Gaucher Disease Type 1: Pediatric Study
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
This was an observational study where researchers reviewed data from 884 pediatric patients with Gaucher disease type 1 who were enrolled in the Gaucher Registry. The study patients had intact spleens and were receiving alglucerase (22.4%) or Cerezyme (77.6%).
- Data up to 8 years after the first treatment infusion or 18 years of age were analyzed
- BMD data were analyzed until 12 years after the first treatment infusion
- 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
Long-term Cerezyme treatment for children with Gaucher disease type 1 improved organ and blood problems, and had an impact on bone problems
CEREZYME REDUCED THE SIZE OF ENLARGED ORGANS OVER 8 YEARS
Median spleen volume at baseline was 23 MN and 4.8 MN after 8 years on Cerezyme (out of 458 patients)
- About half the treatment effect was seen within 1 year of treatment
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Median liver volume at baseline was 2.0 MN and 1.1 MN after 8 years on Cerezyme (out of 420 patients)
- About half the treatment effect was seen within 1 year of treatment
CEREZYME IMPROVED BLOOD PROBLEMS OVER 8 YEARS
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2 g/dL increase in median red blood cell levels (out of 771 patients)
- Essentially no children had anemia after 6 years
- Red blood cell levels increased to normal levels after 8 years
Baseline platelet count was 98 x 103/mm3 and 171 x 103/mm3 after 8 years on Cerezyme (out of 768 patients)
CEREZYME EFFECT ON BONE MINERAL DENSITY (BMD) OVER 12 YEARS
Long-term use of Cerezyme showed an impact on BMD in children
CEREZYME EFFECT ON HEIGHT GROWTH OVER 8 YEARS
Long-term use of Cerezyme improved height growth for children with Gaucher disease type 1. After 8 years, the patients' median height was not substantially different from the median for the healthy population.
Key Efficacy and Safety Studies
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Long-Term Study
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Bone Studies
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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.