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Cerezyme demonstrated improvements in visceral and hematologic parameters over 20 years1
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
In nonsplenectomized patients
-71.1% mean spleen volume
First infusion: 18.2 MN
20 years following first infusion: 4.2 MN (n=74; p<0.0001)
Mean liver volume
In nonsplenectomized patients
-38.0% mean liver volume
First infusion: 1.8 MN
20 years following first infusion: 1.0 MN (n=74; p<0.0001)
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
In nonsplenectomized patients
+2.4 g/dL mean hemoglobin change
First infusion: 11.4 g/dL
20 years following first infusion: 13.8 (n=299; p<0.0001)
In splenectomized patients
First infusion: 11.7 g/dL
20 years following first infusion: 13.4 g/dL
Mean platelet count
In nonsplenectomized patients
+130.4% mean platelet count
First infusion: 91.6 x 103/mm3
20 years following first infusion: 169.1 (n=293; p<0.0001)
In splenectomized patients
First infusion: 229.1 x 109/L
20 years following first infusion: 257.0 x 109/L
Decrease in certain bone parameters related to Gaucher disease type 11,2,a
Patients reporting bone pain
In nonsplenectomized patients
-5.9% patients reporting bone pain in the last month
First infusion: 56 patients reported bone pain
20 years following first infusion: 49 reported bone pain (n=118; p=0.3368)
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
In nonsplenectomized patients
-11.5% patients reporting bone crisis since last assessment
First infusion: 17 patients reported bone crisis
20 years following first infusion: 4 patients reported bone crisis (n=113; p=0.001)
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,a
aThe Cerezyme treatment group from the Gaucher Registry analyses represents patients with Gaucher disease type 1 who received either alglucerase or imiglucerase.
Indication
References: 1. Weinreb NJ, Camelo JS, Charrow J, et al. Mol Genet Metab. 2021;123(2):100-111. 2. Data on file. Sanofi.
