By evaluating patient-reported and electronic health record data, the pragmatic clinical study adds important real-world evidence to inform clinical practice.
Newly published findings from a pragmatic study conducted by the DCRI contribute new real-world evidence to the heart failure field.
In a prospective study of patients with heart failure with reduced ejection fraction (HFrEF) led by DCRI’s Robert Mentz, MD, therapy with sacubitril/valsartan was associated with improvements in quality of life (QOL) at 12 weeks compared with initiation or dose adjustment of ACE-inhibitors/ARB therapy (control). While the difference in QOL was not statistically significant from control following adjustment, these findings, which were recently published in the American Heart Journal and previously presented at the virtual American College of Cardiology Scientific Sessions 2020, add important real-world evidence regarding sacubitril/valsartan initiation in routine practice.
PROVIDE-HF was a prospective study of HFrEF outpatients who recently began taking sacubitril/valsartan compared with controls with recent ACE-inhibitor/ARB initiation or dose change. The study was conducted within PCORnet®, the National Patient-Centered Clinical Research Network. The primary endpoint was mean change in Kansas City Cardiomyopathy Questionnaire (KCCQ) scores through 12 weeks; this tool collects patient-reported outcome data by measuring the patient’s own perception of their health status. Overall, 270 patients had both baseline and 12-week KCCQ data (151 sacubitril/valsartan; 119 control).
“PROVIDE-HF has been a tremendous team effort that brought together multidisciplinary expertise, including clinical researchers and informatics experts at PCORnet sites and from the DCRI, with pragmatic elements of the study helping speed recruitment,” Mentz said. “These highly engaged teams were able to recruit several hundred patients within a year from fewer than 20 U.S. sites—an exceptional achievement. PROVIDE-HF is also one of the first prospective studies to leverage PCORnet electronic health record data to streamline study conduct and reduce the study burden on patients and sites while supporting high-quality data capture.”
Heart failure (HF) is a major and increasing public health problem, with 6 million Americans living with HF, a figure expected to rise to nearly 8 million by 2030. Around one-half of HF patients have HFrEF, which was the focus of PROVIDE-HF. Among recent advances for HF management is the angiotensin receptor–neprilysin inhibitor (ARNI) therapy, sacubitril/valsartan. Based on the findings of the earlier PARADIGM-HF study – funded by Novartis – which showed that sacubitril/valsartan improved clinical outcomes and QOL compared with enalapril for patients with HFrEF. However, prior to PROVIDE-HF, limited data were available about QOL after sacubitril/valsartan initiation in routine practice.
“Another important element of the PROVIDE-HF study design was the use of a ‘smart’ institutional review board (IRB), designed to ease common challenges associated with initiating multi-site research,” said Rosalia Blanco, MBA, the DCRI project leader for the study. “The smart IRB helped expedite protocol approval at the individual institutions, including elements such as the informed consent form. This approach also helped to reduce costs.”
In addition to Mentz and Blanco, DCRI authors on the American Heart Journal paper were Haolin Xu, MS; Emily O’Brien, PhD; Laine Thomas, PhD; Adam DeVore, MD, MHS; Johana Espinoza; and Adrian Hernandez, MD, MHS.