应用简单方法学，采用 HPLC-LS 分析早发性尼曼-匹克病 C 型患者尿液中的羟丙基-β-环糊精。

Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in urine using HPLC-LS in early Niemann-Pick disease type C patient.

机构信息

Departamento de Bioquímica y Biología molecular-A, Facultad de Biología, Universidad de Murcia - Regional Campus of International Excellence "Campus Mare Nostrum", E-30100 Murcia, Spain.

Hospital Universitario Sanitas La Zarzuela, Calle de Pleyades, 25, E-28023 Madrid, Spain.

出版信息

J Chromatogr B Analyt Technol Biomed Life Sci. 2018 Sep 1;1093-1094:47-51. doi: 10.1016/j.jchromb.2018.06.051. Epub 2018 Jun 25.

DOI:10.1016/j.jchromb.2018.06.051

PMID:29990712

Abstract

A new methodology, based on high resolution liquid chromatography with light scatterin detector, is applied to analyze Hydroxypropyl-beta-Cyclodextrin (HPβCD) in urine samples of a child affected by Niemann-Pick Type C disease. The treatment not only stopped disease progression, but has also increased the life expectancy and quality of our patient. The pharmacokinetic of HPβCD in the patient was studied with a 92.8% of HPβCD recovered. At 88 h, no HPβCD was found in the urine. During the treatment, HPβCD has not shown toxicity. Before application of the new treatment, injections were given every two weeks but, we have demonstrated that this can be increased to every four days.

摘要

一种新的方法，基于高效液相色谱与光散射检测器，被应用于分析患有尼曼-匹克 C 型疾病的儿童尿液样本中的羟丙基-β-环糊精（HPβCD）。该治疗不仅阻止了疾病的进展，还延长了我们患者的预期寿命和提高了生活质量。对患者进行了 HPβCD 的药代动力学研究，回收率为 92.8%。在 88 小时时，尿液中未发现 HPβCD。在治疗过程中，HPβCD 未显示出毒性。在应用新的治疗方法之前，每两周注射一次，但我们已经证明可以增加到每四天一次。