Cerezyme

Common name: Imiglucerase

Trade name: Cerezyme

All names: Imiglucerase, Cerezyme, Imiglucerase, Cerezyme

Indications:

For the treatment of type I Gaucher disease (Gaueher disease) disease) leading to anemia, thrombocytopenia, bone disease, and hepatosplenomegaly. Newer complementary clinical applications include long-term enzyme replacement therapy in patients diagnosed with type III metabolic disease and significant non-neurological manifestations.

Treatment of chronic neuropathy type (type III) Gaucher disease.

Usage and dosage:

Intravenous infusion, the infusion time is 1-2 hours. Dosage should be adjusted based on individual patient conditions.

The initial dose range is 2.5U/kg, 3 times a week, to 60U/kg, once every 2 weeks. If the treatment goal is achieved after 2 years, the dose can be changed to 45U/Kg. 60 U/kg every 2 weeks is the dose with the most data.

Adverse reactions:

>10%:

Immunology: Development of IgG antibodies (15%)

1% - 10%:

Cardiovascular disease: chest discomfort (<2%), flushing (<2%), hypotension (<2%), fast heartbeat (<2%)

Central nervous system: chills (<2%), dizziness (<2%), fatigue (<2%), headache (<2%)

Dermatology: pruritus (<2%), rash (<2%), urticaria (<2%)

Gastrointestinal tract: abdominal pain (<2%), diarrhea (<2%), nausea (<2%), vomiting (<2%)

Hypersensitivity: anaphylaxis (7%), angioedema (< 2%)

Neuromuscular and skeletal: back pain (<2%)

Respiratory: cough (<2%), cyanosis (<2%), dyspnea (<2%)

Other: fever (<2%)

<1%:

Post-marketing or case Reports: burning sensation at injection site, discomfort at injection site, itching at injection site, non-immune allergic reaction, pneumonia, pulmonary hypertension, sterile abscess at injection site, swelling at injection site

Contraindications:

Contraindicated for those allergic to this product.

Precautions:

Allergic reactions: have been reported (<1%). Most patients continued to receive conditioning (antihistamines or corticosteroids) with a slower infusion rate. In the event of a severe reaction, treatment should be discontinued immediately and appropriate treatment initiated. Caution should be used in patients who have been previously allergic to, treated with, or have developed antibodies to alglucosidase. It is contraindicated in patients with severe allergy to imiglucerase.

Antibody formation: IgG antibodies are reported to occur in approximately 15% of patients and are observed within 6 months of initiation of treatment; antibodies are rarely produced after 12 months of treatment; may increase risk of allergic reactions.

Pulmonary hypertension/pneumonitis: Observed during treatment; as this is a complication of Gaucher disease, causality has not been established. Patients with respiratory symptoms without fever should be evaluated for pulmonary hypertension.

Polysorbate 80: Some dosage forms may contain polysorbate 80 (also called Tween). Some people may experience an allergic reaction, usually a delayed reaction, after exposure to medicines containing polysorbate 80. Thrombocytopenia, ascites, worsening of pulmonary function, and renal and hepatic failure have been reported in premature infants receiving parenteral products containing polysorbate 80.

Fetal toxicity: Use with caution in pregnant and lactating women.

Storage:

Store at 2-8°C in the dark.

Mechanism of action:

This product is an analog of glucocerebrosidase produced by genetic recombination technology. It is a glycoprotein with 497 amino acids and contains 4 N-ring-linked glycosylation sites. Gaucher disease is a rare metabolic genetic disease caused by congenital deficiency of glucocerebrosidase. The lack of this enzyme causes the accumulation of glucocerebrosidase in macrophages and becomes Gaucher cells. The main symptoms of Gaucher disease are anemia, thrombocytopenia, bone pain, and hepatosplenomegaly. This product catalyzes the hydrolysis of glucocerebroside and degrades it into glucose and ceramide, thereby improving the symptoms of anemia and thrombocytopenia and reducing the size of the liver and spleen.

Safety and efficacy:

Gaucher's disease is characterized by a defect in b-glucocerebrosidase activity, leading to the accumulation of glucocerebroside in tissue macrophages, causing them to become hypertrophied, often in the liver, spleen and bone marrow, and occasionally in the lungs, kidneys and intestines. Secondary hematological consequences include severe anemia and thrombocytopenia, as well as characteristic progressive enlargement of the liver and spleen, and skeletal complications. Injectable imiglucerase catalyzes the hydrolysis of glucocerebroside into glucose and ceramide.

In clinical trials, imiglucerase injection can improve anemia and thrombocytopenia, shrink the liver and spleen, and improve cachexia. The clinical effect is similar to that of alglucosidase injection.

Research shows that after 6 months of using imiglucerase to treat type I Gaucher disease, the number of people reporting bone pain decreased by 37.5%, which can significantly improve patients’ quality of life and growth and development. After 24 months of treatment with imiglucerase, the spleen volume was reduced by 50.9%, the liver volume was reduced by 30.8%, the hemoglobin increased by an average of 2.4g/dL, the platelet count increased by an average of 99.8%, and the organ involvement and blood image were significantly improved.