Is It Possible for Children in Duchenne Muscular Dystrophy to Preserve Cardiac Function with Medical Assistance?

Abstract

In patients with Duchenne muscular dystrophy (DMD), death secondary to cardiac or respiratory failure typically occurs in the second or third decade without treatment. Although cardiac dysfunction is treated with standard heart-failure strategies, it remains insufficient in DMD children. The purpose of this study was to evaluate the efficiency of cardiac medication and noninvasive ventilator support in DMD cardiomyopathy children with analyzing echocardiographic data. Forty-eight DMD children patients were included and divided into 2 groups by left ventricular (LV) ejection fraction (EF) at the time of initial treatment. Group 1: LV EF ≥ 45% and Group 2: LV EF < 45%. p-values were calculated using a Linear mixed model to estimate the association between cardiac medications and echocardiographic measurements. Before and after cardiac medications, the change values were significantly different in interventricular septal thickness at end diastole (IVSd), interventricular septal thickness at end systole (IVSs), left ventricular internal diameter end systole (LVIDs), left ventricular posterior wall thickness end diastole (LVPWd), ejection fraction (EF), fractional shortening (FS), deceleration time (DT), DT slope, Lat A’ and Lat E/E’ (p < 0.05). Group 2 patients revealed to take more kinds of cardiac medications than Group 1 (p < 0.05) including ACEIs, beta-blocker, and inotropics, then LV EF was better preserved in Group 2 than Group 1. It is certainly helpful to take individualized medical combination therapy including inotropic agents for cardiomyopathy in DMD children patients with EF < 45%.

Anti-Remodeling Cardiac Therapy in Patients With Duchenne Muscular Dystrophy, Meta-Analysis Study

“…Twelve studies were included in our meta-analysis ( Duboc et al, 2005 ; Jefferies et al, 2005 ; Kajimoto et al, 2006 ; Rhodes et al, 2008 ; Matsumura et al, 2010 ; Viollet et al, 2012 ; Raman et al, 2015 ; Silva et al, 2017 ; Aikawa et al, 2019 ; Adorisio et al, 2019 ; Dittrich et al, 2019 ; Kim et al, 2020 ). Four studies were RCTs, ( Duboc et al, 2005 ; Raman et al, 2015 ; Silva et al, 2017 ; Dittrich et al, 2019 ) seven were observational, ( Jefferies et al, 2005 ; Kajimoto et al, 2006 ; Rhodes et al, 2008 ; Viollet et al, 2012 ; Adorisio et al, 2019 ; Aikawa et al, 2019 ; Kim et al, 2020 )and one study was open-label. Study characteristics are summarized in Table 1 ( Matsumura et al, 2010 ).…”

“…From the ACEi class, the tested medications were: Enalapril, ( Jefferies et al, 2005 ; Kajimoto et al, 2006 ; Silva et al, 2017 ; Aikawa et al, 2019 ; Dittrich et al, 2019 ; Kim et al, 2020 ) Perindopril, ( Duboc et al, 2005 ) Lisinopril, ( Jefferies et al, 2005 ; Viollet et al, 2012 ) Captopril, ( Jefferies et al, 2005 ) and Cilazapril ( Kajimoto et al, 2006 ; Aikawa et al, 2019 ; Kim et al, 2020 ).…”

“…Seven studies examined pharmacotherapy’s effect on LVEF% changes from baseline ( Jefferies et al, 2005 ; Rhodes et al, 2008 ; Viollet et al, 2012 ; Silva et al, 2017 ; Adorisio et al, 2019 ; Aikawa et al, 2019 ; Kim et al, 2020 ). Viollet et al (2012) performed separate analyses for those who received only ACEi and those who received a combination of ACEi + BBs.…”

“…Twelve studies were included in our meta-analysis (Duboc et al, 2005;Jefferies et al, 2005;Kajimoto et al, 2006;Rhodes et al, 2008;Matsumura et al, 2010;Viollet et al, 2012;Raman et al, 2015;Silva et al, 2017;Aikawa et al, 2019;Adorisio et al, 2019;Dittrich et al, 2019;Kim et al, 2020). Four studies were In total, 439 patients were included in the meta-analysis, of which 286 DMD patients were treated for HF (BBs, ACEi, Eplerenone, Ivabradine, and combination, as described in Table 1).…”

“…RCTs,(Duboc et al, 2005;Raman et al, 2015;Silva et al, 2017;Dittrich et al, 2019) seven were observational,(Jefferies et al, 2005;Kajimoto et al, 2006;Rhodes et al, 2008;Viollet et al, 2012;Adorisio et al, 2019;Aikawa et al, 2019;Kim et al, 2020) and one study was open-label. Study characteristics are summarized in Table 1(Matsumura et al, 2010).…”