The following recommended goals are based upon composite information obtained from the Gaucher Registry. Patient responses may vary and physicians should use their own medical judgment in applying these recommendations in the care of their patients.

Bone Disease
Anemia
Thrombocytopenia
Hepatomegaly
Splenomegaly

Anemia

Anemia is one of the common manifestations of Gaucher disease, with the most frequent presentation among anemic patients being fatigue, although older patients may also present with dyspnea or angina.

Data from the Gaucher Registry reflect that anemia responds rapidly (ie, within 6 months) to enzyme replacement therapy. Therefore, the therapeutic goals for treating anemia in patients with Gaucher disease are to:

increase hemoglobin levels to more than 11.0 g/dL for women and children and more than 12.0 g/dL for men within 12 to 24 months (in the absence of iron deficiency);

eliminate the need for blood transfusions;

reduce the symptoms of anemia; and

maintain the improvement in hemoglobin values achieved.

In severely affected patients (Hb <8.0 g/dL), although significant increases in hemoglobin from baseline are expected during the first 6 months of therapy, mild anemia may persist, especially among those with hypersplenism associated with massive splenomegaly. In these cases, comprehensive hematologic evaluation should be undertaken to exclude coexistent etiologies, including iron deficiency, vitamin B deficiency, anemia of chronic disease and, especially in older individuals, myelodysplasia or immunoproliferative disorders. A decrease in hemoglobin concentration (acute or gradual) should prompt investigation to rule out blood loss due to an intercurrent condition.

For more information about anemia, please contact Genzyme Medical Information at 1-800-745-4447.

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.

Weinreb NJ, Charrow J, Andersson HC, et al. Effectiveness of enzyme replacement therapy in 1,028 patients with type 1 Gaucher disease after 2-5 years of treatment: a report from the Gaucher Registry. Am J Med. 2002; 113:112-119 .

Rosenthal DI, Doppelt SH, Mankin HJ, et al. Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. Pediatrics. 1995;96:629-637.

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.

Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol. 2004;41(4 suppl 5):4-14.