Mol Genet Metab. 2009 Feb;96(2):73-6. Epub 2008 Dec 10.

Enzyme replacement therapy in pediatric patients with Gaucher disease: what should we use as maintenance dosage?

Brunel-Guitton C, Rivard GE, Galipeau J, Alos N, Miron MC, Therrien R, Mitchell G, Lapierre G, Lambert M.

Source

Division of Medical Genetics, Department of Pediatrics, CHU Sainte-Justine, University of Montreal, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, Que. H3T1C5, Canada.

Abstract

INTRODUCTION:

No consensus exists on the minimal dose of enzyme replacement therapy (ERT) effective to maintain therapeutic goals in pediatric Gaucher patients.

OBJECTIVE:

Evaluate the efficacy of low dosage ERT to maintain treatment goals.

RESULTS:

Six patients had a maintenance dose of 30-35U/kg/month. All patients, with the exception of one L444P/L444P homozygote, maintained therapeutic goals.

DISCUSSION:

A low maintenance dose may be adequate in most pediatric patients. L444P homozygotes may require a higher maintenance dosage.

PMID:: 19083253; [PubMed - indexed for MEDLINE]

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Glucosylceramidase

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