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

Bone Disease

In terms of bone disease, the main goal for children and adults with non-neuronopathic Gaucher disease is to retain skeletal function by preventing the onset of new skeletal complications and to relieve and prevent recurrence of acute and chronic bone pain and bone crises.

Children and adults have differences in skeletal physiology that must be taken into account when therapeutic goals for skeletal pathology are set. For children, the aim should be to attain normal or ideal peak skeletal mass and prevent skeletal pathology. In adults, the aim is to maintain the skeleton and preserve function.

The specific therapeutic goals for bone disease are to lessen or eliminate bone pain and bone crises within 1 to 2 years of treatment. The onset or development of bone pathology during enzyme replacement therapy should prompt investigation for loss of mechanical bone integrity requiring changes in therapy with orthopedic intervention. Patients with advanced bone disease may also require orthopedic intervention, physiotherapy, or other pharmacologic treatment.

For more information about bone disease, including osteonecrosis, subchondral joint collapse, skeletal mass, cortical bone mineral density, or trabecular bone mineral density 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.