Imiglucerase in Gaucher's Disease

Abstract

Imiglucerase is an analogue of the human enzyme β-glucocerebrosidase that is produced by recombinant DNA technology. It is used as a replacement enzyme for the treatment of Gaucher’s disease, a rare genetic disorder that causes the functional deficiency of glucocerebrosidase enzyme in lysosomes resulting in the accumulation of lipid substance in lysosomes of macrophage of multiple organs. Mechanism of action of imiglucerase is the hydrolysis of complex lipid accumulated in patients with Gaucher’s disease The drug is indicated to use as enzyme replacement therapy for pediatric and adult patients with Gaucher’s disease type 1. The recommended dosage is 30-60 units per kg of body weight infused within 1-2 hours every two weeks. Imiglucerase is listed in the National List of Essential Medicines B.E.2563 category E(2). This category includes medicines that are labeled for specific patients with specific indications, or prescribed by specialist physicians, or produced by advanced technology, and costly. The drug admixing is very important because it must be compounded by personnels who have knowledge and technique to avoid degradation. One technique is to reconstitute by adding sterile water for injection slowly drop-wise, down inside the wall of each vial, instead of shaking vigorously. It is found that the common adverse reactions (13.8%) are related to imiglucerase administration, frequency or route of administration. Some of adverse reactions are discomfort, pruritus, swelling, burning or abscess at the site of venipuncture.