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Imiglucerase (Cerezyme®)
Gaucher disease
Adults: Non-neuropathic Gaucher's disease, chronic, symptomatic:
a) Usual dosage, 60 units/kg IV over 1to 2 hr every 2 weeks; may range from 2.5 units/kg 3 times weekly to 60 units/kg once every 2 weeks.
b) Children: safety and effectiveness have not been established in children younger than 2 years of age
a) Non-neuropathic Gaucher's disease, chronic, symptomatic:
1) (2 yr and older) usual dosage, 60 units/kg IV over 1to 2 hr every 2 weeks; may range from 2.5 units/kg 3 times weekly to 60 units/kg once every 2 weeks (91-93).
The absence of an improvement in the visceral, haematological and biochemical markers within 6 months may indicate that a higher dose is required. If bone crises continue, the dose should be increased by at least 50% (92, 93).
For type III gaucher disease some clinician recommend higher dosage: 120 U/kg/2 week (8).
IV infusion over 1-2 hours
Hypotension, tachyarrhythmia, cyanosis, flushing, pruritus, rash, urticaria, shivering, abdominal pain, diarrhea, nausea, backache, angioedema, fatigue, fever, chest discomfort.
1b
8. Centre for Evidence-Based Medicine. Secondary Centre for Evidence-Based Medicine 2009. http://www.cebm.net/index.aspx?o=1025.
91. Bartholomew DW, Batshaw ML, Allen RH, et al. Therapeutic approaches to cobalamin-C methylmalonic acidemia and homocystinuria. J Pediatr 1988;112(1):32-9
92. Ninan TK, Thom H, Russell G. Oral vitamin B12 treatment of cobalamin-responsive methylmalonic aciduria. J Inherit Metab Dis 1992;15(6):939-40
93. Shintaku H. Disorders of tetrahydrobiopterin metabolism and their treatment. Current drug metabolism 2002;3(2):123-31
