Improvements were seen in children treated with Cerezyme over 8 years.1
Cerezyme is an established ERT for pediatric patients1,2
Safety and effectiveness have been established in pediatric patients1-4:
- Evidence from adequate and well-controlled studies of Cerezyme and alglucerase in adults and pediatric patients
- Additional data obtained from the medical literature and from postmarketing experience in pediatric patients
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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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 in reducing liver and spleen size 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 Cerezyme1
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.
Indication
References: 1. Andersson H, Kaplan P, Kacena K, Yee J. Eight-year clinical outcomes of long-term enzyme replacement therapy for 884 children with Gaucher disease type 1. Pediatrics. 2008;122(6):1182-1190. 2. Cerezyme (imiglucerase). Prescribing information. Genzyme Corporation, Cambridge, MA. 3. Weinreb NJ, Camelo JS Jr, Charrow J, et al. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Mol Genet Metab. 2021;132(2):100-111. 4. Charrow J, Dulisse B, Grabowski GA, Weinreb NJ. The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease. Clin Genet. 2007;71(3):205-211.
