Halliday BP, et al. - Researchers investigated whether it is safe to withdraw treatment in patients with dilated cardiomyopathy whose symptoms and cardiac function have recovered. Based on their observations, treatment withdrawal will lead to relapse in many patients deemed to have recovered from dilated cardiomyopathy and therefore, they recommended treatment continuation indefinitely until robust predictors of relapse are defined.

Methods

• This was an open-label, pilot, randomised trial.
• Participants were patients with previous dilated cardiomyopathy who were now asymptomatic, whose left ventricular ejection fraction (LVEF) had improved from less than 40% to 50% or greater, whose left ventricular end-diastolic volume (LVEDV) had normalised, and who had an N-terminal pro-B-type natriuretic peptide (NT-pro-BNP) concentration less than 250 ng/L.
• Researchers assessed the impact of phased withdrawal of heart failure medications in the participants.
• They recruited patients from a network of hospitals in the UK.
• Patients were assessed at one centre (Royal Brompton and Harefield NHS Foundation Trust, London, UK), and were randomly assigned (1:1) to phased withdrawal or continuation of treatment.
• Using the same method, patients in the continued treatment group had treatment withdrawn after 6 months.
• A relapse of dilated cardiomyopathy within 6 months, defined by a reduction in LVEF of more than 10% and to less than 50%, an increase in LVEDV by more than 10% and to higher than the normal range, a two-fold rise in NT-pro-BNP concentration and to more than 400 ng/L, or clinical evidence of heart failure, was assessed as primary endpoint, at which point treatments were re-established.
• Intention to treat primary analysis was performed.

Results

• Enrollment of 51 patients was done between April 21, 2016, and Aug 22, 2017.
• A total of 25 and 26 patients were randomly assigned to the treatment withdrawal group and to continue treatment, respectively.
• In treatment withdrawal group vs continue treatment, the primary endpoint of relapse was met by 11 (44%) patients over the first 6 months (Kaplan-Meier estimate of event rate 45·7% [95% CI 28·5–67·2]; p=0·0001).
• According to findings, 25 (96%) of 26 patients assigned initially to continue treatment attempted its withdrawal, after 6 months.
• The primary endpoint of relapse was met by 9 patients during the following 6 months (Kaplan-Meier estimate of event rate 36·0% [95% CI 20·6–57·8]).
• Findings revealed no deaths in either group.
• The treatment withdrawal group experienced three serious adverse events: hospital admissions for non-cardiac chest pain, sepsis, and an elective procedure.