雷帕霉素治疗心脏横纹瘤的效果及患儿远期生存观察

Observation of Efficacy of Rapamycin in Treatment of Cardiac Rhabdomoma and Long-term Survival

目的探究雷帕霉素治疗心脏横纹瘤的临床效果以及患儿远期生存情况。方法选择100例心脏横纹瘤患儿为研究对象,按照患儿治疗方式的差异将其分为实验组与对照组,每组50例患儿。对照组接受依维莫司治疗,实验组患儿接受雷帕霉素治疗,初始剂量为1 mg/(m^(2)·d),维持血药浓度为5~10μg/L,记录接受治疗后2组患儿心脏横纹肌瘤的大小及数量变化,分析雷帕霉素治疗心脏横纹肌瘤的临床疗效及安全性,并就患儿远期生存效果进行比较。结果实验组患儿治疗后瘤体完全消失率50.00%,缩小50%~100%者32.00%,缩小0%~50%者12.00%,无变化者6.00%;对照组患儿治疗后瘤体完全消失率20.00%,缩小50%~100%者20.00%,缩小0%~50%者36.00%,无变化者20.00%。实验组完全消失率明显高于对照组(P<0.05)。将实验组患儿按照年龄段区分并实施组间比较显示,0~1岁儿童瘤体完全消失率明显高于3岁以上儿童(70.00%vs 21.43%)(P<0.05)。2组患儿治疗前肿瘤最大径组间比较差异不具有统计学意义(P>0.05),治疗后,实验组肿瘤最大直径显著小于对照组(P<0.05),肿瘤消失所用时间也显著短于对照组(P<0.05)。随访2年2组患儿生存率差异不大(P>0.05)。治疗期间2组患儿不良反应发生率比较差异不大(P>0.05)。结论雷帕霉素治疗心脏横纹肌瘤具有较好的效果,瘤体消失或缩小率较高,远期生存率以及治疗安全性值得肯定,0~1岁是儿童最佳治疗时间。

Objective To investigate the clinical effect and long-term survival of rapamycin in the treatment of cardiac rhabdomoma.Methods 100 children with cardiac rhabdomoma were selected as the research objects,according to the difference of treatment methods,they were divided into the experimental group and the control group,50 children in each group.The control group was treated with everolimus,the experimental group was treated with rapamycin,the initial dose was 1mg/(m^(2)·D),and the blood concentration was maintained at 5~10μg/L,The changes of the size and quantity of cardiac rhabdomyoma in the 2 groups after treatment were recorded,the clinical efficacy and safety of rapamycin in the treatment of cardiac rhabdomyoma were analyzed,and the long-term survival effects of chlidren were compared.Results In the experimental group,the tumor completely disappeared after treatment,50.00%,50%~100%reduction accounted for 32.00%,0%~50%reduction accounted for 12.00%,and no change accounted for 6.00%.In the control group,the tumor completely disappeared after treatment,20.00%,50%~100%reduction accounted for 20.00%,0%~50%reduction accounted for 36.00%,and no change accounted for 20.00%.The complete disappearance rate of the experimental group was significantly higher than that of the control group(P<0.05).The children in the experimental group were divided according to their age groups and the comparison between the groups showed that the complete disappearance rate of tumors in children between 0 and 1 year old was significantly higher than that in children>3 years old(70.00%vs 21.43%)(P<0.05).There was no significant difference in the maximum tumor diameter between the two groups before treatment(P>0.05).After treatment,the maximum diameter of tumor in the experimental group was significantly smaller than that in the control group(P<0.05),and the time taken for the tumor to disappear was also significantly shorter than that in the control group(P<0.05).There was little difference in the survival rate of children in the 2 groups during the 2-year follow-up(P>0.05).During the treatment period,there was little difference in the incidence of adverse reactions between the 2 groups(P>0.05).Conclusion Rapamycin has a good effect in the treatment of cardiac rhabdomyomas,the long-term survival rate and the safety of treatment are positive.The best treatment time is 0~1 year old children.