Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study.

ABSTRACT:

Objective

We 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).

Methods

We 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 χ(2) test.

Results

Participants treated ≥1 year while ambulatory (n = 252/340) showed a 3-year median delay in LoA (p < 0.001). Fourteen different regimens were observ

SUBMITTER: Bello L 

PROVIDER: S-EPMC4603595 | biostudies-literature | 2015 Sep

REPOSITORIES: biostudies-literature