The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease

• Published in: Clinical genetics 2007-03, Vol.71 (3), p.205-211
• Main Authors: Charrow, J, Dulisse, B, Grabowski, GA, Weinreb, NJ
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Biological and medical sciences; Bone and Bones - drug effects; Bone and Bones - pathology; bone crisis; Bone Diseases - drug therapy; Bone Diseases - etiology; bone pain; Child; enzyme replacement therapy; Errors of metabolism; Female; Follow-Up Studies; Fundamental and applied biological sciences. Psychology; Gaucher disease; Gaucher Disease - complications; Gaucher Disease - drug therapy; General aspects. Genetic counseling; Genetics of eukaryotes. Biological and molecular evolution; Glucosylceramidase - deficiency; Glucosylceramidase - therapeutic use; Humans; imiglucerase; Lipids (lysosomal enzyme disorders, storage diseases); Male; Medical genetics; Medical sciences; Metabolic diseases; Molecular and cellular biology; Pain - drug therapy; Registries; Treatment Outcome
• ISSN: 0009-9163; 1399-0004
• DOI: 10.1111/j.1399-0004.2007.00769.x

The effect of enzyme replacement therapy (ERT) on bone crisis and bone pain was investigated in patients with Gaucher disease (GD) type 1 followed over 4 years. Data from the International Collaborative Gaucher Group Gaucher Registry were used. Only patients with bone crisis and/or bone pain data for 1 year prior to ERT, and for each of 3 years after the start of ERT, were included. Bone crises were reported in 17% of patients during the year before starting ERT. The frequencies of bone crises decreased to 5%,