| Abstrakt: |
With the advancements in medical care for Duchenne Muscular Dystrophy (DMD), particularly the broad implementation of home respiratory therapy, the average lifespan of this patient group has extended to 20-30 years. Currently, the most common cause of mortality in DMD is heart failure due to cardiomyopathy, only followed by respiratory failure. Dilated cardiomyopathy (DCM) in DMD results from dystrophin dysfunction and leads to heart failure (HF). Recommendations for DMD patients (Birkrant 2018) suggest that HF treatment should be conducted by general standards of HF treatment in other types of DCM (D'Amario 2017). At the same time, the potentially significant role of prophylactic, early introduction of pharmacotherapy for the prevention of DCM and HF in DMD is emphasized. The most evidence for the effectiveness of prevention is available for angiotensin-converting enzyme inhibitors (ACEi; Duboc 2005, Duboc 2007, Porcher 2021), hence drugs from this group are commonly used in the first decade of life in DMD patients, regardless of the occurrence of DCM, i.e., in prophylaxis. As a part of the standard of care in our unit, ACEi is introduced around the age of 7. Simultaneously, there are singular reports on the prophylactic effectiveness of beta-blockers and aldosterone receptor antagonists (Jefferies 2005, Ploutz 2017, Raman 2017), yet the strength of evidence is insufficient to universally recommend these drugs for prophylactic use. This points to the necessity of identifying the optimal strategy for prophylaxis and treatment of heart diseases in DMD patients through further research, especially prospective, randomized, double-blind clinical trials. [ABSTRACT FROM AUTHOR] |
