Prior to the introduction of enzyme replacement therapy (ERT), a major focus of Gaucher disease management was symptom relief. Treatments included various pain reduction therapies, blood transfusions, orthopedic surgery for bone and joint involvement, and possibly splenectomy.[1] These measures still have a place in the management of Gaucher disease. However, in 1991, Gaucher disease management shifted with the advent of targeted enzyme replacement therapy, developed by Genzyme Corporation in cooperation with the NIH. With the introduction of Cerezyme® (imiglucerase for injection) in 1994 and with its predecessor, clinicians have been able to address the disease process itself, and therefore alleviate and even reverse many effects of Type 1 Gaucher disease.[2]
This section of the website contains a range of information about ERT, as well as links to publications and guidelines that may assist you in treating and monitoring your patients with Type 1 Gaucher disease.
For an overview of Cerezyme’s indications, please see the Cerezyme Profile.
To access information about the steps required to reconstitute and administer Cerezyme, see Administering Cerezyme.
To aid your treatment of patients with Type 1 Gaucher disease, go to Monitoring Guidelines.
For assistance with discussing ERT and Cerezyme with your patients, see Talking with Patients.
To receive the Cerezyme Product Monograph with detailed information about Type 1 Gaucher disease and enzyme replacement therapy, click here to order it by mail.
Important Safety Information
Approximately 15% of patients have developed immune responses (antibodies). These patients have a higher risk of an allergic reaction (hypersensitivity). Use Cerezyme® (imiglucerase for injection) carefully if you have had an allergic reaction to the product in the past. Symptoms suggestive of allergic reaction happened in 6.6% of patients, and include anaphylactoid reaction (a serious allergic reaction), itching, flushing, hives, an accumulation of fluid under the skin, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. Side effects related to Cerezyme administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported side effects include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Because Cerezyme therapy is administered by intravenous infusion, reactions at the site of injection may occur: discomfort, itching, burning, swelling or uninfected abscess. Cerezyme is available by prescription only. For more information, consult your physician. To learn more, please see full product information (PDF), or contact Genzyme at 1-800-745-4447.
References
1. NIH Technology Assessment Panel on Gaucher Disease. Gaucher disease: current issues in diagnosis and treatment. JAMA. 1996;275:548-553.
2. Prescribing Information for Cerezyme® (imiglucerase for injection).
Did You Know...
Gaucher disease is an autosomal recessive disorder defined by the presence of two mutant alleles for the glucocerebrosidase gene.
Genzyme Corporate
Prior to the introduction of enzyme replacement therapy (ERT), a major focus of Gaucher disease management was symptom relief. Treatments included various pain reduction therapies, blood transfusions, orthopedic surgery for bone and joint involvement, and possibly splenectomy.
