Gaucher Disease Type 1: Long-Term Study
20-year Gaucher Registry study
About the study
This was an observational study where researchers reviewed information that had been collected in the Gaucher Registry for 475 children and adults with Gaucher disease type 1 who were treated with Cerezyme for about 20 years.
Before switching to Cerezyme, some patients in the study received alglucerase—the first ERT developed by Genzyme for people with Gaucher disease type 1.
- Information entry is voluntary and not all the data on every parameter are available for every patient in the Registry. The Gaucher Registry includes patients with a variable range of disease status and management
The following data is for Cerezyme-treated patients in the Registry who had intact spleens.
Cerezyme showed improvements in organ, blood, and bone problems in Gaucher disease type 1 patients over 20 years of treatment. Compared to the start of treatment:
CEREZYME REDUCED THE SIZE OF ENLARGED ORGANS
71% reduction in average spleen volume (out of 74 patients)
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38% reduction in average liver volume (out of 74 patients)
CEREZYME IMPROVED BLOOD PROBLEMS
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2.4 g/dL increase in average red blood cell level (out of 299 patients)
130% increase in average platelet count (out of 293 patients)
CEREZYME REDUCED BONE PROBLEMS
5.9% reduction in the number of patients reporting bone pain
- 56 patients had reported a history of bone pain before treatment
- 49 patients reported bone pain 20 years after first infusion (out of 118 patients)
11.5% reduction in the number of patients reporting bone crisis
- 17 patients had reported a history of bone crisis before treatment
- 4 patients reported bone crisis 20 years after first infusion (out of 113 patients)
The above values improved in patients without spleens as well (except for the spleen volume).
Bone crisis is described as “pain with acute onset which requires immobilization of the affected area, narcotics for the relief of pain, and may be accompanied by 1 or more of the following: periosteal elevation, elevated white blood cell count, fever or debilitation of >3 days” since the last assessment.
The study found that long-term treatment with Cerezyme reduced organ, blood, and bone problems in patients with Gaucher disease type 1.
Key Efficacy and Safety Studies
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Bone Studies
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Pediatric Study
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ERT=enzyme replacement therapy.
Important Safety Information
WARNING: SEVERE ALLERGIC REACTIONS
Allergic reactions, including severe reactions that may be serious or life-threatening (known as anaphylaxis), have occurred during the early course and after repeated treatment with CEREZYME.
Your healthcare professional should initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures. If a severe allergic or anaphylactic reaction occurs, your healthcare professional will immediately stop the infusion and provide appropriate medical treatment.
Seek immediate medical care should symptoms occur.
Cerezyme can cause serious side effects including:
Allergic Reactions (Including Anaphylaxis) and Infusion-Associated Reactions (IARs):
Signs of an allergic reaction reported during or shortly after an infusion included itching, flushing, hives, swelling under the skin, chest discomfort, shortness of breath, coughing, a bluish discoloration of the skin due to diminished oxygen, rapid heart rate, and low blood pressure.
Signs of an infusion reaction included rash, chills, fatigue, infusion-site burning, infusion-site discomfort, or infusion-site swelling, fever, and high blood pressure. Tell your healthcare professional right away if you experience any reactions. Your healthcare professional may slow or stop the infusion or may lower the next dose. Your healthcare professional may decide to give you antihistamine, anti-fever, and/or steroid medications before your infusions and monitor you for new signs and symptoms of a reaction.
Common Side Effects:
- Common side effects reported in adults and children include back pain, chills, dizziness, fatigue, headache, allergic reactions, nausea, fever, and vomiting.
Please see accompanying Full Prescribing Information, including Boxed WARNING.
Cerezyme® (imiglucerase) for injection is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adult and pediatric patients.
Important Safety Information
WARNING: SEVERE ALLERGIC REACTIONS
Allergic reactions, including severe reactions that may be serious or life-threatening (known as anaphylaxis), have occurred during the early course and after repeated treatment with CEREZYME.
Your healthcare professional should initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures. If a severe allergic or anaphylactic reaction occurs, your healthcare professional will immediately stop the infusion and provide appropriate medical treatment.
Seek immediate medical care should symptoms occur.
Cerezyme can cause serious side effects including:
Allergic Reactions (Including Anaphylaxis) and Infusion-Associated Reactions (IARs):
Signs of an allergic reaction reported during or shortly after an infusion included itching, flushing, hives, swelling under the skin, chest discomfort, shortness of breath, coughing, a bluish discoloration of the skin due to diminished oxygen, rapid heart rate, and low blood pressure.
Signs of an infusion reaction included rash, chills, fatigue, infusion-site burning, infusion-site discomfort, or infusion-site swelling, fever, and high blood pressure. Tell your healthcare professional right away if you experience any reactions. Your healthcare professional may slow or stop the infusion or may lower the next dose. Your healthcare professional may decide to give you antihistamine, anti-fever, and/or steroid medications before your infusions and monitor you for new signs and symptoms of a reaction.
Common Side Effects:
- Common side effects reported in adults and children include back pain, chills, dizziness, fatigue, headache, allergic reactions, nausea, fever, and vomiting.
Please see accompanying Full Prescribing Information, including Boxed WARNING.
Cerezyme® (imiglucerase) for injection is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adult and pediatric patients.
Important Safety Information
WARNING: SEVERE ALLERGIC REACTIONS
Allergic reactions, including severe reactions that may be serious or life-threatening (known as anaphylaxis), have occurred during the early course and after repeated treatment with CEREZYME.
Your healthcare professional should initiate CEREZYME in a healthcare setting with appropriate medical monitoring and support measures. If a severe allergic or anaphylactic reaction occurs, your healthcare professional will immediately stop the infusion and provide appropriate medical treatment.
Seek immediate medical care should symptoms occur.
Cerezyme can cause serious side effects including:
Allergic Reactions (Including Anaphylaxis) and Infusion-Associated Reactions (IARs):
Signs of an allergic reaction reported during or shortly after an infusion included itching, flushing, hives, swelling under the skin, chest discomfort, shortness of breath, coughing, a bluish discoloration of the skin due to diminished oxygen, rapid heart rate, and low blood pressure.
Signs of an infusion reaction included rash, chills, fatigue, infusion-site burning, infusion-site discomfort, or infusion-site swelling, fever, and high blood pressure. Tell your healthcare professional right away if you experience any reactions. Your healthcare professional may slow or stop the infusion or may lower the next dose. Your healthcare professional may decide to give you antihistamine, anti-fever, and/or steroid medications before your infusions and monitor you for new signs and symptoms of a reaction.
Common Side Effects:
- Common side effects reported in adults and children include back pain, chills, dizziness, fatigue, headache, allergic reactions, nausea, fever, and vomiting.
Please see accompanying Full Prescribing Information, including Boxed WARNING.
Cerezyme® (imiglucerase) for injection is indicated for the treatment of non-central nervous system (CNS) manifestations of Type 1 or Type 3 Gaucher disease in adult and pediatric patients.