Disease Management in the Treatment of Type I Gaucher Disease

Gaucher disease is a heterogeneous condition that affects multiple organ systems. Because of this heterogeneity, each patient requires an individualized treatment plan based on initial comprehensive and compartmentalized assessments, development of therapeutic goals for each compartment based on the initial assessments and estimated risk for disease progression, individualized dosing to achieve therapeutic goals, regular monitoring of compartments to determine attainment of goals, and adjustment of dosing as necessary for attainment and maintenance of goals. Goals for each compartment depend on factors such as the patient’s age, baseline state, splenectomy status, and presence of irreversible pathology.

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.

1. Weinreb N. Introduction. Advances in Gaucher disease: therapeutic goals and evaluation and monitoring guidelines. Semin Hematol. 2004;41(4 Suppl 5):1-3.