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Arch Neurol Chicago · Jan 2005
Randomized Controlled Trial Comparative Study Clinical TrialIntermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial.
- Ernesto A C Beenakker, Johanna M Fock, Marja J Van Tol, Natalia M Maurits, Hendrik M Koopman, Oebele F Brouwer, and Johannes H Van der Hoeven.
- Department of Neurology, University Hospital Groningen, Hanzeplein 1, 9700 RB Groningen, The Netherlands. e.a.c.beenakker@neuro.azg.nl
- Arch Neurol Chicago. 2005 Jan 1;62(1):128-32.
BackgroundPrednisone treatment is used to prolong ambulation in patients with Duchenne muscular dystrophy (DMD). However, since severe adverse effects often accompany prednisone treatment, it is debatable whether the benefits of prednisone treatment outweigh its adverse effects.ObjectivesTo study the effects of prednisone on muscle function and to determine the extent of steroid-related adverse effects and their influence on the quality of life of ambulant patients with DMD.DesignA randomized, placebo-controlled, crossover trial with 6 months of treatment: prednisone or placebo (0.75 mg/kg daily) during the first 10 days of each month. After a washout period of 2 months, patients received the other regimen for an additional 6 months.SettingUniversity hospital and rehabilitation center in the Netherlands.PatientsSeventeen ambulant patients with DMD aged 5 to 8 years.Main Outcome MeasureChange in muscle function assessed by timed functional testing: running 9 m, climbing 4 standard-sized stairs, and rising from the floor to a standing position.ResultsThe increase in time needed to run 9 m (P = .005) and to climb 4 standard-sized stairs (P = .02) was significantly lower during the prednisone period.ConclusionsPrednisone slowed deterioration of muscle function and muscle force in ambulant patients with DMD. Although adverse effects were present, patient quality of life was not affected. Therefore, short-term prednisone treatment can be recommended to preserve motor functions in ambulant patients with DMD.
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