International Observational Registry for Gaucher Disease Patients Tracking Long-Term Outcomes and Treatments

What this Trial Is About

The Gaucher Outcomes Survey GOS is an ongoing international observational registry for patients diagnosed with Gaucher Disease, regardless of their treatment status or type of treatment they receive. The registry aims to collect long-term data to better understand the natural history of Gaucher Disease and to evaluate the safety and long-term effectiveness of treatments like velaglucerase alfa. This study also seeks to provide a database to support evidence-based management of Gaucher Disease in real-life clinical settings. Participants in GOS are not given any experimental treatments as the study is observational. Patients undergo clinical assessments and receive care as determined by their own treating physicians. Enrollment was temporarily paused in some locations due to the COVID-19 pandemic but has since resumed in several countries. The registry includes patients of all ages and treatment types, tracking their health outcomes over many years. During the study, participants undergo regular clinical evaluations to monitor various health measures such as adverse events, infusion-related reactions, hemoglobin levels, platelet counts, liver and spleen volumes, and bone mineral density. These outcomes are followed from baseline through one year and can continue for up to 20 years. The long-term collection of data helps researchers understand treatment effects and disease progression in real-world practice while ensuring ongoing safety monitoring.

Gaucher Disease Outcome Survey (GOS)