PCR70 Avalglucosidase Alfa (AVA) Improves Symptoms and Functioning in Late-Onset Pompe Disease (LOPD) Patients vs Alglucosidase Alfa (ALG): Post-Hoc Analyses of Patient-Reported Outcomes (PROs) From COMET Trial

Abstract

The phase 3 COMET study (NCT02782741) included multiple PROs that assessed patient’s experience in adults with Pompe disease (PD). Three PROs: Pompe Disease Symptom Scale (PDSS), Pompe Disease Impact Scale (PDIS), Rasch-Built Pompe-Specific Activity (R-PAct) scale specifically measure PD associated symptoms and impacts. These post-hoc analyses were conducted to further illustrate clinical benefits of AVA (n=50) vs. ALG (n=49).