Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.

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Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P

Guidance on the use of miglustat for treating patients with type 1 Gaucher disease.

Am J Hematol. 2005 Nov;80(3):223-9.

PubMed ID

16247743 [ View in PubMed

]

Abstract

Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder due to an autosomal recessive deficiency of glucocerebrosidase. Clinical manifestations include anemia, thrombocytopenia, hepatosplenomegaly, and bone and pulmonary disease. Intravenous enzyme replacement (ERT) with imiglucerase is the accepted standard for treatment of symptomatic patients. More than 3,500 patients worldwide have received ERT with well-documented beneficial effects on the hematological, visceral, skeletal, and pulmonary manifestations, and with resultant improvement in health-related quality of life. Miglustat, an imino sugar that reversibly inhibits glucosylceramide synthase and reduces intracellular substrate burden, is an oral treatment for patients with type 1 GD that was recently approved in the United States for symptomatic patients with mild to moderate clinical manifestations for whom ERT is not an option. Because responses to miglustat are slower and less robust than those observed with ERT, and because miglustat is associated with significant side effects, clinicians who care for patients with GD should become familiar with the limited indications for miglustat use and the circumstances when it may be prescribed appropriately. This review article and position statement represents the current opinion of American physicians with extensive expertise in GD regarding patient management in the context of the availability of standard imiglucerase treatment and the recent introduction of miglustat.

DrugBank Data that Cites this Article

Drugs

Miglustat

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Miglustat	Ceramide glucosyltransferase	Protein	Humans	Yes	Inhibitor	Details