Deflazcor as a Duchenne Muscular Dystrophy Therapy Reported to Be Superior to Prednisone in Clinical Study

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DMD treatment

The effectiveness of Deflazacort (DFZ) againstĀ prednisone was tested in Duchenne muscular dystrophy (DMD) patients, and positive resultsĀ were reportedĀ at the 2016 MDA Clinical Conference in Arlington, Virginia, in a presentation by Marathon Pharmaceuticals titled ā€œEffect of Deflazacort and Prednisone on Muscle Enzymes in the Treatment of Duchenne Muscular Dystrophy.ā€

Pharmacologic therapy in DMD relies on glucocorticoids (GCs) to increaseĀ muscle strength and consequently control disease symptoms. DFZ is a heterocyclic glucocorticoid that is biologically inactive, but when metabolized has been shown to provide anti-inflammatory and immunosuppressant effects.Ā When compared to prednisone, it also results in less weight gain.

The study enrolled 196 boys between the ages of 5 and 15 with an onset of muscle weakness before 5 years of age. Patients were randomized to DFZ at either 0.9 mg/kg/day orĀ 1.2 mg/kg/day, prednisone 0.75 mg/kg/day, or to placebo, and followed forĀ a wholeĀ year. By assessing muscle strength usingĀ a modified Medical Research Council Scale, the team observedĀ that both doses of deflazacort and prednisone were superior to placebo at 12 weeks inĀ improving muscle strength.

A typical feature of DMD patients, frequentlyĀ used for diagnosis, are elevated levels of creatine phosphokinase (CPK) as a result of muscle disease. Moreover, lactate dehydrogenase levels also increase due to liver dysfunction. ResearchersĀ observed that both doses of DFZĀ and prednisone significantlyĀ reducedĀ both these enzymes versus placebo over 12 weeks of treatment, with continued reduction compared to baseline over 52 weeks of treatment. This reduction reflects the anti-inflammatory effect of corticosteroids in the muscle, which normally improves motor strength and function.

The study furtherĀ confirmed that DFZ-treated DMD patients gained significantly less weight compared to prednisone. Importantly, DFZ treated patients hadĀ less glucocorticoid-related adverse effects. These patients had a lower incidence of cushingoid-type syndrome, whichĀ causes high blood pressure and abdominal obesity, and also less psychiatric disturbances.Ā Overall, DFZ showed several advantages compared to prednisone and couldĀ be a better treatment alternative to DMD patients, the researchers reported.

This study will be further discussed at the American Academy of Neurology (AAN) annual meetingĀ set forĀ April inĀ Vancouver, Canada.