Long-term oral hormone therapy delays DMD-related cardiomyopathy

  Studies have shown that in patients with DMD, cardiomyopathy begins at a specific age. This time of onset is related to the type of genetic defect and there are significant individual differences. Therefore, for early detection, regular monitoring of cardiac function is required. Cardiomyopathy does not usually develop until the age of 7-8 years. There is no significant decrease in cardiac function before the onset of cardiomyopathy. However, once cardiomyopathy begins, cardiac function continues to decline, with an absolute decrease in left ventricular ejection fraction of approximately 2.2% per year. In patients with DMD, the rate of decline in myocardial lesions and cardiac function decreased the longer the duration of oral hormone therapy (mean age at initiation of oral hormone therapy in the study was 7 years, median age 6.8 years, and mean length of time on hormone therapy was 7.6 years, median length 7.2 years).  The experts noted that the results of this study were from a retrospective study, not a clinical trial with strictly controlled enrollment conditions and treatment methods, and the reliability of the findings needs to be further verified. However, the results are consistent with previous clinical studies that have concluded that oral hormone therapy can help delay cardiomyopathy. In addition to oral hormone therapy, cardioprotective drugs such as ACEIs, β-blockers, combined αβ-blockers, ARBs, and potassium-protective diuretics have also been shown to slow the progression of DMD cardiomyopathy. The combination of hormones with these drugs may be more effective.  In addition, cardiac magnetic resonance (CMR) is superior to echocardiography (UCG) for the assessment of DMD-related cardiomyopathy. However, UCG has a clear advantage in terms of price, ease of detection and popularity. Therefore, the choice can be specific to different families and at different times.  With the improvement of DMD diagnosis and treatment, the survival of DMD patients continues to be prolonged and the quality of life continues to improve, and more attention is paid to DMD-related cardiomyopathy than ever before. With good early monitoring and optimal treatment, DMD cardiomyopathy is not irrevocable.