XXXX . A pharmacoeconomic appraisal of its use in the treatment of @entity1120 .

@entity1 @entity15675 @entity828 @entity79 @entity1120
@entity15675 is a modified form of @entity1 placental glucocerebrosidase used as enzyme replacement therapy for @entity1 with @entity1120 , in whom functional glucocerebrosidase is deficient. @entity15675 has provided a breakthrough in treatment for @entity1 with this relatively rare disease. With @entity15675 infusions typical disease manifestations are ameliorated or normalised: @entity828 is reduced, haematological parameters improve, and @entity1 experience an increased quality of life usually within 4 to 6 months of treatment. Parameters of @entity79 also respond, but generally over a longer period of treatment. @entity15675 is well tolerated by @entity1 and adults, with few adverse effects reported. Seroconversion occurs in approximately 15% of @entity1 on high-dose therapy, but does not appear to affect the efficacy of treatment. Several dosage regimens have been used to deliver @entity15675 , 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. @entity1 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 @entity15675 is  US3.70 per unit (1994 US dollars); thus, a dosage regimen of 60 IU/kg bodyweight administered every 2 weeks for a @entity1 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 @entity1 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 @entity1 productivity and reduced palliative treatments remain unresolved. Although some @entity1 may obtain increased benefit from high-dosage regimens, the very high cost may preclude general use of these regimens. Healthcare resources consumed by @entity15675 therapy represent a large opportunity cost for other therapeutic areas.(ABSTRACT TRUNCATED AT 400 WORDS)
