Understanding Disease Progression
Type 1 Gaucher disease is progressive, meaning that it gets worse over time if it is left untreated. The rate of progression and specific signs and symptoms can vary widely from one person to another. Some people with Type 1 Gaucher disease experience symptoms during childhood, while others remain symptom-free well into adulthood.
Some advanced symptoms that may develop from Gaucher disease include:
- Advanced liver disease
- Advanced bone disease including bone death
- Pulmonary hypertension (high blood pressure in the arteries of the lungs)
- Decreased life expectancy
Cerezyme treatment has been shown to help reduce, relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease.
- In clinical trials, Cerezyme improved anemia (low red blood cell count), thrombocytopenia (low platelet count), reduced spleen and liver size, and decreased cachexia (a wasting syndrome that results in loss of weight, fatigue, weakness and significant loss of body mass that cannot be reversed nutritionally).
- Additionally, it was observed in the Gaucher Registry that Cerezyme therapy helped reduce or reverse many signs and symptoms of Type 1 Gaucher disease, that included low red blood cell count, low platelet count, bone pain, and enlarged spleen and liver.
To learn more about how treatment with Cerezyme can affect Gaucher disease, please see Cerezyme Effects.
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:
- anemia (low red blood cell count)
- thrombocytopenia (low blood platelet count)
- bone disease
- hepatomegaly or splenomegaly (enlarged liver or spleen)
Important Safety Information
Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.
High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.
Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.
Please see Full Prescribing Information (PDF).