- Article
- Source: Campus Sanofi
- 9 Feb 2024
Cerezyme® evidence
Cerezyme® has shown both early and long-term improvements in visceral and haematological parameters1,2
In a 9 month, non-inferiority, phase 3 study, Cerezyme® demonstrated improvements from baseline:2
- 47.1% reduction in spleen volume with Cerezyme® from baseline
- 21.4% reduction in liver volume with Cerezyme® from baseline
- 25.4 g/dL increase in haemoglobin with Cerezyme® from baseline
- 43.5% increase in platelet count with Cerezyme® from baseline
In a 10 year retrospective study involving 757 patients, those treated with Cerezyme® showed:1
- 73% reduction in spleen volume vs. baseline (19.4±16.2 multiples of normal (MN) vs. 5.2±3.6 MN; p<0.0001; n=107)
- 44% reduction in liver volume vs. baseline (1.8±0.8 MN vs. 1.0±0.2 MN; p<0.0001; n=105)
- 2.4 g/dL increase in haemoglobin level vs. baseline (11.2±1.7 g/dL vs. 13.6±1.6 g/dL; p<0.0001; n=376)
- 75% increase in platelet count vs. baseline (95.3±52.8 x 103/mm3 vs. 166.66±59.7 x 103/mm3; p<0.0001; n=397)
Skeletal complications of Gaucher disease are progressive and therefore, early initiation of Cerezyme® could improve bone outcomes for patients3
Bone involvement is the principal cause of pain, disability and reduced quality of life in Gaucher disease patients of all ages.4 The pathophysiology responsible for bone involvement in Gaucher disease is not completely understood.4
Gaucher disease affects the bone marrow and mineralised components of bone, causing:4
Bone pain and bone crisis
Bone marrow infiltration and plasma cell dyscrasias
Reduced bone mineral density (BMD)
Modelling and remodelling abnormalities of bones, resulting in developmental changes, loss of bone mineral (osteopenia/osteoporosis), cortical thinning, lytic lesions, and fragility fractures
Increased risk of avascular necrosis (AVN)
A major, irreversible complication of type 1 Gaucher disease and can lead to joint destruction, the need for joint replacement surgery, and chronic disability.3
Cerezyme® decreases the occurrence of bone pain and bone crisis vs. baseline5,6
Cerezyme® significantly decreased the number of patients reporting bone pain and bone crises vs. baseline.1
Cerezyme® improved BMD within 12 months vs. baseline5
Patients on Cerezyme® achieved near-normal BMD after 8 years.7 The impact of Cerezyme® on long-term improvements in BMD was dose-related.7
Initiation of Cerezyme® within 2 years of diagnosis was shown to significantly reduce the risk of AVN, compared with treatment initiated at least 2 years after diagnosis.3
Cerezyme® improved visceral, haematological, certain bone parameters, and normalised growth and puberty vs. baseline8–11
Published paediatric data for type 1 patients from the Gaucher Registry demonstrated:
Cerezyme® significantly reduced spleen and liver volumes in children
vs. baseline8,9
Cerezyme® significantly improved haematological parameters in children vs. baseline8,9
Cerezyme® can normalise growth in children with Gaucher disease and result in catch-up growth during treatment (relative to pre-treatment height)8,9
Cerezyme® increased BMD in the lumbar spine
vs. baseline8,9
Cerezyme® reduced or eliminated bone crisis in children during the study duration, compared to before treatment initiation8,9
Cerezyme® resulted in effective amelioration of osteopenia in children and adolescents vs. baseline9
Cerezyme® reduced or eliminated bone pain and improved bone lesions in children within 26 months vs. baseline10
Cerezyme® normalised growth and time of puberty onset in
most patients11
In bone and growth studies, the Cerezyme® treatment group from the Gaucher Registry studies represents patients who received either alglucerase or imiglucerase initially and continued treatment with imiglucerase long term.1,3,8,9,12
Did you know?
A carrier of Gaucher disease has a 50% chance of having a child which carries the Gaucher gene.
Learn more about Cerezyme®
Cerezyme® safety profile
Find out more about the safety and tolerability profile for Cerezyme® (imiglucerase).
Treatment with Cerezyme®
Discover more about treatment with Cerezyme® (imiglucerase) for patients living with Gaucher disease.
Cerezyme® safety profile
Find out more about the safety and tolerability profile for Cerezyme® (imiglucerase).
Treatment with Cerezyme®
Discover more about treatment with Cerezyme® (imiglucerase) for patients living with Gaucher disease.
Do you have questions or need support? We are here to help you.
References
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Mistry PK, Deegan P, Vellodi A, et al. Br J Haematol. 2009;147(4):561–570
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Hughes D, Mikosch P, Belmatoug N, et al. J Bone Miner Res. 2019;34(6):996–1013.
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Wenstrup RJ, Kacena KA, Kaplan P, et al. J Bone Miner Res. 2007;22(1):119–126.
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Andersson H, Kaplan P, Kacena K, et al. Pediatrics. 2008;122(6):1182–1190.
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Mistry PK, Weinreb NJ, Kaplan P, et al. Blood Cells Mol Dis. 2011;46(1):66–72.
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El-Beshlawy A, Ragab L, Youssry I, et al. J Inherit Metab Dis.2006;29(1):92–98.
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Kauli R, Zaizov R, Lazar L, et al. Isr Med Assoc J. 2000;2(2):158–163.
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El-Beshlawy A, Tylki-Szymanska A, Vellodi A, et al. Mol Genet Metab. 2017;120(1–2):47–56.
MAT code Date of preparation: February 2024