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Cerezyme demonstrated long-term efficacy in non-CNS manifestations in patients with Gaucher disease type 3.1
The 2-year ICGG Gaucher Registry analysis
Study description1
The 2-year International Collaborative Gaucher Group (ICGG) Gaucher Registry study was an observational analysis of adult and pediatric patients (7 weeks to 54 years old) with Gaucher disease type 3 who were treated with Cerezyme.
Patients were included in the analysis if they had clinical data at baseline and follow-up assessments for one or more of the following:
- Spleen volume
- Liver volume
- Hemoglobin level
- Platelet count
- Height Z-score
Information is voluntary and not all the data on every parameter are available for every patient in the registry. The ICGG Gaucher Registry includes patients with a variable range of disease status and management.
Cerezyme demonstrated improvements in visceral, hematologic, and growth parameters in patients with Gaucher disease type 31
An observational study of patients with Gaucher disease type 3 treated with Cerezyme over 2 yearsa
Visceral parameters
Improvements in mean spleen volume from baseline
Improvements in mean liver volume from baseline
Hematologic parameters
Improvement in mean hemoglobin levels
Baseline: 10 g/dL
Mean change after 2 years: +1.8 g/dL (n=118)
Improvements in mean platelet count
Baseline: 149 × 103/mm3
Mean change after 2 years: +105 × 103/mm3 (n=116)
Pediatric growth
Improvements in mean height Z-score from baseline
MN=multiples of normal.
aThe 2-year summaries include measurements within 1-3 years after treatment initiation due to the lack of predefined data collection timepoints within the registry.1
Cerezyme demonstrated improvements in visceral, hematologic, and growth parameters over 2 years in patients with Gaucher disease type 3.1
Indication
Reference: 1. Cerezyme (imiglucerase). Prescribing information. Genzyme Corporation, Cambridge, MA.
