Dr. Barbara Burton discussed the long-term comparative effectiveness of pegvaliase versus medical nutrition therapy with and without sapropterin in adults with phenylketonuria, highlighting pegvaliase's superior efficacy in reducing blood phenylalanine levels and increasing protein intake.
0:48 - Background on PKU: Explanation of PKU as a metabolic disorder and traditional treatments involving a phenylalanine-restricted diet and sapropterin.
1:57 - Study Population: Participants aged 16 or older with baseline blood phenylalanine concentrations ≥600 µmol/L.
2:41 - Outcome Measures: Blood phenylalanine levels, proportions achieving specific blood phenylalanine targets, and intact protein intake.
3:57 - Results: Pegvaliase significantly reduced blood phenylalanine levels compared to other treatments.
4:38 - Protein Intake: Higher intact protein intake in the pegvaliase group compared to other groups.
7:48 - Conclusion: Pegvaliase offers durable efficacy in reducing blood phenylalanine levels and increasing protein intake, providing a promising treatment for adults with PKU.