Physical examination
Hematologic
Biochemical
Visceral & Pulmonary
Skeletal
Patient-reported Quality-of-Life

Hematologic Assessments

Blood tests to determine hematological parameters used to assess patient condition should include hemoglobin levels and platelet counts. Baseline levels of at least one Gaucher disease biomarker (serum proteins shown to be elevated in Gaucher disease), such as chitotriosidase, angiotensin-converting enzyme (ACE) and tartrate-resistant acid phosphatase (TRAP), should also be measured. Of these, chitotriosidase may be preferred, because it is the most sensitive of the biomarkers. However, for approximately 6% of patients who do not express chitotriosidase, one of the other markers may be used.

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.

Charrow J, Andersson HC, Kaplan P, et al. The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med. 2000;160:2835-2843.

Weinreb NJ, Aggio MC, Andersson HC, et al. Gaucher disease type 1: Revised recommendations on evaluations and monitoring for adult patients. Semin Hematol. 2004;41(4 suppl 5):15-22.