title: Dehydroepiandrosterone for myotonic dystrophy type 1.
abstract: BACKGROUND
  Myotonic dystrophy type 1 may be associated with low circulating dehydroepiandrosterone (DHEA) levels. This study was aimed at investigating the efficacy and safety of DHEA in myotonic dystrophy type 1 patients.
  
  
  METHODS
  This was a prospective, multicenter, randomized, double-blind, placebo-controlled trial conducted from February 2005 to January 2006 at 10 university-affiliated neuromuscular disease centers in France. Seventy-five ambulatory adults with myotonic dystrophy type 1 received an oral replacement dose (100 mg/d) or a pharmacologic dose (400 mg/d) of DHEA, or placebo. The primary endpoint was the relative change in the manual muscle testing (MMT) score from baseline to week 12. Secondary outcome measures included changes from baseline to week 12 in quantitative muscle testing and timed functional testing, respiratory and cardiac function, and quality of life. This study was registered with ClinicalTrials.gov identifier NCT00167609.
  
  
  RESULTS
  The median (1st, 3rd quartile) relative changes in MMT score from baseline to week 12 after randomization were 3.1 (-0.9, 6.7), 1.9 (-2.7, 3.5), and 2.2 (0, 7.9), in the DHEA 100 mg, DHEA 400 mg, and placebo groups, respectively. There were no differences between placebo and combined DHEA groups (p = 0.34), placebo and DHEA 100 mg (p = 0.86), or placebo and DHEA 400 mg (p = 0.15). There were also no evidence for a difference between groups for the changes from baseline to week 12 in any secondary outcome.
  
  
  CONCLUSIONS
  There is no evidence that a 12-week treatment with replacement or pharmacologic doses of dehydroepiandrosterone improves muscle strength in ambulatory myotonic dystrophy type 1 patients.
authors.affiliation: Centre de référence Maladies Neuromusculaires Nantes-Angers, Département de Neurologie, Centre Hospitalier Universitaire d'Angers, France.
authors.forename: I
authors.initials: I
authors.lastname: Pénisson-Besnier
authors.forename: M
authors.initials: M
authors.lastname: Devillers
authors.forename: R
authors.initials: R
authors.lastname: Porcher
authors.forename: D
authors.initials: D
authors.lastname: Orlikowski
authors.forename: V
authors.initials: V
authors.lastname: Doppler
authors.forename: C
authors.initials: C
authors.lastname: Desnuelle
authors.forename: X
authors.initials: X
authors.lastname: Ferrer
authors.forename: M-C A
authors.initials: MC
authors.lastname: Bes
authors.forename: F
authors.initials: F
authors.lastname: Bouhour
authors.forename: C
authors.initials: C
authors.lastname: Tranchant
authors.forename: E
authors.initials: E
authors.lastname: Lagrange
authors.forename: A
authors.initials: A
authors.lastname: Vershueren
authors.forename: D
authors.initials: D
authors.lastname: Uzenot
authors.forename: P
authors.initials: P
authors.lastname: Cintas
authors.forename: G
authors.initials: G
authors.lastname: Solé
authors.forename: J-Y
authors.initials: JY
authors.lastname: Hogrel
authors.forename: P
authors.initials: P
authors.lastname: Laforêt
authors.forename: C
authors.initials: C
authors.lastname: Vial
authors.forename: A L
authors.initials: AL
authors.lastname: Vila
authors.forename: S
authors.initials: S
authors.lastname: Sacconi
authors.forename: J
authors.initials: J
authors.lastname: Pouget
authors.forename: B
authors.initials: B
authors.lastname: Eymard
authors.forename: S
authors.initials: S
authors.lastname: Chevret
authors.forename: D
authors.initials: D
authors.lastname: Annane
mesh_terms: D000328:Adult; D003687:Dehydroepiandrosterone / Q000009:adverse effects / Q000627:therapeutic use*; D004311:Double-Blind Method; D023381:Endpoint Determination; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D018482:Muscle, Skeletal / Q000187:drug effects / Q000503:physiopathology; D009223:Myotonic Dystrophy / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D011446:Prospective Studies; D011788:Quality of Life
chemical_list: D003687:Dehydroepiandrosterone