A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy.
A randomized, double-blind trial of lisinopril and losartan for the treatment of cardiomyopathy in duchenne muscular dystrophy.
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DOI：
10.1371/currents.md.2cc69a1dae4be7dfe2bcb420024ea865
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发表时间：
2013-12-12
期刊：
影响因子：
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通讯作者：
Mendell, Jerry R
中科院分区：
文献类型：
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作者：
Allen, Hugh D;Flanigan, Kevin M;Mendell, Jerry R
目的：本研究旨在比较血管紧张素转换酶抑制剂（ACE-I）（赖诺普利）与血管紧张素受体阻滞剂（ARB）（氯沙坦）治疗Duchenne型肌营养不良症（DMD）男孩心肌病（CM）的有效性和安全性。ACE-I和ARB都被认为是有效的治疗选择。ARB与DMD小鼠模型中的骨骼肌再生有关。问题是，如果两者之一，是更有效的CM治疗DMD仍然存在。本多中心双盲前瞻性研究的目的是比较赖诺普利与氯沙坦在治疗新诊断的CM男孩DMD.METHODS的疗效和安全性：超声心动图技术员间和观察者内变异性进行了测试，在2个不同的日子2个不同的男孩DMD CM。结果采用配对t检验进行比较。22例新诊断DMD CM（超声心动图射血分数（EF）> 10% EF下降）的男孩。由于研究资金终止，aCE-I组的3名男孩接受了3次访视。2例因EF低而退出。只要他们留在研究中，他们的所有数据都被纳入分析中。基线时平均EF相似（47.5%- ACE-I，48.4%- ARB）。1年后，各组分别显著改善至54.6%和55.2%（p= 0.02）。有两个治疗组之间没有差异，在1 year.CONCLUSIONS：观察员间和观察员内的可靠性研究表明，超声心动图之间没有差异的系列检查。两组EF改善情况相同。在治疗DMD相关CM的男孩的一年期间，赖诺普利和氯沙坦之间的EF改善没有治疗差异。试验注册：ClinicalTrials.gov NCT 01982695。
OBJECTIVES: This study sought to compare the effectiveness and safety of an angiotensin converting enzyme inhibitor (ACE-I) (lisinopril) vs. an angiotensin receptor blocker (ARB) (losartan) for the treatment of cardiomyopathy (CM) in boys with Duchenne muscular dystrophy (DMD).BACKGROUND: Development of CM is universal in boys with DMD. ACE-I and ARB have both been suggested as effective treatment options. ARBs have been associated with skeletal muscle regeneration in a mouse model of DMD. The question of which, if either, is more effective for CM treatment in DMD remains. The purpose of this multicenter double-blind prospective study was to compare efficacy and safety of lisinopril versus losartan in the treatment of newly diagnosed CM in boys with DMD.METHODS: Echocardiographic technician inter- and intraobserver variability were tested on 2 separate days on 2 different boys with DMD CM. Results were compared with paired t-testing. Twenty-two boys with newly diagnosed DMD CM (echocardiographic ejection fraction (EF) 10% EF drop. Three boys in the aCE-I group had 3 visits, due to study funding termination. Two were withdrawn because of low EF. All their data are included in the analysis for as long as they remained in the study. Mean EF's were similar at baseline (47.5%- ACE-I, 48.4%- ARB). After 1 year each group significantly improved to 54.6% and 55.2% respectively (p=.02). There was no difference between the 2 treatment groups at 1 year.CONCLUSIONS: Inter-observer and intra-observer reliability studies showed no differences between echocardiographers on serial examinations. EF improved equally in the two groups. There is no therapeutic difference in EF improvement between lisinopril and losartan over the one-year duration for treatment of boys with DMD-related CM.TRIAL REGISTRATION: ClinicalTrials.gov NCT01982695.