Corticosteroid Regimen Delays Loss of Ambulation in Duchenne Muscular Dystrophy

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Glucocorticoid corticosteroid regimens show promise in delaying loss of independent ambulation (LoA) in patients with Duchenne muscular dystrophy, according to data published in Neurology.

Luca Bello, MD, of the Children’s National Medical Center in Washington, D.C., and colleagues studied 340 patients from the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS) to see how use of prednisone or prednicolone (PRED) or deflazacort (DFZ), including frequency of administration and dosing, effected onset of LoA.

In total, 14 different glucocorticoid corticosteroid regimens were observed, with nondaily treatment common for PRED (37%) and rare fo DFZ (3%). Patients who were treated ≥ one year while ambulatory showed a three-year median delay in LoA. DFZ was linked to later LoA than PRED (hazard ratio 0.294 ± 0.053 vs 0.490 ± 0.08, P = 0.003; 2-year difference in median LoA with daily administration, P < 0.001), and the average dose for daily PRED (0.56 mg/kg/d, 75% of recommended) was lower than daily DFZ (0.75 mg/kg/d, 83% of recommended, p < 0.001).

Those taking DFZ showed later onset of LoA, but increased frequency of side effects, including growth delay, cushingoid appearance, and cataracts.  Results were complicated by differences in standards of care and dosing, which is why the study authors recommend that a randomized, blinded trial of glucocorticoid corticosteroid regimens be conducted for Duchenne muscular dystrophy. 

muscular dystrophy
Corticosteroid Regimen Delays Loss of Ambulation in Duchenne Muscular Dystrophy

Researchers aimed to perform an observational study of age at loss of independent ambulation (LoA) and side-effect profiles associated with different glucocorticoid corticosteroid (GC) regimens in Duchenne muscular dystrophy (DMD).

Luca Bello, MD, of the Children's National Medical Center in Washington, D.C., and colleagues studied 340 participants in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use. Effects of prednisone or prednisolone (PRED)/deflazacort (DFZ), administration frequency, and dose were analyzed by time-varying Cox regression. Side-effect frequencies were compared using χ2 test.

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