Trial participants with HFrEF had similar adjusted all-cause mortality compared to trial-eligible registry patients (SMR 0.97; 95% CI 0.92-1.03), but higher adjusted cardiovascular mortality.
Observational (n=63,836)
Yes
Are patient characteristics and outcomes similar between HFrEF patients in randomized clinical trials and observational registries?
Over half of HFrEF patients in contemporary registries would be eligible for trials, and while crude event rates are lower in trials, case-mix adjusted cardiovascular mortality is higher in trial participants.
Effect estimate: SMR 0.97 (95% CI 0.92-1.03)
Absolute Event Rate: 7% vs 12%
BACKGROUND: Heart failure (HF) trials have stringent inclusion and exclusion criteria, but limited data exist regarding generalizability of trials. We compared patient characteristics and outcomes between patients with HF and reduced ejection fraction (HFrEF) in trials and observational registries. METHODS AND RESULTS: Individual patient data for 16 922 patients from five randomized clinical trials and 46 914 patients from two HF registries were included. The registry patients were categorized into trial-eligible and non-eligible groups using the most commonly used inclusion and exclusion criteria. A total of 26 104 (56%) registry patients fulfilled the eligibility criteria. Unadjusted all-cause mortality rates at 1 year were lowest in the trial population (7%), followed by trial-eligible patients (12%) and trial-non-eligible registry patients (26%). After adjustment for age and sex, all-cause mortality rates were similar between trial participants and trial-eligible registry patients standardized mortality ratio (SMR) 0.97; 95% confidence interval (CI) 0.92-1.03 but cardiovascular mortality was higher in trial participants (SMR 1.19; 1.12-1.27). After full case-mix adjustment, the SMR for cardiovascular mortality remained higher in the trials at 1.28 (1.20-1.37) compared to RCT-eligible registry patients. CONCLUSION: In contemporary HF registries, over half of HFrEF patients would have been eligible for trial enrolment. Crude clinical event rates were lower in the trials, but, after adjustment for case-mix, trial participants had similar rates of survival as registries. Despite this, they had about 30% higher cardiovascular mortality rates. Age and sex were the main drivers of differences in clinical outcomes between HF trials and observational HF registries.
Lim et al. (Thu,) conducted a observational in Heart failure with reduced ejection fraction (HFrEF) (n=63,836). Trial participation vs. Observational registry population (trial-eligible) was evaluated on All-cause mortality (SMR 0.97, 95% CI 0.92-1.03). Trial participants with HFrEF had similar adjusted all-cause mortality compared to trial-eligible registry patients (SMR 0.97; 95% CI 0.92-1.03), but higher adjusted cardiovascular mortality.