A new practice guideline on corticosteroids for children with Duchenne muscular dystrophy (DMD) says 0.75 milligrams/kilogram/day (mg/kg/day) of prednisone offers significant benefit and should be offered to patients to improve strength and pulmonary function.
That recommendation is based on moderate evidence from a review of studies from 2004 to 2014.
Deflazacort also has treatment benefits, the review showed. However, high-quality data are lacking on the long-term efficacy of either medication, according to the guideline.
The AAP-endorsed guideline, Corticosteroid Treatment of Duchenne Muscular Dystrophy, from the American Academy of Neurology, updated from 2005, compares what is known on the drugs’ effects on survival, quality of life, development of scoliosis, and motor, pulmonary and cardiac function in patients with DMD.
Deflazacort also may improve strength and pulmonary function; delay loss of ambulation by 1.4 to 2.5 years; and increase survival over five to 15 years of treatment, but the evidence for these was considered to be weaker.
There was insufficient evidence to show prednisone increased survival.
The medications were equally effective in improving timed motor function, reducing the need for scoliosis surgery and delaying the onset of cardiomyopathy. Not enough evidence was available on the drugs’ impact on cardiac function.
Because significant adverse events can occur with corticosteroids, side effects should be discussed with families in advance and informed consent obtained. Patients and families should have a voice in the corticosteroid chosen, the guideline noted. Adverse effects of corticosteroids include short stature, behavioral changes, fractures and cataracts in patients with DMD, which “should be managed proactively.”
Specifically, prednisone use was found to result in greater weight gain, plus a Cushingoid appearance and hirsutism, while deflazacort was linked to more cataracts, though most are not vision-impairing.
Patients treated with prednisone are advised to receive 0.75 mg/kg/d. An alternate regimen of 10 mg/kg/weekend was equally effective over a 12-month period, but long-term outcomes are unknown. The dose can be reduced to 0.3 mg/kg/d if significant adverse events occur. For deflazacort, evidence was lacking to support a preferred dose.
There also was not enough evidence for bisphosphonates as an added therapy to corticosteroids to increase survival, nor was evidence available to make a recommendation on how to improve bone health. The American College of Rheumatology advises patients taking corticosteroids for three months or longer to receive 1,200 mg/d of calcium and 800 IU/d of vitamin D.
More research is needed on various topics related to treatment of DMD, including whether corticosteroids have an effect on quality of life.