Datamonitor Healthcare attended the 2016 American Heart Association (AHA) Scientific Sessions and brings you the latest research on heart disease. Analyst Kevin Shannon shares his insight on one of the presented chronic heart failure studies, the EFFECT-HF study.
Topline results from the Phase III EFFECT-HF study of Injectafer (ferric carboxymaltose; Genesis Pharma/The Galenica Group/Zeria) and its impact on exercise capacity in chronic heart failure patients with iron deficiency were presented at the 2016 American Heart Association (AHA) Scientific Sessions in New Orleans. The data presented were in line with previous studies investigating Injectafer in heart failure, and provide strong evidence that focusing on iron deficiency as an independent therapeutic target may benefit these patients. Investigators at AHA 2016 seemed to encourage immediate changes in prescribing practices by arguing that increased exercise capacity, measured by oxygen consumption (VO2), should be considered as an outcome in itself. They claimed that this is an important issue facing heart failure patients, as it has a significant negative impact on their lives.
However, there was some concern over the increased rate of serious adverse events and hospitalizations. As a result, long-term cardiovascular outcomes trials should be the next step in the development of Injectafer for this patient population, as further outcomes and safety data are needed.
The EFFECT-HF trial demonstrated that Injectafer improved peak VO2 relative to placebo in patients with class II/III heart failure with reduced ejection fraction and iron deficiency, meeting the primary endpoint of the study. Results were independent of anemia, although patients with hemoglobin ≥15g/dL were excluded from the trial. The drug also had significant improvements in New York Heart Association classification of heart failure class and quality of life relative to placebo, confirming findings from the FAIR-HF and CONFIRM-HF studies.
The results from this trial, combined with data from FAIR-HF and CONFIRM-HF, provide substantial evidence that iron deficiency is a legitimate independent therapeutic target in heart failure. Treating this patient population with Injectafer has been shown to significantly improve quality of life and exercise capacity. These endpoints, on their own, are an important outcome to heart failure patients, as many are restricted in their daily activities due to fatigue and dyspnea.
However, the EFFECT-HF trial highlighted some safety concerns associated with Injectafer treatment, prompting the need for additional trials investigating outcomes such as survival and worsening heart failure before changes in guidelines should be made. Treatment with Injectafer was associated with an increased risk of hospitalization and serious adverse events, primarily cardiovascular in nature. This contradicts data from the FAIR-HF and CONFIRM-HF trials, which demonstrated that Injectafer therapy led to numerically lower rates of hospitalization and significantly reduced rates of hospitalization due to heart failure. Now that the results of EFFECT-HF have raised doubts about the safety of Injectafer in this patient population, further investigation into the effects of the drug will be essential.