Cell Therapy in Chagas Cardiomyopathy (Chagas Arm of the MiHeart Study): A Multicenter Randomized Trial

Background —Previous studies suggested that transplantation of autologous bone marrow derived mononuclear cells (BMNC) improves heart function in chronic chagasic cardiomyopathy (CCC). We report the results of the first randomized trial of BMNC therapy in CCC. Methods and Results —Patients aged 18-75 years with CCC, NYHA class III or IV, LVEF less than 35%, and optimized therapy were randomized to intracoronary injection of autologous BMNC or placebo. Primary endpoint was the difference in LVEF from baseline to 6 and 12 months after treatment between groups. Analysis was by intention to treat and powered to detect an absolute between-group difference of 5%. Between July 2005 and October 2009 234 patients were enrolled. Two abandoned the study and 49 were excluded due to protocol violation. The remaining 183 patients, 93 in the placebo group and 90 in the BMNC group, had trimmed mean age of 52.4 years (50.8 to 54.0) and LVEF of 26.1% (25.1 to 27.1) at baseline. Median number of injected BMNCs was 2.20 x 108 (1.40-3.50 x 108). Change in LVEF did not differ significantly between treatment groups: trimmed mean ΔEF= 3.0 (1.3 to 4.8) for BMNC and 2.5 (0.6 to 4.5) for placebo (p=0.519) at 6 months; ΔEF= 3.5 (1.5 to 5.5) for BMNC and ΔEF= 3.7 (1.5 to 6.0) for placebo (p=0.850) at 12 months. Left ventricular systolic and diastolic volumes, NYHA class, Minnesota life quality questionnaire, BNP concentrations, and 6-min walking test did also not differ between groups. Conclusions —Intracoronary injection of autologous BMNCs does not improve left ventricular function or quality of life in patients with CCC. Clinical Trial Registration Information —ClinicalTrials.gov; Identifier: [NCT00349271][1] [1]: /lookup/external-ref?link_type=CLINTRIALGOV&access_num=NCT00349271&atom=%2Fcirculationaha%2Fearly%2F2012%2F04%2F19%2FCIRCULATIONAHA.111.067785.atom