Drug utilisation study of eliglustat for the treatment of Gaucher disease type 1 in Europe using electronic healthcare records
Description
- Gaucher disease (GD) is a rare genetic disorder from the group of lysosomal storage disorders. It is characterized by the buildup of glucocerebroside in certain organs and tissues, particularly the spleen, liver, and bone marrow. This buildup occurs due to a deficiency of an enzyme called glucocerebrosidase, which is responsible for breaking down glucocerebroside. As a result, the excessive accumulation of glucocerebroside leads to the enlargement of organs, anemia, low platelet count, and bone abnormalities. Symptoms can vary in severity and may include fatigue, bruising, bone pain, and an enlarged abdomen. Treatment options include enzyme replacement therapy and substrate reduction therapy.
As part of the Risk Management Plan and as requested by the European Medicine Agency (EMA), this study investigates the drug utilization of Eliglustat, which has been introduced the German market in 2015 as treatment for GD. In particular, the concomitant use of other drugs that are relevant for potential interactions is investigated in persons receiving eliglustat. The concomitant use of such potentially interacting drugs may, on the one hand, lead to undesirably increased or decreased plasma concentrations of eliglustat. On the other hand, the plasma concentrations of other drugs may be increased by the concomitant use of eliglustat. These interactions can increase the risk of side effects or lead to reduced efficacy of eliglustat.
Thus, in this multicenter study, along with three other study centers (France, Israel, Denmark), the use of concomitant medications with inducing or reducing effects on these enzymes are carefully monitored in patients under treatment with Eliglustat.
Funding period
- Begin: June 2021
End: June 2024
Sponsor
- Pharmaceutical industry