title: 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 observed. Nondaily treatment was common for PRED (37%) and rare for DFZ (3%). DFZ was associated with 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). Average dose was lower for daily PRED (0.56 mg/kg/d, 75% of recommended) than daily DFZ (0.75 mg/kg/d, 83% of recommended, p < 0.001). DFZ showed higher frequencies of growth delay (p < 0.001), cushingoid appearance (p = 0.002), and cataracts (p < 0.001), but not weight gain.
  
  
  CONCLUSIONS
  Use of DFZ was associated with later LoA and increased frequency of side effects. Differences in standards of care and dosing complicate interpretation of this finding, but stratification by PRED/DFZ might be considered in clinical trials. This study emphasizes the necessity of a randomized, blinded trial of GC regimens in DMD.
  
  
  METHODS
  This study provides Class IV evidence that GCs are effective in delaying LoA in patients with DMD.
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Luca
authors: L
authors: Bello
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Heather
authors: H
authors: Gordish-Dressman
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Lauren P
authors: LP
authors: Morgenroth
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Erik K
authors: EK
authors: Henricson
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Tina
authors: T
authors: Duong
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Eric P
authors: EP
authors: Hoffman
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC.
authors: Avital
authors: A
authors: Cnaan
authors: From the Children's National Medical Center (L.B., H.G.-D., L.P.M., T.D., E.P.H., A.C.), Washington, DC; University of California Davis Medical Center (E.K.H., C.M.M.), Sacramento, CA; and The George Washington University (E.P.H., A.C.), Washington, DC. cmmcdonald@ucdavis.edu.
authors: Craig M
authors: CM
authors: McDonald
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D006801:Humans; D008297:Male; D055815:Young Adult; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage; D005500:Follow-Up Studies; D005938:Glucocorticoids / Q000008:administration & dosage; D038622:Internationality; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000188:drug therapy; D011239:Prednisolone / Q000008:administration & dosage; D011241:Prednisone / Q000008:administration & dosage; D011282:Pregnenediones / Q000008:administration & dosage
chemical_list: D000893:Anti-Inflammatory Agents; C021988:deflazacort; D005938:Glucocorticoids; D011239:Prednisolone; D011241:Prednisone; D011282:Pregnenediones