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Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy.

Publication ,  Journal Article
Zhang, H; Dickson, PI; Stiles, AR; Chen, AH; Le, SQ; McCaw, P; Beasley, J; Millington, DS; Young, SP
Published in: Clin Chim Acta
September 2020

AIMS: To validate a liquid chromatography-tandem mass spectrometry (UPLC-MS/MS) method for the measurement of glycosaminoglycans (GAGs) in plasma and serum. To establish plasma, cerebrospinal fluid (CSF) and urine reference intervals. To compare GAGs in serum with that in urine and CSF from patients with MPS I. METHODS: Dermatan sulfate (DS), heparan sulfate (HS), and chondroitin sulfate (CS) in serum/plasma, urine and CSF were methanolysed into dimers and analyzed using pseudo isotope dilution UPLC-MS/MS assay. Serum, CSF and urine DS and HS were quantified for 11 patients with mucopolysaccharidosis (MPS) type I before and after treatment with Aldurazyme® (laronidase) enzyme replacement therapy (ERT). RESULTS: The method showed acceptable imprecision and recovery for the quantification of serum/plasma CS, DS, and HS. The serum, urine, and CSF DS and HS concentrations were reduced after 26 weeks of ERT in 4 previously untreated patients. Serum DS and HS concentrations normalized in some patients, and were mildly elevated in others after ERT. In contrast, urine and CSF DS and HS values remained elevated above the reference ranges. Compared with serum GAGs, urine and CSF DS and HS were more sensitive biomarkers for monitoring the ERT treatment of patients with MPS I.

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Published In

Clin Chim Acta

DOI

EISSN

1873-3492

Publication Date

September 2020

Volume

508

Start / End Page

179 / 184

Location

Netherlands

Related Subject Headings

  • Tandem Mass Spectrometry
  • Mucopolysaccharidosis I
  • Humans
  • Heparitin Sulfate
  • Glycosaminoglycans
  • General Clinical Medicine
  • Enzyme Replacement Therapy
  • Dermatan Sulfate
  • Chromatography, Liquid
  • 3202 Clinical sciences
 

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Zhang, H., Dickson, P. I., Stiles, A. R., Chen, A. H., Le, S. Q., McCaw, P., … Young, S. P. (2020). Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy. Clin Chim Acta, 508, 179–184. https://doi.org/10.1016/j.cca.2020.05.035
Zhang, Haoyue, Patricia I. Dickson, Ashlee R. Stiles, Agnes H. Chen, Steven Q. Le, Patricia McCaw, James Beasley, David S. Millington, and Sarah P. Young. “Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy.Clin Chim Acta 508 (September 2020): 179–84. https://doi.org/10.1016/j.cca.2020.05.035.
Journal cover image

Published In

Clin Chim Acta

DOI

EISSN

1873-3492

Publication Date

September 2020

Volume

508

Start / End Page

179 / 184

Location

Netherlands

Related Subject Headings

  • Tandem Mass Spectrometry
  • Mucopolysaccharidosis I
  • Humans
  • Heparitin Sulfate
  • Glycosaminoglycans
  • General Clinical Medicine
  • Enzyme Replacement Therapy
  • Dermatan Sulfate
  • Chromatography, Liquid
  • 3202 Clinical sciences