| Object:To investigate the efficacy and safety of human umbilical cord mesenchymal stem cells(hUC-MSCs)treatment of cardiomyopathy with cardiomegaly and heart failure in children,and to provide a basis for its promotion in clinical practice.Methods:This was a non-randomized and open-source study of 73 children with cardiomypathy with cardiomegaly and heart failure.According to the intent-to-treat of the children and their parents,they were divided into two groups: hUC-MSCs treatment group and conventional treatment group.We collected characteristics of these patients,and compared the short-term and medium-term efficacy and safety indexes of two groups Results:1.Forty children were treated with hUC-MSCs-treatment,of which 17 were DCM,17 were LVNC,5 were EFE,1 was ARVC,and male to female ratio was 1.1: 1,average height was(98.58 ± 25.28)cm,the average weight was(20.04 ± 16.32)kg,the average age was(62.08 ± 44.06)months,and the average course of disease was(36.25 ± 29.02)months before enrollment.Thirty-three children were treated with congventional-treatment,of which 28 were DCM,4 were LVNC,and 1 was EFE,and male to female ratio 0.94: 1,the average height was(87.61 ± 34.28)cm,average weight was(13.76 ± 12.20)kg,average age was(23.88 ± 28.04)months,and average course of disease(19.58 ± 24.04)months before enrollment.There were 24 cases with heart failure NYHA class III/ ? in the hUC-MSCs treatment group and 26 cases in the conventional treatment group.Peripheral blood NT-proBNP was(4211.68 ±7234.88)pg/ml in hUC-MSCs treatment group and(17961.59 ± 14713.08)pg/ml in the conventional treatment group.The Z score of LVPWd and IVSd of the two groups were in the normal range,and the differences were not statistically significant;while the Z score of LVDd,LVM and LVEDV,and LVMI,LVEDVI was higher than normal without statistically significant.2.At the end of follow-up,there was no death in the hUC-MSCs treatment group,and the average survival time was(26.75 ± 17.80)months.In the conventional treatment group,4 children died,and 29 children survived,with an average survival(19.03 ± 13.98)months.The improvement rate of NYHA classification was 77.5% and 57.58% in hUC-MSCs treatment group and conventional treatment group,respectively;the improvement of NT-proBNP was(-2950.33 ± 6014.95)pg/ml and(-11740.10± 14572.18)pg/ml in hUC-MSCs treatment group and conventional treatment group,respectively.3.LVEF increased by(22.14 ± 13.70)% and LVFS increased by(9.37±7.83)% in the hUC-MSCs treatment group;and Z score of LVDd,LVM and LVEDV decreased(4.84 ± 3.89),(6.94 ± 7.54)and(11.67 ± 13.13),respectively.In the conventional treatment group,LVEF increased by(10.73 ± 15.38)% and LVFS increased by(5.11±8.72)%;Z score of LVDd,LVM,and LVEDV decreased(2.07±4.30),(2.92 ± 6.67)and(4.77 ± 7.56),respectively.4.The rate of abnormal ECG improvement in the hUC-MSCs treatment group was94.12%,compared with 70.59% in the conventional treatment group.There were no significant differences in liver and kidney function between the two groups of children with cardiomyopathy.The hUC-MSCs treatment group had no adverse effects such as immune rejection.Conclusions:Human umbilical cord mesenchymal stem cells have a good short to medium-term effect in treating pediatric cardiomypathy with cardiomegaly and heart failure,which can improve the quality of life without significant adverse effects. |
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