Cerezyme* improved long-term visceral and hematologic manifestations in pediatric patients in an 8-year ICGG Gaucher Registry study7
This observational study used data derived from the International Collaborative Gaucher Group (ICGG) Gaucher Registry submitted between 1991 and January 2006. Data were retrospectively analyzed for all patients (n=884) with Gaucher disease type 1 who had intact spleens and were receiving alglucerase (22.4%) or Cerezyme (77.6%).Read more...
This observational study used data derived from the International Collaborative Gaucher Group (ICGG) Gaucher Registry submitted between 1991 and January 2006. Data were retrospectively analyzed for all patients (n=884) with Gaucher disease type 1 who had intact spleens and were receiving alglucerase (22.4%) or Cerezyme (77.6%).
The mean ERT dose was 78.6 U/kg per 4 weeks (SD: 35.0 U/kg per 4 weeks)
Treatment response to Cerezyme was studied for:
Improvements were seen in children treated with Cerezyme* over 8 years
0% of children on Cerezyme had anemia after 6 years
The DXA Z-scores for patients in the 95th, 75th, 50th, 25th, and 5th percentiles at first infusion were 1.22, 0.49, -0.35, -1.19, and -1.93, respectively. At 12 years, the DXA Z-scores for patients in the 95th, 75th, 50th, 25th, and 5th percentiles were 1.87, 1.13, 0.29, -0.55, and -1.29. N=127; BMD Z-score intercepts and slopes (change over time) were monitored for 12 years.
Timing of pediatric treatment should consider that most bone mineral is accrued in the first 2 decades of life and BMD peaks in the third decade
Cerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:
Warnings and Precautions:
Hypersensitivity and Infusion-Associated Reactions: Hypersensitivity reactions, some of which are serious and include anaphylaxis have been reported. Hypersensitivity and other infusion-associated reactions have been reported during or shortly after infusion and include pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, cough, cyanosis, tachycardia, and hypotension. Patients with antibody to imiglucerase have a higher risk of hypersensitivity reactions. Not all patients with symptoms of hypersensitivity have detectable IgG antibody. Consider periodic monitoring during the first year of treatment for IgG antibody formation.
If a severe hypersensitivity reaction occurs, discontinue Cerezyme treatment and initiate appropriate medical treatment. Consider the risks and benefits of readministering Cerezyme to individual patients following a severe reaction. If Cerezyme is readministered, consider reducing the rate of infusion, pretreat with antihistamines and/or corticosteroids, and monitor patients for new signs and symptoms of a severe hypersensitivity reaction.
Adverse reactions reported in adults include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting.
Adverse reactions reported in pediatric patients 2 years of age and older are similar to adults.
Approximately 15% of patients treated and tested to date have developed IgG antibody to Cerezyme during the first year of therapy. Patients who developed IgG antibody did so largely within 6 months of treatment and rarely developed antibodies to Cerezyme after 12 months of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity. Patients with antibody to Cerezyme have higher risk of hypersensitivity reaction.
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To report SUSPECTED ADVERSE REACTIONS, contact Sanofi Medical Information at 1-800-745-4447, Option 2.