A randomized controlled trial of early surgery in Duchenne muscular dystrophy. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. Night splinting does not increase ankle range of motion in people with Charcot-Marie-Tooth disease: a randomised, cross-over trial.
We performed a randomized controlled trial of early surgical treatment of contractures in 20 boys with Duchenne muscular dystrophy, age 4-6 yr. Surgery consisted of release of hip flexors, removal of iliotibial bands, and lengthening of tendo Achilles bilaterally. All patients were monitored for at least 12 months post-randomization, and assessed quantitatively for muscle strength and function. Surgery corrected the deformities, but had no beneficial effect on strength or function. Indeed, data in the second year showed more rapid deterioration of function in some of the operated boys. There appeared to be continued evolution of pathology following surgery, as assessed by sequential muscle ultrasound and muscle biopsy. We cannot recommend this type of surgery as a routine treatment. We performed a randomized, double-blind, controlled six-month trial of prednisone in 103 boys with Duchenne's muscular dystrophy (age, 5 to 15 years). The patients were assigned to one of three regimens: prednisone, 0.75 mg per kilogram of body weight per day (n = 33); prednisone, 1.5 mg per kilogram per day (n = 34); or placebo (n = 36). The groups were initially comparable in all measures of muscle function. Both prednisone groups had significant improvement of similar degree in the summary scores of muscle strength and function. Improvement began as early as one month and peaked by three months. At six months the high-dose prednisone group, as compared with the placebo group, had improvement in the time needed to rise from a supine to a standing position (3.4 vs. 6.2 seconds), to walk 9 m (7.0 vs. 9.7 seconds), and to climb four stairs (4.0 vs. 7.1 seconds), in lifting a weight (2.1 vs. 1.2 kg), and in forced vital capacity (1.7 vs. 1.5 liters) (P less than 0.001 for all comparisons). There was an increase in urinary creatinine excretion (261 vs. 190 mg per 24 hours), which suggested an increase in total muscle mass. However, the prednisone-treated patients who had required long-leg braces (n = 5) or wheelchairs (n = 11) continued to require them. The most frequent side effects were weight gain, cushingoid appearance, and excessive hair growth. We conclude from this six-month study that prednisone improves the strength and function of patients with Duchenne's muscular dystrophy. However, further research is required to identify the mechanisms responsible for these improvements and to determine whether prolonged treatment with corticosteroids may be warranted despite their side effects. What is the effect of wearing splints at night to stretch the plantarflexors on dorsiflexion range of motion (ROM) in people with Charcot-Marie-Tooth disease?
                Randomised, assessor-blinded, cross-over trial.
                14 people (1 dropout) aged 7 to 30 years with Charcot-Marie-Tooth disease Type 1A and with < or = 15 degrees dorsiflexion range of motion (ROM).
                A splint holding the ankle in maximum dorsiflexion was worn nightly on one leg for 6 weeks followed by the opposite leg for the subsequent 6 weeks.
                The primary outcome was dorsiflexion ROM; secondary outcomes were eversion ROM, and dorsiflexion, eversion, and inversion strength, measured before and after splinting, and three months later.
                There was no significant difference between the experimental and the control intervention in terms of ROM or strength. Wearing the splint at night increased dorsiflexion ROM by 1 degree (95% CI -3 to 4; p = 0.72) and eversion ROM by 1 degree (95% CI -1 to 3; p = 0.28) compared to not wearing the splint. Wearing the splint increased dorsiflexion strength by 41 N (95% CI -53 to 135; p = 0.38), reduced eversion strength by 6 N (95% CI -112 to 101; p = 0.92) and reduced inversion strength by 8 N (95% CI -110 to 95; p = 0.88) compared to not wearing the splint.
                Wearing night splints does not increase ankle ROM or strength in people with Charcot-Marie-Tooth disease Type 1A.
There is no evidence of significant benefit from any intervention for increasing ankle range of motion in Charcot-Marie-Tooth disease type 1A or Duchenne muscular dystrophy. Further research is required.