VP228 Post-hoc analyses of prednisolone use and hepatotoxicity in clinical trials of intravenous onasemnogene abeparvovec

Onasemnogene abeparvovec (OA) is a one-time intravenous gene replacement therapy approved for treatment of SMA. Acute serious liver injury, acute liver failure, and aminotransferase elevations can occur with OA and are acknowledged potential risks for OA that may be immune-mediated. Here, we report the findings from post-hoc analyses exploring differences in hepatotoxicity outcomes based on starting dose of 1 vs. 2 mg/kg/day, the time course of prednisolone administration and comparisons to protocol guidelines and shifts in ALT/AST values or other aspects of hepatotoxicity before and during the tapering period.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: T. Macek, S. Wen, E. O'Brien, I. Alecu, S. Tauscher-Wisniewski, B. McGill Source Type: research