Improvements in Bone Disease with Imiglucerase Therapy for Gaucher Disease
Author Information
Author(s): Katherine B. Sims, Gabriel M. Pastores, Naomi J. Weinreb, Jean Barranger, Bruce E. Rosenbloom, Sharon Packman, Paul Kaplan, Hugh Mankin, Rafael Xavier, James Angell, Mary A. Fitzpatrick, David Rosenthal
Primary Institution: Massachusetts General Hospital
Hypothesis
Does imiglucerase therapy improve skeletal manifestations in patients with type 1 Gaucher disease?
Conclusion
Imiglucerase therapy significantly reduces bone pain and crises while increasing bone mineral density in patients with type 1 Gaucher disease over 48 months.
Supporting Evidence
- 39% of patients reported bone pain at 48 months compared to 73% at baseline.
- 11 of the 13 patients with a history of bone crises had no recurrences during the study.
- Mean Z score for spine improved from -0.72 to -0.09 by month 48.
- Mean Z score for femoral neck improved from -0.59 to -0.17 at month 36.
Takeaway
This study shows that a medicine called imiglucerase helps people with a disease called Gaucher disease feel less bone pain and have stronger bones.
Methodology
A multicenter, open-label, single-cohort, prospective study evaluating the effectiveness of imiglucerase in treating skeletal manifestations of type 1 Gaucher disease.
Potential Biases
Potential bias due to the lack of a control group and the open-label design.
Limitations
The study was non-randomized and open-label, which may introduce bias.
Participant Demographics
Median age 43 years; 70% were of Ashkenazic Jewish ethnicity.
Statistical Information
P-Value
p = 0.042 for spine Z score improvement, p = 0.035 for femoral neck Z score improvement.
Statistical Significance
p<0.001
Digital Object Identifier (DOI)
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