A drug that is actively used to treat patients with the first or third type of Gaucher disease. Used for prolonged enzyme replacement therapy in patients with and without neuropathic manifestations.
Indications of the cerezima
Among the main symptoms of the disease, which can be the basis for prescribing the drug, it is worth mentioning the following:
- Anemia (except for iron deficiency anemia).
- Diseases of bone tissue (except for diseases that are accompanied by a deficiency of vitamin D).
- Splenomegaly or hepatomegaly.
The product is available in the form of a lyophilizate, with which you can prepare two solutions for infusions (200 and 400 units). In the pharmacy, the drug can be purchased in small bottles of 20 ml. The drug contains an active component of 200 (400) ED imiglucerase. This glucocerebrosidase in a modified form, which is created with the help of recombinant DNA technology from the ovaries of Chinese hamsters.
Ceramide and glucose are catalyzed by imiglucerase due to the hydrolysis of gluco-lipid glucocerebroside through normal degradation of membrane lipids. In general, the formation of glucocerebroside occurs through hematopoietic exchange in cells. In Gaucher's disease, the patient exhibits a deficiency in the activity of such an enzyme as β-glucocerebrosidase. Because of this, macrophages of the tissues accumulate glucocerebroside lipids. They are called "Gaucher cells" and can be completely filled with this lipid.
As a rule, Gaucher cells can be found in the bone marrow, liver, intestine, spleen, kidneys and even lungs. Secondary manifestations of the disease experts consider anemia and thrombocytopenia, which lead to severe hepatosplenomegaly. Perhaps the most serious complications in Gaucher's disease are complications in the development of the skeleton (osteonecrosis, remodeling deficiency, osteopenia).
After intravenous infusion of four doses of the drug in a patient an hour later, a stable activity of the enzymes is achieved. After the procedure, the activity of enzymes in the plasma begins to decrease after 10 minutes. Some experts believe that the duration of the infusion and the dosage of the drug in no way affect the indicators, but clinical studies were conducted on a small number of patients.
Use of the cerezima during pregnancy
At the moment, there is not enough information about how the drug Cerezyme affects the health of pregnant women and the development of the fetus. That is why its use in treatment during pregnancy is not recommended. Only in case of emergency, Cerezim can be appointed by the attending physician.
Dosing and administration
Gaucher's disease is multisystemic and heterogeneous in nature, so only the attending physician can determine the correct dosage after examining the patient. Infusion is carried out intravenously with the help of a dropper one or two hours. The rate of administration of the drug should not exceed 1 unit / kg / min. The first two weeks of the dose is 60 U / kg. This dose is administered once. Treatment is worth continuing if there has been no allergic reaction to the remedy. Standard drug is administered once every two weeks, but the dosage is gradually increased.