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Cerezyme demonstrated improvements in visceral and hematologic parameters over 20 years.1
The 20-year ICGG Gaucher Registry analysis
Study description1
The 20-year International Collaborative Gaucher Group (ICGG) Gaucher Registry study is a retrospective, observational, single-arm study of patients with Gaucher disease type 1 who were treated with Cerezymea for about 20 years.
Patient population |
Patient stratification |
Definitions |
Analysis limitations |
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Within the database, patients (n=475) were identified with clinical data at baseline, 10 years, and 20 years of follow-up for:
| Patients were stratified into nonsplenectomized (n=310) and splenectomized (n=165) groups. |
Bone pain was defined as being present if the patient reported this event as occurring in the past month before the medical visit. Bone crisis was defined as pain with acute onset that requires immobilization of the affected area, narcotics for the relief of pain, and may be accompanied by 1 or more of the following: periosteal elevation, elevated white blood cell count, fever, or debilitation >3 days since the last assessment. | Information entry is voluntary and not all the data on every parameter are available for every patient in the registry. ICGG Gaucher Registry includes patients with a variable range of disease status and management. |
aThe Cerezyme treatment group from the ICGG Gaucher Registry analyses represents patients with Gaucher disease type 1 who received either alglucerase or imiglucerase.
Improvements in visceral parameters1,2
Mean spleen volume
71.1% reduction in mean spleen volume in nonsplenectomized patients
First infusion: 18.2 MN
20 years following first infusion: 4.2 MN
Mean liver volume
38% reduction in mean liver volume in nonsplenectomized patients
First infusion: 1.8 MN
20 years following first infusion: 1.0 MN
In splenectomized patients
First infusion: 2.3 MN
20 years following first infusion: 1.0 MN
Improvements in hematologic parameters1,2
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Mean hemoglobin level
2.4 g/dL increase in mean hemoglobin level in nonsplenectomized patients
First infusion: 11.4 g/dL
20 years following first infusion: 13.8 g/dL
In splenectomized patients
First infusion: 11.7 g/dL
20 years following first infusion: 13.4 g/dL
Mean platelet count
130.4% increase in mean platelet count in nonsplenectomized patients
First infusion: 91.6 × 103/mm3
20 years following first infusion: 169.1 × 103/mm3
In splenectomized patients
First infusion: 229.1 × 109/L
20 years following first infusion: 257.0 × 109/L
Decrease in certain bone parameters related to Gaucher disease type 11,2
Patients reporting bone pain
5.9% reduction in nonsplenectomized patients reporting bone pain
First infusion: 56 patients reported bone pain
20 years following first infusion: 49 reported bone pain
In splenectomized patients
First infusion: 41 patients reported bone pain
20 years following first infusion: 26 reported bone pain
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Patients reporting bone crisis
11.5% reduction in nonsplenectomized patients reporting bone crisis
First infusion: 17 patients reported bone crisis
20 years following first infusion: 4 patients reported bone crisis
In splenectomized patients
First infusion: 22 patients reported bone crisis
20 years following first infusion: 0 patients reported bone crisis
MN=multiples of normal.
Cerezyme demonstrated improvements in visceral, hematologic, and certain bone parameters over 20 years.1
Indication
References: 1. 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. 2. Data on file. Sanofi.
