

Farm Hosp. 2017 Nov 1;41(6):678-687. doi: 10.7399/fh.10834.

Abstract

Ocular cystinosis is a rare disease characterised by the deposit of cystine crystals on the corneal surface, which hinder patients' eyesight. Oral cysteamine is given as cysteamine; however, it does not reach the cornea due to the lack of corneal vascularization making necessary its  administration by the topical ocular route. The aim of the present study is to  determine the stability of an ophthalmic hydrogel of cysteamine, which can be  potentially prepared at hospital pharmacy departments, under different preservation conditions during a follow-up of 30 days. Different physical  and chemical parameters were evaluated: osmolality, pH and  cysteamine concentration, which has been measured by a method of ultra  performance liquid chromatography-tandem mass spectrometer (UPLC-MS/MS).  Descriptive assays were also performed, such as transparency measurement and  microbiological assays in order to verify its sterility. The obtained results  allow us to conclude that the cysteamine hydrogel is stable during 30 days,  being recommendable its preservation in refrigerated conditions.

摘要

眼胱氨酸病是一种罕见疾病,其特征是胱氨酸晶体沉积在角膜表面,这会妨碍患者视力。口服半胱胺是以半胱胺形式给药;然而,由于角膜缺乏血管化,它无法到达角膜,因此需要通过眼部局部给药途径。本研究的目的是确定一种可在医院药房潜在制备的半胱胺眼用水凝胶在30天随访期间不同保存条件下的稳定性。评估了不同的物理和化学参数:渗透压、pH值和半胱胺浓度,后者通过超高效液相色谱-串联质谱仪(UPLC-MS/MS)方法进行测定。还进行了描述性试验,如透明度测量和微生物学试验以验证其无菌性。所得结果使我们能够得出结论,半胱胺水凝胶在30天内是稳定的,建议在冷藏条件下保存。

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Efficacy of topical cysteamine in nephropathic cystinosis.局部用半胱胺治疗肾病性胱氨酸病的疗效
Br J Ophthalmol. 2017 Sep;101(9):1234-1237. doi: 10.1136/bjophthalmol-2016-309278. Epub 2017 Jan 5.

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