ROADMAP at Two Years: Primary Outcomes Remain Intact, New Insights Emerge

Decision-making for patients with non-inotrope-dependent heart failure has just gotten a bit easier — or at least better-informed — thanks to the publication of two-year results of the ROADMAP trial comparing left ventricular assist device (LVAD) therapy with optimal medical management (OMM).

The two-year results showed that LVADs’ advantage over OMM on the primary composite end point of survival on original therapy with improvement in six-minute walk distance extends out to two years. But the latest results also add some treatment-guiding insights, including evidence of a reduction in adverse events with LVAD therapy in the second (versus the first) year of follow-up and evidence that there is no increase in mortality with delaying LVAD implantation among OMM patients who are monitored closely by a heart failure specialist.

“The one-year ROADMAP results showed that survival with improved functional status was better with LVADs than with optimal medical management, although survival alone was similar in the two groups,” notes ROADMAP lead investigator and Cleveland Clinic cardiologist Randall Starling, MD, MPH. “But factors beyond survival are highly important to decision-making about LVADs in this ambulatory population. So we decided to evaluate ROADMAP’s pre-specified primary end point and quality of life (QoL)-related results at two years of follow-up to better understand long-term benefits and risks of LVADs relative to optimal medical management.”

ROADMAP: A deep dive into decision-making

Briefly, ROADMAP — whose one-year results were published in Journal of the American College of Cardiology (JACC) in 2015 — was a prospective, nonrandomized, observational study of 200 ambulatory patients (97 with an LVAD, 103 on OMM) with New York Heart Association class IIIb/IV heart failure.

“This was designed as a nonrandomized trial in which patients and their doctors could decide between an LVAD or optimal medical management,” Dr. Starling explains. “Detailed efforts were made to capture data that would shed light on why patients and their providers made their therapy choice and to gather patient-reported outcomes.”

Outcomes at year 2

As detailed in the full two-year study report, published in JACC: Heart Failure, notable findings from ROADMAP at two-year follow-up included the following:

• Compared with the OMM group, the LVAD group had a higher rate of survival with improved functional status, improved QoL and lower levels of depression.
• Survival among the 23 patients in the OMM group who received delayed LVAD placement (18 within 12 months; 5 between 12 and 24 months) was statistically comparable to survival in the original LVAD group.
• The hospitalization rate was higher throughout the study in the LVAD group than in the OMM group.
• Rates of major adverse events were higher in the LVAD group than in the OMM group throughout the study, but rates in the LVAD group significantly declined in year 2 versus year 1.

Implications for decision-making

“One of the most provocative findings is that despite the LVAD group having more hospitalizations, more adverse events and worse self-reported pre-intervention QoL, LVAD treatment was associated with greater improvements in depression and QoL compared with optimal medical management at both one year and two years,” Dr. Starling observes. “This suggests that patients living with heart failure may choose to accept a certain burden of adverse events to enhance their functional capacity and QoL. Patient choices may not always be intuitive to physicians.”

Another important clinical takeaway, he notes, is that survival is not compromised if a patient delays LVAD therapy — so long as the patient’s OMM is monitored closely by a heart failure specialist and access to an LVAD and transplantation is maintained. “But this delayed decision does delay improvements in the patient’s functional status and QoL,” he adds.

Bottom line

While acknowledging ROADMAP’s limitations as a nonrandomized trial with potential biases in patient selection and patient-reported outcomes, Dr. Starling points out that it provides the first evidence that non-inotrope-dependent patients electing LVAD placement will reap gains in functional capacity and QoL compared with their counterparts who choose medical therapy.

“Until a randomized trial of these questions is undertaken in this population,” he adds, “ROADMAP provides information to help physicians counsel patients on which treatment option makes the most sense for them in terms of both survival and quality of life.”