LOW-DOSE ENZYME REPLACEMENT THERAPY FOR GAUCHERS-DISEASE - EFFECTS OFAGE, SEX, GENOTYPE, AND CLINICAL-FEATURES ON RESPONSE TO TREATMENT

Although alglucerase therapy has become the treatment of choice for sy mptomatic patients with Gaucher's disease, the low-dose/high-frequency regimen introduced as a means to reduce the high cost of treatment ha s raised major controversy. We evaluated the efficacy and safety of lo w-dose alglucerase in 29 patients with Gaucher's disease who completed 6 to 28 months of therapy. All received intravenous alglucerase at a monthly dose of 30 units/kg, given usually in equal doses 3 times a we ek. All patients responded well to treatment. The hematological improv ement and the reduction in organomegaly were satisfactory. No correlat ion was found between age, sex, genotype, previous splenectomy, or sev erity score index and the response to treatment. Patients with a great er degree of hepatomegaly tended to have a more pronounced decrease in liver size, although this reduction did not reach statistical signifi cance. We confirmed that a low-dose/high-frequency regimen of alglucer ase was as effective as a high-dose/low-frequency protocol in the trea tment of Gaucher's disease, even in the severely ill. Whenever cost is an issue, we recommend using this low-dose regimen.