Cerezyme^® (imiglucerase for injection) has been shown to help relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease. However, treatment experiences may vary from one person to the next. Some signs and symptoms may improve within months, while others may take years to get better. In patients with more advanced disease, signs and symptoms may not return to normal, and some may be irreversible.

The following information can help patients and families learn about the kinds of improvements that have been observed after continued use of enzyme replacement therapy (ERT). It is important for patients to discuss their own treatment responses with their physicians. Please see important safety information below.

Areas of improvement

An analysis of a large observational database (Gaucher Registry) was recently published in a professional medical journal. This analysis evaluated the effect of ERT in more than 1,000 patients who had been treated for up to 5 years. This report presents the following findings:

Hemoglobin levels in most patients with anemia increased to normal or near normal levels within the first 6 to 12 months of therapy. Most patients continued to improve or maintained these higher hemoglobin levels when treated for 3 to 5 years.

A majority of patients with moderate thrombocytopenia (low platelet count) displayed normal platelet counts after 2 years of therapy; those with severe thrombocytopenia were less likely to achieve this. Patients without spleens (splenectomized) had a more rapid early increase in platelet count than those whose spleens had not been removed.

Half of the patients with moderate liver enlargement (between 1.25 times normal and 2.5 times normal) showed a decrease in liver volume to less than 1.25 times normal after 2 years of therapy. Liver volume rarely normalized when a patient's pretreatment volume was greater than 2.5 times normal. Some patients may have experienced changes in the liver prior to therapy that cannot be reversed.

After 2 years of therapy, half of the patients with moderate enlargement and 4% of those with severe enlargement showed a decrease in spleen volume to less than 5 times normal. These decreases were sustained through 5 years of treatment, but no further decreases were shown.

Individual results may vary depending on severity of disease and response to treatment. It is important to discuss your own response to therapy with your physician. Please see important safety information below.

The Gaucher Registry

The Gaucher Registry is an observational database that contains clinical information about more than 2,600 patients with Gaucher disease. It includes more than 2,000 patients receiving ERT and documents the effects of the therapy on various signs and symptoms. For more than a decade, the Gaucher Registry has been a global resource to the medical and patient communities.

Physicians participate in the Registry by submitting patient data obtained through routine clinical practice. Since its inception, more than 900 physicians in 43 countries have contributed data to the Gaucher Registry. The resulting collaborations have improved patient care by helping shape management guidelines for Gaucher disease. For more information, you can visit the Gaucher Registry website.

Important Safety Information

Side effects related to Cerezyme® (imiglucerase for injection) 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. Symptoms suggestive of allergic reaction 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. Approximately 15% of patients have developed immune responses (antibodies); periodic monitoring by your physician is suggested. Patients should notify their physician immediately if they experience any side effects with treatment. Cerezyme is available by prescription only. For more information, consult your physician. To learn more, please see the full product information (PDF) or contact Genzyme at 1-800-745-4447.