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Classification: Treatment of Gaucher's disease
Action/Kinetics: Gaucher's disease is a rare congenital disorder of lipid metabolism where there is a deficiency in beta-glucocerebrosidase leading to an accumulation of lipid glucocerebroside in the liver, spleen, and bone marrow. Symptoms include an enlarged spleen, increased skin pigmentation, bone lesions, severe anemia, and thrombocytopenia. Alglucerase, which is a modified form of beta-glucocerebrosidase, is derived from human placental tissue; it catalyzes the hydrolysis of the glycolipid glucocerebroside to glucose and ceramide, which is part of the normal degradation pathway for membrane lipids. Following IV infusion, steady-state enzyme levels were observed in 60 min. t 1/2: 3.6-10.4 min.
Uses: Chronic enzyme replacement in clients with confirmed diagnosis of type I Gaucher's disease who meet the following criteria: moderate to severe anemia, thrombocytopenia with bleeding tendency, significant hepatomegaly or splenomegaly, and bone disease.
Special Concerns: Use with caution during lactation. Since alglucerase is derived from human placental tissue, there is always the risk of some viral contamination; however, the drug product has been found to be free from hepatitis B surface antigen and for antigens of HIV (HIV-1).
Side Effects: Nausea, vomiting, chills, abdominal discomfort, slight fever.
How Supplied: Injection: 10 U/mL, 80 U/mL