Alglucerase is a modified form of human placental glucocerebrosidase used as 
enzyme replacement therapy for patients with Gaucher's disease, in whom 
functional glucocerebrosidase is deficient. Alglucerase has provided a 
breakthrough in treatment for patients with this relatively rare disease. With 
alglucerase infusions typical disease manifestations are ameliorated or 
normalised: hepatosplenomegaly is reduced, haematological parameters improve, 
and patients experience an increased quality of life usually within 4 to 6 
months of treatment. Parameters of bone disease also respond, but generally over 
a longer period of treatment. Alglucerase is well tolerated by children and 
adults, with few adverse effects reported. Seroconversion occurs in 
approximately 15% of patients on high-dose therapy, but does not appear to 
affect the efficacy of treatment. Several dosage regimens have been used to 
deliver alglucerase, and the comparative benefits of these remain controversial. 
High-dose regimens of 60 IU/kg bodyweight administered every 2 weeks are clearly 
effective; however, smaller dosages given more frequently are also effective and 
incur a greatly reduced acquisition cost. Patient responses are variable, and 
the dosage regimen should be tailored to individual needs. Dosage regimens may 
be considerably reduced for the maintenance phase of treatment, but clinical 
experience is as yet insufficient to establish the minimum dosages required in 
the long term. Acquisition cost of alglucerase is $US3.70 per unit (1994 US 
dollars); thus, a dosage regimen of 60 IU/kg bodyweight administered every 2 
weeks for a patient weighing 70kg costs $US404,040 per year. The minimal costs 
per quality-adjusted life year saved (QALY) have been estimated for 3 dosage 
regimens over a 10-year period. Cost per QALY was $US147,000 for 60 IU/kg 
bodyweight administered every 2 weeks, $US75,000 for 30 IU/kg every 2 weeks, and 
$US49,000 for 2.3 IU/kg administered 3 times per week. These costs were 
calculated assuming immediate death with no treatment, which suggests that the 
actual costs per QALY for most patients with type 1 or 3 disease are likely to 
be much higher. Drug administration costs may become a significant part of the 
cost during maintenance therapy; in addition, possible cost savings due to 
increased patient productivity and reduced palliative treatments remain 
unresolved. Although some patients may obtain increased benefit from high-dosage 
regimens, the very high cost may preclude general use of these regimens. 
Healthcare resources consumed by alglucerase therapy represent a large 
opportunity cost for other therapeutic areas.(ABSTRACT TRUNCATED AT 400 WORDS)