However no consensus remains regarding the timing of treatment. Personal experience Our work group has
constantly focused attention to the myocardial involvement in DMD. A compendium of the results obtained in the field can be found in Engel & Franzini-Armstrong’s textbook “Myology” (5). The therapeutic approach of cardiomyopathy has only recently been accepted and is based on the use of ACE inhibitors and beta-blockers to prevent Galunisertib concentration cardiac function Inhibitors,research,lifescience,medical deterioration. Digitalis, diuretics and anticoagulants are used in the acute phases, such as congestive heart failure episodes. We are convinced that the therapeutics are more effective when administered very early in the course of disease – please remember the latin saying “to prevent is better than to cure” – before the fibrosis is established. Dystrophyn plays a critical role in the Inhibitors,research,lifescience,medical myocardium by connecting the cytoskeleton to the external membrane, so that its absence causes membrane fragility, loss of transductional force and myocite necrosis, promoted by mechanical stress (33, 34). The efficacy and the progressive benefit over time of ACEis are consistent with a hemodynamic effect and/or a specific antifibrotic effect of this class of drugs and are concordant with experimental observations made in animal models (35, 36). Long-term therapy with DFZ is also Inhibitors,research,lifescience,medical effective in slowing down the
progression of fibrosis in the dystrophin deficient heart. Our group adopted deflazacort in the treatment of DMD boys since 1990.
In 2004 we published (37) in cooperation with the Toronto group the results of a prolonged Inhibitors,research,lifescience,medical observation on 69 DMD patients, treated for at least 4 years by two different treatment protocols (0.6 mg/kg/day, 20 days on/10 days off [N-Protocol] vs. 0.9 mg/kg/day [T-Protocol]) comparing both the long-term benefits and side effects. With respect to the group of 49 untreated DMD boys, the report illustrated the long-term beneficial effects on muscle function and motor performance Inhibitors,research,lifescience,medical of deflazacort treatment in both protocols. However the high dose protocol (T-protocol) seemed to be more effective but frequently associated with asymptomatic cataracts. In the same year we presented at the Mediterranean Society of Myology Congress the results on cardiac function of a long-term period of observation of 60 DMD boys treated with DFZ at the dosage of 0.6 mg/kg/ day for 20 days/month (38). The mean age at the not enrollment was 5.6 years (range 4-11.7); the follow up was 83.7 months on average (range 36-144 months). All the patients had a fourth-month cardiac evaluation by ECG and echocardiography. The following parameters were evaluated: PQ interval, PQ segment, QT interval, QT dispersion, Cardiomyopathic Index (QT/PQ, adjusted for HR), presence of Arrhythmias or Blocks, presence of T wave anomalies, by the ECG; four chambers dimension, wall thicknesses, Ejection Fraction, Fractional Shortening, ultrasonic integrated backscatter (IBS), by the echocardiogram.
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