Genzyme Announces Publication of Study Showing Impact of Cerezyme on Bone Health for Patients with Type 1 Gaucher Disease
Cerezyme, an enzyme replacement therapy, is the standard of care for patients with Type 1 Gaucher disease. Patients with Gaucher disease are at increased risk for developing bone complications, including osteopenia, osteoporosis and fractures. To evaluate the effect of long-term Cerezyme use on bone mineral density, the investigators conducted the largest-ever retrospective analysis of more than 500 patients participating in the International Collaborative Gaucher Group (ICGG) Gaucher Registry. The ICGG Gaucher Registry has been sponsored by Genzyme since 1991 and is overseen by an international board of scientific advisors.
The study compared the outcomes of 342 patients with Type 1 Gaucher disease who received treatment with Cerezyme to 160 patients who did not receive treatment. Both the treated and untreated patients had bone mineral density values significantly below normal at baseline. The patients who received Cerezyme were dosed at a range from 15-60 units per kilogram of body weight administered every two weeks. Bone mineral density was assessed by dual energy x-ray absorptiometry (DXA) of the lumbar spine.
The analysis found that patients receiving Cerezyme experienced a significant increase in bone mineral density over time when compared with patients who were not treated. Furthermore, the improvements in bone mineral density were found to be dose-dependent, with those patients receiving higher doses of Cerezyme achieving greater increases in bone mineral density. On average, the patients receiving Cerezyme at 60 units per kilogram biweekly achieved scores that approached those in the general population, whereas the untreated patients experienced no improvement or declining bone mineral density over the same period.
The investigators found that achieving normal (age- and sex-adjusted) bone mineral density scores may require eight years or more of sustained treatment with Cerezyme, longer than is required to reach normal values for other disease parameters, such as hematological parameters or reductions in liver and spleen volume. They conclude that a normal bone mineral density score should be a therapeutic goal for patients with Type 1 Gaucher disease.
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