Type 1 Gaucher disease is a chronic, multisystemic condition that is highly heterogeneous.
Because of this variability, management of patients with Type 1 Gaucher disease cannot be homogeneous. A "one size fits all" approach is generally not useful. Patients with Type 1 Gaucher disease often require personalized approaches to therapy.
A patient-centered, goal-oriented approach is key to working towards optimal outcomes for patients with Type 1 Gaucher disease. Patients may look to a health care provider as a health advocate who can help them understand and manage the physical, mental, and social aspects of the disorder. Each patient requires:
- An individualized treatment plan, including therapeutic goals and individualized dosing, based on initial comprehensive and compartmentalized assessments of skeletal, hematologic, and visceral manifestations
- Support in addressing both mental and physical health issues, potentially including referral to a genetic counselor and/or patient advocacy organization where the patient can share experiences with others who are living with the disease
- Coordinated care, which may require the treating physician to educate other providers, including school nurses for pediatric patients, about Type 1 Gaucher disease.
Because patient needs vary, disease management approaches must be flexible. This section contains ideas and resources for managing many of the challenges of Type 1 Gaucher disease.
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:
- bone disease
- 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).