Cerezyme® (imiglucerase for injection) has been shown to help reduce, relieve or reverse many of the signs and symptoms of Type 1 Gaucher disease. However, treatment experiences may vary from one person to the next. Some signs and symptoms may improve within months, while others may take years to get better. Especially in patients with more advanced disease, signs and symptoms may not return to normal, and some may be irreversible.
The following information can help patients and families learn about the kinds of improvements that have been observed after continued use of Cerezyme. It is important for patients to discuss their own treatment responses with their doctors. Please see important safety information below.
Areas of improvement
In clinical trials, Cerezyme improved anemia (low red blood cell count), thrombocytopenia (low blood 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, an analysis of a large observational database (Gaucher Registry) was recently published in a professional medical journal. This analysis evaluated the effect of enzyme replacement therapy in more than 1,000 patients who had been treated for up to 5 years. This report presents the following observations:
Increased hemoglobin levels
Hemoglobin levels in most patients with anemia increased to normal or near normal levels within the first 6 to 12 months of therapy. Most patients continued to improve or maintained these higher hemoglobin levels when treated for 3 to 5 years.
Increased platelet counts
A majority of patients with moderate thrombocytopenia (low platelet count) displayed normal platelet counts after 2 years of therapy; those with severe thrombocytopenia were less likely to achieve this. Patients without spleens (splenectomized) had a more rapid early increase in platelet count than those whose spleens had not been removed.
Reduced liver size
Half of the patients with moderate liver enlargement (between 1.25 times normal and 2.5 times normal) showed a decrease in liver volume to less than 1.25 times normal after 2 years of therapy. Liver volume rarely normalized when a patient's pretreatment volume was greater than 2.5 times normal. Some patients may have experienced changes in the liver prior to therapy that cannot be reversed.
Reduced spleen size
After 2 years of therapy, half of the patients with moderate enlargement and 4% of those with severe enlargement showed a decrease in spleen volume to less than 5 times normal. These decreases were sustained through 5 years of treatment, but no further decreases were shown.
Individual results may vary depending on severity of disease and response to treatment. It is important to discuss your own response to therapy with your doctor. Please see important safety information below.
The Gaucher Registry
The Gaucher Registry is an observational database that contains clinical information about more than 5,600 patients with Type 1 Gaucher disease. It includes more than 2,000 patients receiving enzyme replacement therapy and documents the effects of the therapy on various signs and symptoms. For more than a decade, the Gaucher Registry has been a global resource to the medical and patient communities.
Doctors participate in the Registry by submitting patient data obtained through routine clinical practice. Since its inception, more than 900 doctors in 43 countries have contributed data to the Gaucher Registry. The resulting collaborations have contributed to improving care by helping shape management guidelines for Gaucher disease. For more information, you can visit the Gaucher Registry website.
Improvement in Bone Symptoms
Type 1 Gaucher Disease patients experience a wide range of bone symptoms, ranging from no symptoms at all to severe pain and disability. It was observed in the Gaucher Registry that Cerezyme therapy decreased the frequency of bone pain and bone crises (severe bone pain), and improved bone mineral density in patients with documented skeletal 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:
- 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).