Improvements were seen in children treated with Cerezyme over 8 years1
Cerezyme is an established ERT for pediatric patients1,2
Safety and effectiveness have been established in pediatric patients 2 years of age and older with Gaucher disease type 12:
- Evidence from adequate and well-controlled studies of Cerezyme and alglucerase in adults and pediatric patients 12 years of age and older
- Additional data obtained from the medical literature and from postmarketing experience in pediatric patients as young as 2 years of age
- The safety and effectiveness in patients younger than 2 years have not been established
Cerezymea improved long-term visceral and hematologic manifestations in pediatric patients with Gaucher disease type 1 in an 8-year ICGG Gaucher Registry study.1
Study description1
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%).
| Treatment response to Cerezyme was studied for | Data included | Analysis limitations |
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The mean ERT dose was 78.6 U/kg per 4 weeks (SD: 35.0 U/kg per 4 weeks). |
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Studied in the largest reported group of treated pediatric patients (n=884) with Gaucher disease type 1 around the world.1
Cerezymea reduced spleen and liver volumes in children1
Spleen volume
Liver volume
- Approximately half of the treatment effect was achieved after ~1 year of treatment
- Volume reductions continued over the entire 8-year study period
Visceral and hematologic improvements were seen in children with GD1 treated with Cerezymea over 8 years.
Cerezymea improved hematologic parameters in children1
Hemoglobin level
Platelet count
- Improvements in median normalized hemoglobin level were demonstrated during the first year of treatment
- Median hematologic parameters increased to levels similar to those in the normal population after 8 years
Essentially no patients suffered from anemia after 6 years of Cerezyme.1
Cerezymea effect on bone mineral density (BMD) over 12 years in children1
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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, respectively. 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.1
aThe Cerezyme treatment group from the Gaucher Registry analyses represents pediatric patients with Gaucher disease type 1 who received either alglucerase or imiglucerase.
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Indication
References: 1. Andersson H, Kaplan P, Kacena K, Yee J. Pediatrics. 2008;122(6):1182-1190. 2.Cerezyme (imiglucerase). Prescribing information. Genzyme Corporation, Cambridge, MA.
