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

Adverse reactions 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 adverse events include nausea, vomiting, abdominal pain, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Adverse events associated with the route of administration include discomfort, pruritus, burning, swelling or sterile abscess at the site of venipuncture. Symptoms suggestive of hypersensitivity include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension. Approximately 15% of patients have developed IgG antibodies; periodic monitoring is suggested. Side effects should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. 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.