Skip to main content
release_alert
Welcome to the new Scholars 3.0! Read about new features and let us know what you think.
cancel
Journal cover image

Quantitative computed tomography for enzyme replacement therapy in Pompe disease.

Publication ,  Journal Article
Yonee, C; Toyoshima, M; Young, SP; Maruyama, S; Higuchi, I; Narita, A; Maegaki, Y; Nanba, E; Ohno, K; Kawano, Y
Published in: Brain Dev
November 2012

OBJECTIVE: Pompe disease is an autosomal recessive disorder caused by deficiency of the lysosomal enzyme, acid alpha-glucosidase (GAA). To the best of our knowledge, no studies have reported the results of systematic and sequential CT analyses before and during ERT. In this study we have treated three patients with late onset Pompe disease by ERT, and investigated the efficacy of treatment by computed tomography number. METHODS: We measured the serial changes in the computed tomography (CT) number of multiple organs in three patients with late onset of Pompe disease during 24 months of enzyme replacement therapy (ERT). RESULTS: Before treatment, the liver and muscle CT numbers were higher in these patients than in the controls. The liver CT number decreased after performing ERT. Furthermore, the urinary glucose tetrasaccharide levels, a biomarker of glycogen accumulation, were elevated before ERT and reduced thereafter. CONCLUSIONS: The findings in these cases suggest that the elevation of the liver CT number represents glycogen accumulation in the liver and that the analysis of the liver CT number is therefore a useful tool for assessing the efficacy of ERT.

Duke Scholars

Published In

Brain Dev

DOI

EISSN

1872-7131

Publication Date

November 2012

Volume

34

Issue

10

Start / End Page

834 / 839

Location

Netherlands

Related Subject Headings

  • alpha-Glucosidases
  • Tomography, X-Ray Computed
  • Oligosaccharides
  • Neurology & Neurosurgery
  • Muscle, Skeletal
  • Liver
  • Humans
  • Glycogen Storage Disease Type II
  • Glycogen
  • Female
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Yonee, C., Toyoshima, M., Young, S. P., Maruyama, S., Higuchi, I., Narita, A., … Kawano, Y. (2012). Quantitative computed tomography for enzyme replacement therapy in Pompe disease. Brain Dev, 34(10), 834–839. https://doi.org/10.1016/j.braindev.2012.01.013
Yonee, Chihiro, Mitsuo Toyoshima, Sarah P. Young, Shinsuke Maruyama, Itsuro Higuchi, Aya Narita, Yoshihiro Maegaki, Eiji Nanba, Kousaku Ohno, and Yoshifumi Kawano. “Quantitative computed tomography for enzyme replacement therapy in Pompe disease.Brain Dev 34, no. 10 (November 2012): 834–39. https://doi.org/10.1016/j.braindev.2012.01.013.
Yonee C, Toyoshima M, Young SP, Maruyama S, Higuchi I, Narita A, et al. Quantitative computed tomography for enzyme replacement therapy in Pompe disease. Brain Dev. 2012 Nov;34(10):834–9.
Yonee, Chihiro, et al. “Quantitative computed tomography for enzyme replacement therapy in Pompe disease.Brain Dev, vol. 34, no. 10, Nov. 2012, pp. 834–39. Pubmed, doi:10.1016/j.braindev.2012.01.013.
Yonee C, Toyoshima M, Young SP, Maruyama S, Higuchi I, Narita A, Maegaki Y, Nanba E, Ohno K, Kawano Y. Quantitative computed tomography for enzyme replacement therapy in Pompe disease. Brain Dev. 2012 Nov;34(10):834–839.
Journal cover image

Published In

Brain Dev

DOI

EISSN

1872-7131

Publication Date

November 2012

Volume

34

Issue

10

Start / End Page

834 / 839

Location

Netherlands

Related Subject Headings

  • alpha-Glucosidases
  • Tomography, X-Ray Computed
  • Oligosaccharides
  • Neurology & Neurosurgery
  • Muscle, Skeletal
  • Liver
  • Humans
  • Glycogen Storage Disease Type II
  • Glycogen
  • Female