Department of Cardiology, Cardiovascular Centre, Korea University Anam Hospital, Korea University College of Medicine, Seoul, Korea.
Department of Internal Medicine, Yonsei University College of Medicine, Seoul, Korea.
ESC Heart Fail. 2022 Aug;9(4):2199-2206. doi: 10.1002/ehf2.13970. Epub 2022 May 17.
Despite advances in contemporary cardiopulmonary therapies, cardiomyopathy remains the leading cause of death in patients with Duchenne muscular dystrophy (DMD). Also, the long-term clinical outcomes of patients with DMD and cardiomyopathy is unknown. This study investigated long-term clinical outcomes and their associated factors in patients with late-stage DMD.
A total of 116 patients with late-stage DMD (age > 15 years) were enrolled in this retrospective study. All enrolled patients were followed up at a single tertiary referral hospital. LV systolic dysfunction was dichotomously defined as reduced [left ventricular ejection fraction (LVEF) ≤ 40%] vs. preserved [>40%] based on the initial echocardiographic result. The primary endpoint was all-cause death. The secondary endpoint was a composite event defined as death or unexpected hospitalization due to cardiovascular reasons including chest pain, dyspnoea, and generalized oedema. The patients were divided into preserved (n = 84, 72.4%) and reduced LVEF groups (n = 32, 27.6%). The mean age was 20.8 ± 5.9 years, the mean disease duration, 8.8 ± 3.7 years, and the mean follow-up duration, 1708 ± 659 days. For primary endpoint, the reduced LVEF group showed a lower rate of overall survival (Reduced LVEF vs. Preserved LVEF; 81.3% vs. 98.8%, log-rank P = 0.005). In the multivariable Cox regression analysis, brain-natriuretic peptide (BNP) level (adjusted hazard ratio [HR] 1.088, 95% confidence interval [CI] 1.019-1.162, P = 0.011) and diuretic use (adjusted HR 9.279, 95%CI 1.651-52.148, P = 0.011) were significant predictors of all-cause death in patients with DMD. For the secondary endpoint, the reduced LVEF group had a lower rate of freedom from composite events than the preserved LVEF group (65.6% vs. 86.9%, log-rank P = 0.005). In the multivariable Cox regression analysis, BNP level (adjusted HR 1.057, 95%CI 1.005-1.112, P = 0.032) and diuretic use (adjusted HR 4.189, 95% CI 1.704-10.296, P = 0.002) were significant predictors of the composite event in patients with DMD.
Patients with DMD and reduced LVEF had worse clinical outcomes than those with preserved LVEF. BNP level and diuretic use were associated with adverse clinical outcomes in patients with late-stage DMD, irrespective of LVEF.
尽管当代心肺治疗取得了进展，但心肌病仍然是杜氏肌营养不良症（DMD）患者死亡的主要原因。此外，DMD 合并心肌病患者的长期临床结局尚不清楚。本研究旨在调查晚期 DMD 患者的长期临床结局及其相关因素。
共纳入 116 例年龄>15 岁的晚期 DMD 患者，进行回顾性研究。所有纳入的患者均在一家三级转诊医院接受随访。根据初始超声心动图结果，将左心室收缩功能障碍分为降低（[左心室射血分数（LVEF）≤40%]）与保留（>40%）两类。主要终点为全因死亡。次要终点为定义为死亡或因心血管原因（包括胸痛、呼吸困难和全身性水肿）而意外住院的复合事件。将患者分为保留组（n=84，72.4%）和 LVEF 降低组（n=32，27.6%）。平均年龄为 20.8±5.9 岁，平均病程为 8.8±3.7 年，平均随访时间为 1708±659 天。在主要终点方面，LVEF 降低组的总生存率较低（LVEF 降低组 vs. 保留组；81.3% vs. 98.8%，log-rank P=0.005）。多变量 Cox 回归分析显示，脑钠肽（BNP）水平（调整后的危险比[HR]1.088，95%置信区间[CI]1.019-1.162，P=0.011）和利尿剂使用（调整后的 HR 9.279，95%CI 1.651-52.148，P=0.011）是 DMD 患者全因死亡的显著预测因素。在次要终点方面，LVEF 降低组的复合事件发生率低于保留组（65.6% vs. 86.9%，log-rank P=0.005）。多变量 Cox 回归分析显示，BNP 水平（调整后的 HR 1.057，95%CI 1.005-1.112，P=0.032）和利尿剂使用（调整后的 HR 4.189，95%CI 1.704-10.296，P=0.002）是 DMD 患者复合事件的显著预测因素。
与保留 LVEF 的患者相比，LVEF 降低的 DMD 患者的临床结局更差。BNP 水平和利尿剂使用与晚期 DMD 患者的不良临床结局相关，而与 LVEF 无关。
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