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杜氏肌营养不良症患儿在医疗辅助下有可能保留心脏功能吗?

Is It Possible for Children in Duchenne Muscular Dystrophy to Preserve Cardiac Function with Medical Assistance?

作者信息

Kim Han Geul, Eun Lucy Youngmin, Park Han Ki

机构信息

Division of Pediatric Cardiology, Department of Pediatrics, Yonsei University College of Medicine, Seoul 03722, Korea.

Division of Cardiovascular Surgery, Department of Thoracic and Cardiovascular Surgery, Yonsei University College of Medicine, Seoul 03722, Korea.

出版信息

Children (Basel). 2020 Nov 22;7(11):249. doi: 10.3390/children7110249.

Abstract

In patients with Duchenne muscular dystrophy (DMD), death secondary to cardiac or respiratory failure typically occurs in the second or third decade without treatment. Although cardiac dysfunction is treated with standard heart-failure strategies, it remains insufficient in DMD children. The purpose of this study was to evaluate the efficiency of cardiac medication and noninvasive ventilator support in DMD cardiomyopathy children with analyzing echocardiographic data. Forty-eight DMD children patients were included and divided into 2 groups by left ventricular (LV) ejection fraction (EF) at the time of initial treatment. Group 1: LV EF ≥ 45% and Group 2: LV EF < 45%. -values were calculated using a Linear mixed model to estimate the association between cardiac medications and echocardiographic measurements. Before and after cardiac medications, the change values were significantly different in interventricular septal thickness at end diastole (IVSd), interventricular septal thickness at end systole (IVSs), left ventricular internal diameter end systole (LVIDs), left ventricular posterior wall thickness end diastole (LVPWd), ejection fraction (EF), fractional shortening (FS), deceleration time (DT), DT slope, Lat A' and Lat E/E' ( < 0.05). Group 2 patients revealed to take more kinds of cardiac medications than Group 1 ( < 0.05) including ACEIs, beta-blocker, and inotropics, then LV EF was better preserved in Group 2 than Group 1. It is certainly helpful to take individualized medical combination therapy including inotropic agents for cardiomyopathy in DMD children patients with EF < 45%.

摘要

在杜氏肌营养不良症(DMD)患者中,未经治疗的情况下,继发于心脏或呼吸衰竭的死亡通常发生在第二或第三个十年。尽管采用标准的心力衰竭治疗策略来治疗心脏功能障碍,但对于DMD患儿来说仍然不够。本研究的目的是通过分析超声心动图数据来评估心脏药物和无创通气支持对DMD心肌病患儿的疗效。纳入了48例DMD患儿患者,并根据初始治疗时的左心室(LV)射血分数(EF)分为2组。第1组:LV EF≥45%;第2组:LV EF<45%。使用线性混合模型计算P值,以估计心脏药物与超声心动图测量值之间的关联。在使用心脏药物前后,舒张末期室间隔厚度(IVSd)、收缩末期室间隔厚度(IVSs)、收缩末期左心室内径(LVIDs)、舒张末期左心室后壁厚度(LVPWd)、射血分数(EF)、缩短分数(FS)、减速时间(DT)、DT斜率、Lat A'和Lat E/E'的变化值有显著差异(P<0.05)。第2组患者服用的心脏药物种类比第1组更多(P<0.05),包括ACEIs、β受体阻滞剂和正性肌力药物,然后第2组的LV EF比第1组保存得更好。对于EF<45%的DMD患儿心肌病患者,采用包括正性肌力药物在内的个体化药物联合治疗肯定是有帮助的。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fb96/7700218/4ec1b73951d0/children-07-00249-g001.jpg

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