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JIMD Reports, Volume 25

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Cover of 'JIMD Reports, Volume 25'

Table of Contents

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    Book Overview
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    Chapter 421 Coenzyme Q10 and Pyridoxal Phosphate Deficiency Is a Common Feature in Mucopolysaccharidosis Type III.
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    Chapter 454 The Pathobiochemistry of Gastrointestinal Symptoms in a Patient with Niemann-Pick Type C Disease
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    Chapter 456 PNPO Deficiency and Cirrhosis: Expanding the Clinical Phenotype?
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    Chapter 457 The Spectrum of Krabbe Disease in Greece: Biochemical and Molecular Findings.
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    Chapter 458 Liver Fibrosis Associated with Iron Accumulation Due to Long-Term Heme-Arginate Treatment in Acute Intermittent Porphyria: A Case Series
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    Chapter 459 Exercise Intolerance and Myoglobinuria Associated with a Novel Maternally Inherited MT-ND1 Mutation.
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    Chapter 461 Vitamin E Improves Clinical Outcome of Patients Affected by Glycogen Storage Disease Type Ib
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    Chapter 462 New Cases of DHTKD1 Mutations in Patients with 2-Ketoadipic Aciduria
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    Chapter 465 Urine Beta2-Microglobulin Is an Early Marker of Renal Involvement in LPI
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    Chapter 466 Improvement of Diffusion Tensor Imaging (DTI) Parameters with Decoppering Treatment in Wilson’s Disease
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    Chapter 467 Screening Mucopolysaccharidosis Type IX in Patients with Juvenile Idiopathic Arthritis
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    Chapter 469 GM2-Gangliosidosis, AB Variant: Clinical, Ophthalmological, MRI, and Molecular Findings
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    Chapter 472 Pitfalls in Diagnosing Neuraminidase Deficiency: Psychosomatics and Normal Sialic Acid Excretion.
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    Chapter 480 Successful Domino Liver Transplantation from a Patient with Methylmalonic Acidemia
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    Chapter 483 Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease
Attention for Chapter 483: Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease
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Chapter title
Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease
Chapter number 483
Book title
JIMD Reports, Volume 25
Published in
JIMD Reports, January 2015
DOI 10.1007/8904_2015_483
Pubmed ID
Book ISBNs
978-3-66-249667-1, 978-3-66-249668-8
Authors

Ozlem Goker-Alpan, Michael J. Gambello, Gustavo H. B. Maegawa, Khan J. Nedd, Daniel J. Gruskin, Larry Blankstein, Neal J. Weinreb, Goker-Alpan, Ozlem, Gambello, Michael J., Maegawa, Gustavo H. B., Nedd, Khan J., Gruskin, Daniel J., Blankstein, Larry, Weinreb, Neal J.

Abstract

Agalsidase alfa and agalsidase beta, recombinant enzyme preparations for treatment of Fabry disease (FD), have different approved dosing schedules: 0.2 mg/kg and 1.0 mg/kg every other week (EOW), respectively. This open-label, multicenter, exploratory phase 4 study evaluated plasma globotriaosylsphingosine (lyso-GL-3) and plasma and urine globotriaosylceramide (GL-3) levels at baseline and 2, 4, and 6 months after the switch from agalsidase alfa (0.2 mg/kg EOW for ≥12 months) to agalsidase beta (1.0 mg/kg EOW) in 15 male patients with FD. Immunoglobulin (Ig)G antidrug antibody titers were assessed, and safety was monitored throughout the study. Plasma lyso-GL-3 concentrations decreased significantly within 2 months after switch and reductions continued through month 6 (mean absolute changes, -12.8, -16.1, and -16.7 ng/mL at 2, 4, and 6 months, respectively; all P < 0.001). The mean percentage reduction from baseline was 39.5% (P < 0.001) at month 6. For plasma GL-3, the mean absolute change from baseline (-0.9 μg/mL) and percentage reduction (17.9%) at month 6 were both significant (P < 0.05). Urine GL-3 measurements showed intra-patient variability and changes from baseline were not significant. No clinical outcomes were assessed in this 6-month study, and, therefore, no conclusions can be drawn regarding the correlation of observed reductions in glycosphingolipid concentrations with clinically relevant outcomes. There were no differences in IgG antidrug antibody titers between the two enzymes. The switch from agalsidase alfa to agalsidase beta was well tolerated. Plasma lyso-GL-3 and GL-3 levels reduced after switching from agalsidase alfa to agalsidase beta, indicating that agalsidase beta has a greater pharmacodynamic effect on these markers at the recommended dose. These data further support the use of agalsidase beta 1.0 mg/kg EOW as enzyme replacement therapy in FD.

Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 32 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Spain 1 3%
Unknown 31 97%

Demographic breakdown

Readers by professional status Count As %
Researcher 6 19%
Other 5 16%
Student > Bachelor 3 9%
Professor > Associate Professor 3 9%
Student > Master 3 9%
Other 4 13%
Unknown 8 25%
Readers by discipline Count As %
Medicine and Dentistry 11 34%
Biochemistry, Genetics and Molecular Biology 3 9%
Agricultural and Biological Sciences 3 9%
Nursing and Health Professions 2 6%
Pharmacology, Toxicology and Pharmaceutical Science 2 6%
Other 2 6%
Unknown 9 28%