Gaucher Disease Type 1: Bone Studies
8-year Gaucher Registry study
This is one of 2 Registry studies that supported the findings of the 48-month bone trial
About the study
In this 8-year observational study, researchers reviewed data from adult patients enrolled in the Gaucher Registry who had a history of lumbar spine bone mineral density (BMD) measurements recorded.
- Up to 8 years of BMD measurements were analyzed for 160 patients who received no treatment and for 342 patients treated with Cerezyme
- 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
Cerezyme impact on spine BMD with long-term treatment
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Long-term impact
- Cerezyme increased the mean lumbar spine Z-score for BMD over 8 years
Did you know?
A way to measure bone health is through bone mineral density, or BMD. A BMD test measures the density and strength of your bones.
4-year Gaucher Registry study
This is one of 2 Registry studies that supported the findings of the 48-month bone trial
About the study
In this 4-year observational study researchers reviewed data from adult and pediatric patients enrolled in the Gaucher Registry who had bone crisis and/or bone pain information for 1 year before treatment, and for each of 3 years after the start of Cerezyme. The year before treatment was used for comparison.
- Bone crisis was defined as an episode of severe bone pain that lasts for more than 3 days that required pain relief, restricted movement, and might be seen with other symptoms, including fever
- Bone pain is defined as pain caused by Gaucher disease that was experienced in the 30-day period prior to a doctor’s patient progress report to the Gaucher Registry
- 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
Cerezyme effect on bone pain and bone crises
| Patients experiencing bone pain | Patients experiencing bone crises | |
| Before treatment | 49% (119 out of 244 patients) | 17% (38 out of 219 patients) |
| After 1 year of ERT | 30% (74 out of 244 patients) | 4.6% (10 out of 219 patients) |
| After 3 years of ERT | 30% (73 out of 244 patients) | 2.7% (6 out of 219 patients) |
Bone crises are often associated with acute bone infarction. They typically begin with regional dull, aching pains that become intense and excruciating over about 3 days.
Key Efficacy and Safety Studies
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Long-Term Study
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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.