title: One-year follow-up of feasibility and safety of the first U.S., randomized, controlled study using 3-dimensional guided catheter-based delivery of autologous skeletal myoblasts for ischemic cardiomyopathy (CAuSMIC study).
abstract: OBJECTIVE
  The aim of this study was to test safety and feasibility of myoblast transplantation with the Biosense-NOGA (Diamond Bar, California) 3-dimensional-guided endomyocardial delivery system.
  
  
  BACKGROUND
  Previous Phase-1 trials showed feasibility of epicardial injection of myoblasts. However, catheter-based delivery has several advantages: it can be applied on high-risk patients, the procedure can be repeated, and it is associated with less morbidity and mortality.
  
  
  METHODS
  Twenty-three subjects, with previous myocardial infarction and heart failure, New York Heart Association (NYHA) functional class II to IV, were enrolled, 11 control and 12 treatment subjects. To assess safety, physical exam, electrocardiogram, continuous rhythm monitoring, quality of life assessments, and heart function were evaluated at baseline and follow-up until 1 year.
  
  
  RESULTS
  There was favorable safety: no difference between groups in arrhythmias, and no deaths. Treated subjects showed sustained improvements in NYHA and Minnesota Living with Heart Failure Questionnaire (MLHFQ) compared with control subjects (NYHA, -1.0 point in treatment vs. +0.3 point in control group, p < 0.0004; MLHFQ, -14 point in treatment vs. +1 point in the control group, p = 0.004). Blinded core laboratory echocardiography evaluations showed sustained reductions in the treatment versus control in end diastolic diameter (-0.03 cm vs. +0.05 cm, p = 0.07) and end systolic diameter (-0.05 cm vs. +0.1 cm, p = 0.07). Finally, NOGA voltage mapping demonstrated improved voltage measurements (+1.0 mV, p = 0.008).
  
  
  CONCLUSIONS
  This trial of myoblast transplantation via catheter into heart failure patients demonstrated safety and feasibility. Treated patients showed improvement in NYHA, MLHFQ, ventricular viability, and evidence of reverse ventricular remodeling. These data demonstrate positive safety outcomes and warrant initiation of larger phase 2, double-blind, placebo-controlled clinical trials.
authors.affiliation: Clinical Cardiovascular Cell Therapy, University of California, San Diego, La Jolla, California 92037-1300, USA. ndib@cardiostem.com
authors.forename: Nabil
authors.initials: N
authors.lastname: Dib
authors.forename: Jonathan
authors.initials: J
authors.lastname: Dinsmore
authors.forename: Zaki
authors.initials: Z
authors.lastname: Lababidi
authors.forename: Bee
authors.initials: B
authors.lastname: White
authors.forename: Susan
authors.initials: S
authors.lastname: Moravec
authors.forename: Ann
authors.initials: A
authors.lastname: Campbell
authors.forename: Amy
authors.initials: A
authors.lastname: Rosenbaum
authors.forename: Katayoun
authors.initials: K
authors.lastname: Seyedmadani
authors.forename: Wael A
authors.initials: WA
authors.lastname: Jaber
authors.forename: Craig S
authors.initials: CS
authors.lastname: Rizenhour
authors.forename: Edward
authors.initials: E
authors.lastname: Diethrich
mesh_terms: D000368:Aged; D002311:Cardiomyopathy, Dilated / Q000628:therapy*; D005240:Feasibility Studies; D005260:Female; D006333:Heart Failure / Q000000981:diagnostic imaging / Q000628:therapy*; D006352:Heart Ventricles / Q000473:pathology; D006801:Humans; D021621:Imaging, Three-Dimensional; D008297:Male; D008875:Middle Aged; D032448:Myoblasts, Skeletal / Q000637:transplantation*; D011788:Quality of Life; D011795:Surveys and Questionnaires; D013997:Time Factors; D014182:Transplantation, Autologous; D014463:Ultrasonography