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A Randomized Controlled Trial

Ernesto A. C. Beenakker, MD; Johanna M. Fock, MD; Marja J. Van Tol, MD; Natalia M. Maurits, PhD; Hendrik M. Koopman, PhD; Oebele F. Brouwer, MD, PhD; Johannes H. Van der Hoeven, MD, PhD

Arch Neurol. 2005;62:128-132.

Background  Prednisone 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.

Objectives  To 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.

Design  A 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.

Setting  University hospital and rehabilitation center in the Netherlands.

Patients  Seventeen ambulant patients with DMD aged 5 to 8 years.

Main Outcome Measure  Change 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.

Results  The 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.

Conclusions  Prednisone 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.

Author Affiliations: Department of Neurology, University Hospital Groningen, Groningen (Drs Beenakker, Fock, Maurits, Brouwer, and Van der Hoeven), Rehabilitation Centre "De Hoogstraat," Utrecht (Dr Van Tol), and Department of Paediatrics, Leiden University Medical Center, Leiden (Dr Koopman), the Netherlands.

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