The Gaucher Outcome Survey (GOS) is a long-term observational survey being developed in conjunction with leading international experts on Gaucher disease. Patient participation in GOS will be voluntary and GOS will be open to patients with Gaucher disease of any phenotype. Patients who are naïve to treatment, patients who are currently or have been previously treated with VPRIV, as well as patients who have been exposed to or are currently receiving other treatments for Gaucher disease may be included. It is designed to gain a better understanding of the clinical course of the disease and its response to VPRIV therapy, thereby improving the clinical management of patients affected by Gaucher disease.
The objectives of the Survey are:
• To characterize the clinical course of the patient population with Gaucher disease of any type.
• To characterize the population of patients affected with Gaucher disease who are receiving treatment.
• To monitor the safety and effectiveness of VPRIV® (velaglucerase alfa) enzyme replacement therapy.
• To collect data on the long-term safety and effectiveness of VPRIV treatment in patients with Gaucher disease who are new to therapy or who have been switched from another agent.
• To enhance the understanding of the response of VPRIV in patients with Gaucher disease.
• To collect data on the impact of treatment on the health-related quality of life for patients with Gaucher disease.
• To provide a platform for the collection of data on the clinical experience with VPRIV and to serve as a database for evidence-based management of Gaucher disease.
• To collect ongoing information on safety and effectiveness in support of regulatory and pharmacovigilance obligations
Principle Investigator: Nicola Longo
Principle Department: Pediatric Genetics
Co Investigator: Nicola Longo
Co Investigator: Dave Viskochil
Co Investigator: Ashley Warnock
Co Investigator: Lorenzo Botto