INDIVIDUALIZED LOW-DOSE ALGLUCERASE THERAPY FOR TYPE-1 GAUCHERS-DISEASE

Previous studies have shown that enzyme supplementation therapy with a lglucerase for type 1 Gaucher's disease is effective at doses of 30-13 0 U/kg per month. Since both the clinical presentation and the respons e to therapy in Gaucher's disease are highly variable, individual dosi ng seems indicated, This notion, as well as the high costs of alglucer ase and the unknown long-term side-effects, led us to investigate the efficacy of an individualised very low dose of alglucerase. Twenty-fiv e adults with symptomatic type 1 Gaucher's disease (thirteen splenecto mised) received alglucerase 1.15 U/kg three times a week (15 U/kg per month). Every 6 months, the dose was halved, maintained, or doubled, a ccording to the response (based on haematological variables and liver and spleen volume). After 6 months of treatment, eighteen (72%) patien ts had a response (seventeen moderate, one good). After 12 months (in nineteen patients) and 18 months (in seven patients), all had sustaine d improvement. Severe splenomegaly resulted in slower haematological r esponses. Our results are similar to those obtained by others with hig her-dose regimens and better than a low-dose regimen of 10 U/kg every 2 weeks. We conclude that very low initial doses of alglucerase, when administered frequently, are effective and cost-saving in the treatmen t of type 1 Gaucher's disease.