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- Title
The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease.
- Authors
Charrow, J; Dulisse, B; Grabowski, G A; Weinreb, N J
- Abstract
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%, <1% and 3% for 1, 2, and 3 years after initiation of treatment, respectively (p < 0.0001). Bone pain followed a similar pattern of response. Bone pain was reported in 49% of patients the year before treatment and decreased to 30% in the first year, 29% in the second year, and 30% in the third year of ERT (p < 0.0001). ERT is associated with a reduction in bone crisis and bone pain in patients with GD type 1 . This study shows that significant improvements in symptoms of skeletal disease are achievable clinical outcomes and treatment goals in GD type 1.
- Publication
Clinical genetics, 2007, Vol 71, Issue 3, p205
- ISSN
0009-9163
- Publication type
Journal Article
- DOI
10.1111/j.1399-0004.2007.00769.x