Title |
Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs
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Published in |
Journal of Inherited Metabolic Disease, June 2009
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DOI | 10.1007/s10545-009-1198-5 |
Pubmed ID | |
Authors |
M. B. Passage, A. W. Krieger, M. C. Peinovich, T. Lester, S. Q. Le, P. I. Dickson, E. D. Kakkis |
Abstract |
Intravenous enzyme replacement therapy with recombinant human α-L-iduronidase (rhIDU) is used weekly to treat mucopolysaccharidosis (MPS) I. We tested continuous administration of rhIDU at two dosing levels (0.58 mg/kg per week and 2 mg/kg per week) in MPS I dogs, and compared the efficacy of continuous infusion with the clinically used 0.58 mg/kg weekly three-hour infusion. Peak plasma concentrations of rhIDU were much higher in weekly-treated dogs (mean 256 units/ml) than steady-state concentrations in dogs treated with continuous infusion (mean 1.97 units/ml at 0.58 mg/kg per week; 8.44 units/ml at 2 mg/kg per week). Dogs receiving continuous IV rhIDU, even at a higher (2 mg/kg per week) dose, had consistently lower iduronidase levels in tissues than dogs receiving a weekly (0.58 mg/kg per week) dose. GAG storage was also less improved by continuous intravenous infusion. Adverse events were similar in all dosing groups. We found that continuous administration of 2 mg/kg per week rhIDU to MPS I dogs was insufficient to achieve GAG storage reduction comparable to 0.58 mg/kg weekly dosing. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Unknown | 2 | 100% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 14 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Other | 4 | 29% |
Student > Master | 2 | 14% |
Researcher | 2 | 14% |
Student > Ph. D. Student | 1 | 7% |
Student > Doctoral Student | 1 | 7% |
Other | 1 | 7% |
Unknown | 3 | 21% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 7 | 50% |
Biochemistry, Genetics and Molecular Biology | 2 | 14% |
Agricultural and Biological Sciences | 1 | 7% |
Pharmacology, Toxicology and Pharmaceutical Science | 1 | 7% |
Unknown | 3 | 21% |