Kisel Janneta, Ballard Emily, Suh Eui-Sik, Hart Nicholas, Kapetanakis Stam, Srivastava Shelley, Marino Philip, Murphy Patrick, Steier Joerg
Lane Fox Unit, Guy's and St Thomas' NHS Trust, London, UK.
Faculty of Life Sciences and Medicine, King's College London, London, UK.
J Thorac Dis. 2023 Feb 28;15(2):812-819. doi: 10.21037/jtd-22-1528. Epub 2023 Feb 8.
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder characterised by progressive muscle wasting impacting mobility, ventilation and cardiac function. Associated neuromuscular cardiomyopathy remains a major cause of morbidity and mortality. We investigated the effects of cardioprotective medications [angiotensin-converting enzyme inhibitors (ACE-I), beta-blockers] on clinical outcomes in DMD patients.
This was a retrospective cohort study (reference: 2021/12469) of DMD patients at a tertiary centre between 1993-2021 screening the electronic records for demographics, comorbidities, medication, disease specific features, echocardiography, hospitalisations, and ventilator use.
A total of 68 patients were identified aged 27.4 (6.6) years, of which 52 were still alive. There was a difference in body mass index (BMI) between survivors and deceased patients [23.8 (5.9) 19.9 (3.8) kg/m, P=0.03]. Home mechanical ventilation (HMV) was required in 90% of patients, 85% had DMD associated cardiomyopathy. About 2/3 of all hospitalisations during the observation period were secondary to cardiopulmonary causes. The left ventricular ejection fraction (LVEF) at initial presentation was 44.8% (10.6%) and declined by 3.3% [95% confidence interval (CI): 0.4% to -7.0%] over the follow up period (P=0.002). A total of 61 patients were established on ACE-I for 75.9% (35.1%), and 62 were on beta-blockers for 73.6% (33.5%) of the follow up period. There was a significant LVEF decline in those taking ACE-I for limited periods compared to those permanently on ACE-I (P=0.002); a similar effect was recorded with beta-blockers (P=0.02).
Long-term use of ACE-I and beta-blockers is associated with a reduced decline in LVEF in patients with DMD and may be protective of adverse cardiovascular ill health.
杜氏肌营养不良症（DMD）是一种神经肌肉疾病，其特征为进行性肌肉萎缩，影响运动能力、通气功能和心脏功能。相关的神经肌肉性心肌病仍然是发病和死亡的主要原因。我们研究了心脏保护药物[血管紧张素转换酶抑制剂（ACE-I）、β受体阻滞剂]对DMD患者临床结局的影响。
这是一项对一家三级中心1993年至2021年期间的DMD患者进行的回顾性队列研究（参考文献：2021/12469），筛查电子记录中的人口统计学、合并症、用药情况、疾病特定特征、超声心动图、住院情况和呼吸机使用情况。
共确定68例患者，年龄为27.4（6.6）岁，其中52例仍存活。存活患者和死亡患者的体重指数（BMI）存在差异[23.8（5.9）对19.9（3.8）kg/m²，P = 0.03]。90%的患者需要家庭机械通气（HMV），85%的患者患有DMD相关心肌病。在观察期内，约2/3的住院是由心肺原因引起的。初次就诊时左心室射血分数（LVEF）为44.8%（10.6%），在随访期间下降了3.3%[95%置信区间（CI）：0.4%至-7.0%]（P = 0.002）。共有61例患者在随访期间的75.9%（35.1%）使用ACE-I，62例患者在随访期间的73.6%（33.5%）使用β受体阻滞剂。与长期使用ACE-I的患者相比，短期使用ACE-I的患者LVEF显著下降（P = 0.002）；β受体阻滞剂也有类似效果（P = 0.02）。
长期使用ACE-I和β受体阻滞剂与DMD患者LVEF下降减少有关，可能对不良心血管健康具有保护作用。
