Evaluation of the Long-Term Effects of AAV1/SERCA2a Gene Therapy on Clinical Events in Heart Failure Patients Using a Novel Statistical Model: Results and Implications for Future Trials

Abstract

Time to first event incompletely measures the efficacy of heart failure therapy. Since morbid events are closely correlated, a different approach incorporating multiple cardiovascular-related events (CV death, worsening HF, MI, and HF-related hospitalization) was used to evaluate results of the Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) trial, a phase 2 study of AAV1/SERCA2a in patients with advanced heart failure.