Enzyme replacement therapy for Gaucher's disease

Modified placental human glucocerebrosidase (alglucerase) and recombinant glucocerebrosidase (imiglucerase) are effective means of treating Type 1 Gaucher's disease. Amelioration of hepatosplenomegaly and of haematological manifestations is usually apparent within 6 months. Bone disease responds more slowly but within several years improvement is evident in most patients. Analysis of a large body of data demonstrates that the rate of response of all manifestations of Gaucher's disease is independent of dose over the range of 30 to 260 U/kg body weight per month. Even the response to 15 U/kg per month appears to be equivalent under most circumstances; treatment failures are the same in patients treated with 15, 30 and 130 U/kg per month. Patients with severe manifestations respond more rapidly than those with mild disease, and this, too, is true at all but the 15 U/kg per month dosage level. All available data thus support the administration of no more than 15 to 30 U of alglucerase or imiglucerase per kg/month. Frequent dosing, i.e. three times weekly, appears to be the most effective means of administration.