Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry

• Published in: Journal of clinical medicine Vol. 13; no. 10; p. 2782
• Main Authors: Deegan, Patrick, Lau, Heather, Elstein, Deborah, Fernandez-Sasso, Diego, Giraldo, Pilar, Hughes, Derralynn, Zimran, Ari, Istaiti, Majdolen, Gadir, Noga, Botha, Jaco, Revel-Vilk, Shoshana
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 01.05.2024
• Subjects: Age; Anemia; Care and treatment; Clinical medicine; Clinical trials; Demographics; Diagnosis; Enrollments; enzyme replacement therapy; Enzymes; ERT; Gaucher disease; Gaucher's disease; Genetic aspects; GOS; Liver; Magnetic resonance imaging; Participation; Patients; Plasma; registry; Spleen; Thrombocytopenia; Ultrasonic imaging; velaglucerase alfa; United Kingdom; United Kingdom > UK; registry; Gaucher disease; enzyme replacement therapy; ERT; velaglucerase alfa; GOS; long term
• ISSN: 2077-0383; 2077-0383
• DOI: 10.3390/jcm13102782

: Gaucher disease (GD) is a rare, autosomal, recessive condition characterized by hepatosplenomegaly, thrombocytopenia, anemia, and bone abnormalities, often requiring life-long treatment. Velaglucerase alfa has improved hematologic and visceral parameters in clinical trials; however, limited long-term efficacy and safety data are available. : The Gaucher Outcome Survey (GOS), a structured and validated international registry for patients with confirmed GD, provides an opportunity to evaluate long-term data from patients receiving velaglucerase alfa. : This analysis included 376 treatment-naïve children and adults with GD enrolled in GOS, including 20 with type 3 GD, who initiated velaglucerase alfa through participation in clinical trials or as part of their clinical management and continued treatment for a mean (range) time of 6.6 (0.003-18.6) years. Initial improvements in hematologic and visceral parameters and the biomarkers glucosylsphingosine (lyso-GL1) and chitotriosidase were observed after one year of treatment and were maintained throughout the follow-up period. Of 129 (34.3%) patients who developed adverse events during the follow-up period, events were considered related to treatment in 33 (8.8%). None led to treatment discontinuation. There were 21 deaths overall, none of which were considered related to treatment. : This analysis of data from the GOS registry supports the safety and efficacy of velaglucerase alfa in patients with GD.