Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials

Background:In Gaucher disease type 1 (GD1), deficient lysosomal acid β-glucosidase activity leads to accumulation of glucosylceramide and formation of lipid-laden macrophages known as Gaucher cells. Patients experience a range of disease manifestations including anemia, thrombocytopenia, hepatosplenomegaly, and debilitating bone complications that impair quality of life. Hematologists are central to disease recognition and management. Most patients, even those without overt symptoms of bone involvement, have radiologic evidence of bone disease. Eliglustat, an oral substrate-reduction therapy, is a first-line treatment for adults with GD1 who have extensive, intermediate or poor CYP2D6 metabolizer phenotypes (>90% of patients). Oral eliglustat partially inhibits glucosylceramide synthase, thereby reducing glucosylceramide accumulation, unlike enzyme replacement therapy (ERT), the historic standard of care for GD1, which augments acid β-glucosidase activity in mononuclear phagocytes and is administered in biweekly infusions.Methods:We analyzed bone mineral density T- and Z-scores and occurrence of bone pain and bone crises in 4 completed clinical trials sponsored by Sanofi Genzyme that involved 393 GD1 patients treated with eliglustat for 4-8 years: Phase 2 (NCT00358150), Phase 3 ENGAGE (NCT00891202), Phase 3 ENCORE (NCT00943111), and Phase 3 EDGE (NCT01074944). Healthy reference scores are>-1 for T-scores and>-2 for Z-scores. Bone marrow burden (BMB) scores ...
Source: Blood - Category: Hematology Authors: Tags: 201. Granulocytes, Monocytes, and Macrophages: Poster II Source Type: research

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Source: Annali Italiani di Chirurgia - Category: Surgery Tags: Ann Ital Chir Source Type: research
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Source: Preventive Medicine - Category: International Medicine & Public Health Source Type: research
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Source: SafetyLit - Category: International Medicine & Public Health Tags: Alcohol and Other Drugs Source Type: news
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Source: SafetyLit - Category: International Medicine & Public Health Tags: Alcohol and Other Drugs Source Type: news
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Source: SafetyLit - Category: International Medicine & Public Health Tags: Alcohol and Other Drugs Source Type: news
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