What is the role of stem cells in the treatment of dilated cardiomyopathy?

Updated: Mar 02, 2021
  • Author: Vinh Q Nguyen, MD, FACC; Chief Editor: Gyanendra K Sharma, MD, FACC, FASE  more...
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Human embryonic stem cells have been differentiated ex vivo to derive cardiac myocyte stem cells. When transplanted into rats that had left anterior descending coronary artery ligation, these stem cells have been shown to attenuate the adverse remodeling seen with extensive infarcts. [147]

Autologous stem cells have been given both intramyocardially and intravenously for the treatment of congestive heart failure, with varying results. Much of the early data from these trials seem to suggest that delivery mechanisms to the myocardium and the use of concomitant cytokines are equally in need of further investigation. [148]

An evaluation of the safety and efficacy of ixmyelocel-T, administered via minithoracotomy or intramyocardial catheter injections, in 2 prospective randomized phase 2A trials in patients with dilated cardiomyopathy (DCM) stratified by ischemic or nonischemic status revealed evidence that intramyocardial injection with ixmyelocel-T reduced major adverse cardiovascular events and improved symptoms in patients with ischemic DCM but not in patients with nonischemic DCM. [149] In the IMPACT-DCM trial, 39 patients were randomized to either ixmyelocel-T or standard-of-care control in a 3:1 ratio; ixmyelocel-T was administered intramyocardially via minithoracotomy. In the Catheter-DCM trial, 22 patients were randomized to either ixmyelocel-T or standard-of-care control in a 2:1 ratio; ixmyelocel-T was administered intramyocardially with the NOGA Myostar catheter.

Relative to control patients, fewer ischemic patients treated with ixmyelocel-T experienced a major adverse cardiovascular event during follow-up, but nonischemic patients did not have a similar benefit. [149] The most common major adverse cardiovascular event was exacerbation of heart failure. Those in the ischemic population who received ixmyelocel-T had improved NYHA class, 6-minute walk distance, and Minnesota Living with Heart Failure Questionnaire scores compared to the control group; again, the nonischemic group did now demonstrate a similar trend. [149]

In a nonrandomized prospective study involving 14 patients with end-stage ischemic heart disease who underwent transendocardial injection of autologous mononuclear bone marrow cells, Perin et al noted a significant reduction in total reversible defect, improvement in global LV function, reduction in end-systolic volume, and improvement in LVEF from 20% at baseline to 29%. [150]

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