Prognostic impact of left ventricular noncompaction in patients with Duchenne/Becker muscular dystrophy — Prospective multicenter cohort study

Neuromuscular comorbidity, heart failure, and atrial fibrillation as prognostic factors in left ventricular hypertrabeculation/noncompaction

“…Various specific NMDs such as Becker muscular dystrophy, Barth syndrome, mitochondriopathy, dystrobrevinopathy, myotonic dystrophy type 1 and type 2, zaspopathy, laminopathy, myoadenylatedeaminase deficiency, Duchenne muscular dystrophy, Charcot-Marie-Tooth disease 1A, inclusion-body myopathy, and oculopharyngodistal myopathy have been described in association with LVHT [11,[18][19][20]. It is unknown if this association is coincidental or causal.…”

“…That LVHT patients do not suffer from cardiac problems only is also reflected by the causes of death: Only 41 % died because of cardiac reasons; in the remaining cases, dysfunction of other organs led to death. Recently, it was shown that among patients with Duchenne/Becker muscular dystrophy those with LVHT had a worse prognosis than those without LVHT [11]. (4) NMD patients frequently have reduced or missing mobility and thus perform only a reduced amount of cardiovascular training.…”

“…The data on the longterm prognosis of LVHT derive from relatively small series with short observation periods (. Table 1) [2][3][4][5][6][7][8][9][10][11]. If systematically screened, LVHT is associated with neuromuscular disorders (NMDs) in the majority of the cases [12].…”

Therapeutic Strategy for Heart Failure in Becker Muscular Dystrophy

“…7) In terms of the corticosteroid treatment, associations with 1) glucocorticoid description, timing of initiation, and dosage, 2) genetic background of dystrophine gene mutation, 3) left ventricular noncompaction, 4) renal function, and 5) respiratory function or nocturnal ventilation are remaining issues that need to be addressed. 3,7,[24][25][26] Further studies to optimize treatment for improving prognosis and quality of life in patients with BMD are necessary in the near future. …”

“…Although respiratory failure was previously the major cause of death, heart failure and life threatening arrhythmia have now emerged as the leading causes of death in dystrophic patients. 2,3) Nevertheless, it seems that cardiac managements remain highly variable and generally undertreated. [4][5][6] Further understanding and optimization of treatment strategies for heart failure are of critical importance to improve prognosis and quality of life in patients with BMD.…”