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Clinical Use of Ventricular Assist Devices
The National Heart, Lung, and Blood Institute (NHLBI) convened a working group of experts on heart failure cardiology, cardiac surgery, clinical trial design, medical ethics, and regulatory affairs on March 27-28, 2008 in Crystal City, Virginia to advise the NHLBI on research directions for the treatment of advanced heart failure through clinical trials using ventricular assist devices (VADs).
The working group addressed three challenges of the 2007 NHLBI Strategic Plan: (1) to enhance the evidence available to guide the practice of medicine, and improve public health (Challenge 2.4); (2) to accelerate the translation of basic research findings into clinical studies and trials and to promote the translation of clinical research findings back to the laboratory (Challenge 2.1); and (3) to discover biomarkers that differentiate clinically relevant disease subtypes and that identify new molecular targets for application to prevention and diagnosis—including imaging, and therapy (Challenge 1.2).
VADs are currently used to bridge patients to heart transplantation, to allow their own hearts to recover or as permanent (“destination”) therapy. Using VADs to bridge to heart transplantation is inherently limited by the number of available donor organs and VAD-assisted cardiac recovery occurs in only a very limited number of cases. Due to associated device risks, destination therapy is currently limited in the US to those advanced heart failure patients who are not transplant eligible, have less than two years of life expectancy, and are on maximal heart failure medication. Recognizing the clinical advances offered by the current generation of smaller, more reliable, rotary VADs and in patient care and selection, the working group considered the highest priority to be a clinical trial to assess the use of VAD therapy in patients who are less ill than those currently eligible for destination therapy. Discussions focused on critical issues related to the potential trial such as: equipoise, patient characteristics, expected treatment effects, anticipated survival rates, trial results in similar patient populations, device characteristics and costs, the use of multiple devices, results of recent VAD trials, risk factors, competing therapies, and regulatory issues.
The working group reached consensus on the following points:
The Working Group proposed that a randomized clinical trial be designed to test the hypothesis whether VAD therapy may improve both survival and quality of life in those advanced heart failure patients who are neither inotrope-dependent nor exercise-intolerant and have not yet developed serious consequences such as malnourishment, end-organ damage, and immobility. The importance of testing a strategy of therapy rather than a specific device was emphasized, noting that the field of mechanical circulatory support would best be served by including multiple devices, if feasible. The Working Group considered NHLBI’s leadership role as necessary for the scientific objectivity in the trial design and administration, as well as to enhance access to shared data. NHLBI could also best facilitate the potential for including more than one device in the trial. Additionally, a large majority of the funding for the trial should be provided by industry and NHLBI could help to facilitate the industrial partnerships for the trial.
The following patient criteria and trial design characteristics were recommended.
A manuscript is planned for publication in a peer-reviewed journal.
J. Timothy Baldwin, Ph.D., NHLBI, NIH
Working Group Members:
NHLBI Program Staff
Last updated: May 22, 2008