Genzyme Corporation (CAMBRIDGE, Massachusetts) announced that long-term use of Cerezyme® (imiglucerase for injection) significantly improves bone mineral density (BMD) in patients with Type 1 Gaucher disease in a dose-dependent manner. Cerezyme, an enzyme replacement therapy, is the standard of care for patients with Type 1 Gaucher disease who are at increased risk for developing bone complications, including osteopenia, osteoporosis, and fractures. To evaluate the effect of long-term Cerezyme use on BMD, the study 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 BMD 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 2 weeks. BMD was assessed by dual energy x-ray absorptiometry of the lumbar spine.

The analysis found that patients receiving Cerezyme experienced a significant increase in BMD over time when compared with patients who were not treated. Furthermore, the improvements in BMD were found to be dose-dependent, with those patients receiving higher doses of Cerezyme achieving greater increases in BMD. 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 BMD over the same period.

The investigators found that achieving normal (age- and sex-adjusted) BMD scores may require 8 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 BMD score should be a therapeutic goal for patients with Type 1 Gaucher disease.

—A. Techman

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