THERAPY

Low-dose therapy forGaucher'sdisease

A low-dose of alglucerase should be tried initiallyin patients with type 1 Gaucher's disease, according toDr Carla Hollak and colleagues from the Netherlands.'

They report that after 6 months, 18/25 (72%) suchpatients had responded to alglucerase ['Ceredase'] 1.5Ulkg thrice-weekly. The dosage was then halved,maintained or doubled every 6 months according toresponse. Response was sustained between 6 and 18months. By 12 months, 50% of the initialnonresponders had also responded.

Dr Hollak and colleagues say that the response totherapy in Gaucher's disease is highly variable andthere is currently no way to predict which patientswill respond to very low doses of alglucerase. Theysuggest that using very low initial doses is the onlyway to identify patients who will respond to low dosesof this very expensive agent, and thus prevent costlyoverdosing. 1

Although alglucerase is the treatment of choice forGaucher's disease, the recombinant human-derived~-glucocerebrosidase, imiglucerase ['Cerezyme'],may be an attractive cost-effective alternative, say DrAri Zimran and colleagues from Israel. 2

They found low-dose imiglucerase therapy resultedin satisfactory clinical responses, similar to thoseachieved with alglucerase, in 10 patients withGaucher's disease. There was no statisticallysignificant difference between the 2 administrationschedules used in the study: 15 Ulkg once every otherweek or 2.5 Ulkg thrice-weekly.'1. Hollak C, et al. Individualised low-dose alglucerase therapy for type 1Gaucher's disease. Lancet 345: 1474-1478, 10 Jun 19952. Zimran A, et al.Replacement therapy with imiglucerase for type 1 Gaucher's disease. Lancet 345:1479-1480,10 Jun 1995 800314492

0156-270319510991-000151$01.00°Adi. International Limited 1995. All rights reserved INPHARMA~17 Jun 1995

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