Abstract
Gaucher disease has been the first lysosomal storage disorder for which an enzyme replacement therapy has been approved in the 1990s and was the first to receive approval for afirst-line substrate reduction therapy in 2015. Eliglustat treatment has been started in Austria in patients recruited to aclinical trial, followed by its long-term extension and prescription treatment overall covering up to 10years. In this case series the experience of treating Gaucher patients with eliglustat in Vienna is summarized. Patients were either switched from enzyme replacement therapy or were therapy naïve. Significant improvements were shown in hematological (thrombocytes, hemoglobin) and visceral (spleen volume) manifestations as well as in biomarkers (chitotriosidase, glucosylsphingosine [lyso-GL1], angiotensin converting enzyme) in aroutine setting in atherapy-naïve patient. Stability was found in switch patients with slight improvement in bone density. Eliglustat was generally very well tolerated. Patient selection and regular monitoring is required to ensure effective and safe use.
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