Understanding Disease Progression

Type 1 Gaucher disease typically follows a progressive course. The disorder may result in debilitating or disabling symptoms that may become difficult or impossible to reverse. Disease progression can vary widely and have an unpredictable course.1

Physical consequences

  • Bone disease
    • Skeletal damage can cause long-term disability and mortality1
    • Osteosclerosis and vertebral compression may become irreversible if untreated1
    • Bone crises are often attributed to synovitis or osteomyelitis, resulting in missed or delayed diagnosis2
  • Hepatomegaly and Splenomegaly
    • Patients with type 1 Gaucher disease may be at risk for irreversible spleen and liver damage3
    • In rare cases, increased liver volume may result in cirrhosis, portal hypertension, and variceal bleeding3
  • Anemia and Thrombocytopenia
    • Hypersplenism can result in hematologic abnormalities such as anemia and thrombocytopenia1
  • Shortened life expectancy
    • Life expectancy may be decreased by approximately 9 years due to cardiovascular, cerebrovascular, liver and pulmonary diseases5
    • Symptomatic patients may die prematurely from complications related to infection, heart disease, liver disease, bleeding, and severe pulmonary disease5

Indication & Usage

Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

  1. anemia
  2. thrombocytopenia
  3. bone disease
  4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies, and these patients have a higher risk of hypersensitivity reaction. Therefore periodic monitoring is suggested; caution should be exercised in patients with antibodies or prior symptoms of hypersensitivity. Symptoms suggestive of hypersensitivity occurred in 6.6% of patients, and include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension.

Reactions 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 adverse events include nausea, abdominal pain, vomiting, 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 at the site of injection.

To report suspected adverse reactions, contact Genzyme at 800-745-4447, option 2 or FDA at 800-FDA-1088 or http://www.fda.gov/Safety/MedWatch

Please see Full Prescribing Information (PDF).

References

  1. Charrow J, Esplin JA, Gribble TJ, et al. Gaucher disease: recommendations on diagnosis, evaluation, and monitoring. Ach Intern Med. 1998;158:1754-1760.
  2. Kishnani P, Skeletal and Hematologic Pathology of Type 1 Gaucher Disease, Duke University School of Medicine, CME Course, 2008.
  3. Beutler E, Grabowski G. Gaucher disease. In:Scriber CR, Beaudet AL, Sly WS, et al, eds. The Metabolic and Molecular Bases of Inherited Disease. 7th ed. New York, NY: McGraw-Hill; 1995; 2:2641-2661.
  4. Rosenbloom BE, Weinreb NJ, Zimran A, et al. Gaucher Rosenbloom BE, Weinreb NJ, Zimran A, et al. Gaucher 16. Karem E, Elstein D, Abrahamov A, et al. Pulmonary function abnormalities in type 1 Gaucher disease. Eur Respir J 1996;9(2)340-345.
  5. Weinreb NJ, Deegan P, Kacena KA, et al. Life expectancy in Gaucher disease type 1. Am J Hematol. 2008;83(12):896-900.
It has been observed in Registry data that Cerezyme therapy improved bone pain as early as 3 months, decreased bone crisis within 12 months, and improved bone mineral density after 24 months.