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

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.

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.

Keywords: Clinical case; Cyclodextrin; HPLC; Methodology; Niemann-Pick disease type C; Pediatrics.

Publication types

  • Case Reports

MeSH terms

  • 2-Hydroxypropyl-beta-cyclodextrin* / chemistry
  • 2-Hydroxypropyl-beta-cyclodextrin* / pharmacokinetics
  • 2-Hydroxypropyl-beta-cyclodextrin* / therapeutic use
  • 2-Hydroxypropyl-beta-cyclodextrin* / urine
  • Anticholesteremic Agents* / chemistry
  • Anticholesteremic Agents* / pharmacokinetics
  • Anticholesteremic Agents* / therapeutic use
  • Anticholesteremic Agents* / urine
  • Child, Preschool
  • Chromatography, High Pressure Liquid / methods*
  • Humans
  • Light
  • Niemann-Pick Disease, Type C* / drug therapy
  • Niemann-Pick Disease, Type C* / metabolism
  • Orphan Drug Production
  • Reproducibility of Results
  • Scattering, Radiation

Substances

  • Anticholesteremic Agents
  • 2-Hydroxypropyl-beta-cyclodextrin