The effectiveness of Deflazacort (DFZ) against prednisone was tested in Duchenne muscular dystrophy (DMD) patients, and positive results were reported at the 2016 MDA Clinical Conference in Arlington, Virginia, in a presentation by Marathon Pharmaceuticals titled “Effect of Deflazacort and Prednisone on Muscle Enzymes in the Treatment of Duchenne Muscular Dystrophy.”
Pharmacologic therapy in DMD relies on glucocorticoids (GCs) to increase 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. 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 or 1.2 mg/kg/day, prednisone 0.75 mg/kg/day, or to placebo, and followed for a whole year. By assessing muscle strength using a modified Medical Research Council Scale, the team observed that both doses of deflazacort and prednisone were superior to placebo at 12 weeks in improving muscle strength.
A typical feature of DMD patients, frequently 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. Researchers observed that both doses of DFZ and prednisone significantly reduced 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 further confirmed that DFZ-treated DMD patients gained significantly less weight compared to prednisone. Importantly, DFZ treated patients had less glucocorticoid-related adverse effects. These patients had a lower incidence of cushingoid-type syndrome, which causes high blood pressure and abdominal obesity, and also less psychiatric disturbances. Overall, DFZ showed several advantages compared to prednisone and could 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 meeting set for April in Vancouver, Canada.