Glucocorticoid therapy for Duchenne cardiomyopathy: A Hobson's choice?
Glucocorticoid therapy for Duchenne cardiomyopathy: A Hobson's choice?
复制标题
糖皮质激素治疗杜氏心肌病：霍布森的选择？
DOI：
10.1161/jaha.115.001896
复制
发表时间：
2015
影响因子：
5.4
通讯作者：
Cripe,LindaH
中科院分区：
文献类型：
--
作者：
Raman,SubhaV;Cripe,LindaH
好像接受杜氏肌营养不良症（DMD）的诊断还不够可怕，考虑一下当父母得知这种疾病对儿子心脏的渐进和无情的影响时的影响。2.不懈地寻找有效的治疗方法来对抗无情的心肌病发作。那些能够在致力于跨学科DMD护理的中心获得消息灵通的团队的人可能会得到及时和敏感的心脏受累筛查，并使用血管紧张素转换酶抑制剂和b受体阻滞剂等药物，这些药物对广泛的心肌疾病有益。3，4那些无法进入这些中心的人可能不得不自己教育经验不足的临床医生进行适当的诊断测试和药物治疗，并从倡导组织或他们自己的网络搜索中收集证据。对于决策至关重要的是来自高质量临床试验的已发表数据，理想情况下，这些数据是基于具有机械洞察力的临床前研究，这些研究提供了支持或反对特定治疗的证据。在这期JAHA中，Tandon及其同事描述了他们对一个大型单中心DMD患者队列的临床获得的心脏磁共振扫描以及从病历中记录的类固醇使用情况的分析。5报告了两项主要调查结果。首先，他们证实了先前的结果，即年龄增加是左心室收缩功能恶化的危险因素。因为更多的损坏
As if receiving a diagnosis of Duchenne muscular dystrophy (DMD) were not terrible enough, consider the impact when a parent learns of the disease’s progressive and inexorable effects on their son’s heart. 2 A relentless search for effective therapies against relentless cardiomyopathy ensues. Those with access to well-informed teams at centers dedicated to interdisciplinary DMD care may get timely and sensitive screening for cardiac involvement, with institution of agents such as angiotensin-converting enzyme inhibitors and b-blockers that benefit a broad spectrum of myocardial diseases. 3, 4 Those without access to such centers may themselves have to educate less-experienced clinicians on appropriate diagnostic testing and medical therapy, armed with evidence collected from advocacy organizations or their own web-based searches. Crucial to decision-making are published data from high-quality clinical trials, ideally based on mechanistically insightful preclinical investigations, that provide evidence in favor of or against a particular treatment.In this issue of JAHA, Tandon and colleagues describe their analysis of clinically-acquired cardiac magnetic resonance scans in conjunction with steroid use recorded from the medical record in a large, single-center DMD patient cohort. 5 Two major findings are reported. First, they confirm prior results showing that increasing age is a risk factor for worsening left ventricular systolic function. As more damaged