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The association between cardiac involvement and long-term clinical outcomes in patients with Duchenne muscular dystrophy

 Jung-Joon Cha  ;  In-Soo Kim  ;  Jong-Youn Kim  ;  Eui-Young Choi  ;  Pil-Ki Min  ;  Young Won Yoon  ;  Byoung Kwon Lee  ;  Bum-Kee Hong  ;  Hyuck Moon Kwon  ;  Han Eol Cho  ;  Won Ah Choi  ;  Seong-Woong Kang  ;  Se-Joong Rim 
 ESC HEART FAILURE, Vol.9(4) : 2199-2206, 2022-08 
Journal Title
Issue Date
Adolescent ; Adult ; Cardiomyopathies* ; Diuretics / therapeutic use ; Humans ; Muscular Dystrophy, Duchenne* / complications ; Retrospective Studies ; Stroke Volume ; Ventricular Dysfunction, Left* ; Ventricular Function, Left ; Young Adult
Brain-natriuretic peptide ; Diuretics ; Duchenne muscular dystrophy ; LV systolic dysfunction ; Long-term clinical outcome
Aims: 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.

Methods and results: 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.

Conclusions: 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.
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1. College of Medicine (의과대학) > Dept. of Internal Medicine (내과학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Rehabilitation Medicine (재활의학교실) > 1. Journal Papers
Yonsei Authors
Kang, Seong Woong(강성웅) ORCID logo https://orcid.org/0000-0002-7279-3893
Kwon, Hyuck Moon(권혁문) ORCID logo https://orcid.org/0000-0001-9901-5015
Kim, In-Soo(김인수) ORCID logo https://orcid.org/0000-0003-2801-5514
Kim, Jong Youn(김종윤) ORCID logo https://orcid.org/0000-0001-7040-8771
Min, Pil Ki(민필기) ORCID logo https://orcid.org/0000-0001-7033-7651
Yoon, Young Won(윤영원) ORCID logo https://orcid.org/0000-0002-0907-0350
Lee, Byoung Kwon(이병권) ORCID logo https://orcid.org/0000-0001-9259-2776
Rim, Se Joong(임세중) ORCID logo https://orcid.org/0000-0002-7631-5581
Cho, Han Eol(조한얼) ORCID logo https://orcid.org/0000-0001-5625-3013
Cha, Jung-Joon(차정준) ORCID logo https://orcid.org/0000-0002-8299-1877
Choi, Won Ah(최원아) ORCID logo https://orcid.org/0000-0003-0403-8869
Choi, Eui Young(최의영) ORCID logo https://orcid.org/0000-0003-3732-0190
Hong, Bum Kee(홍범기) ORCID logo https://orcid.org/0000-0002-6456-0184
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