title: Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up.
abstract: BACKGROUND
  Corticosteroid treatment is the standard of care in Duchenne muscular dystrophy (DMD), but the optimal age to initiate treatment and dosage pattern remain a matter of discussion.
  
  
  METHODS
  We performed a long-term study of alternate-day corticosteroids in five 2- to 4-year-old DMD patients. The primary outcome measure was prolongation of the ability to walk.
  
  
  RESULTS
  One patient lost ambulation at age 10. Four patients, aged 16 to 18 were fully ambulant, and 3 of them could still climb stairs. Respiratory function was moderately reduced in 2. Left ventricular ejection fraction was > 45%. Short stature and delayed puberty were the most relevant side effects. Although the negative impact of corticosteroid treatment on growth rate remained their major concern, parents and patients stated that they preferred corticosteroid therapy.
  
  
  CONCLUSIONS
  Long-term corticosteroid treatment is effective in prolonging function but not in recovering lost function, and its early use seems appropriate.
authors.affiliation: Laboratory of Musculoskeletal Cell Biology, Rizzoli Orthopedic Institute, Bologna, Italy. luciano.merlini@ior.it
authors.forename: Luciano
authors.initials: L
authors.lastname: Merlini
authors.forename: Monia
authors.initials: M
authors.lastname: Gennari
authors.forename: Elisabetta
authors.initials: E
authors.lastname: Malaspina
authors.forename: Ilaria
authors.initials: I
authors.lastname: Cecconi
authors.forename: Annarita
authors.initials: A
authors.lastname: Armaroli
authors.forename: Saverio
authors.initials: S
authors.lastname: Gnudi
authors.forename: Beril
authors.initials: B
authors.lastname: Talim
authors.forename: Alessandra
authors.initials: A
authors.lastname: Ferlini
authors.forename: Alessandro
authors.initials: A
authors.lastname: Cicognani
authors.forename: Emilio
authors.initials: E
authors.lastname: Franzoni
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000009:adverse effects* / Q000494:pharmacology / Q000627:therapeutic use*; D005500:Follow-Up Studies; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology*; D011241:Prednisone / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use; D011282:Pregnenediones / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use; D011446:Prospective Studies; D011628:Puberty, Delayed / Q000139:chemically induced; D013318:Stroke Volume / Q000187:drug effects / Q000502:physiology; D016896:Treatment Outcome; D016138:Walking / Q000502:physiology
chemical_list: D000305:Adrenal Cortex Hormones; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone