Duchenne muscular dystrophy, a progressive muscle disorder that occurs in males, causes a gradual decline in muscle strength. This progressive decline is associated with the development of scoliosis. Previous studies have shown that the use of glucocorticoids slows the progression of scoliosis, but it is unknown if the spine remains straight in the long term. The present follow-up study of fifty-four boys who had been diagnosed with Duchenne muscular dystrophy examined if glucocorticoid treatment has a long-term effect on the prevalence of scoliosis. While still walking, they were enrolled in a non-randomized comparative study of the glucocorticoid deflazacort. Thirty boys (56%) received glucocorticoid treatment, a decision made by their families and physicians. In this study, scoliosis is defined as a Cobb angle measurement of >20° on posteroanterior radiographs. Every four to six months, they were examined for the development of scoliosis, and the duration of follow-up for surviving patients was fifteen years. Five boys (21%) in the non-treatment group and one boy (3%) in the glucocorticoid treatment group died. At the most recent follow-up, of the boys who survived, six (20%) of thirty boys in the glucocorticoid treatment group and twenty-two (92%) of twenty-four boys in the non-treatment group developed scoliosis and underwent spinal surgery. After fifteen years of follow-up, the survivorship analysis (avoiding surgery) was 78% (95% confidence interval, 57% to 89%) in the treatment group and 8.3% (95% confidence interval, 0.8% to 28%) in the non-treatment group. Significance (p = 5.8 × 10-7) was calculated with log-rank and chi-square tests. None of the patients in the glucocorticoid group developed scoliosis after ten years of deflazacort treatment. The results from this study clearly demonstrate that daily deflazacort prolongs life, delays deterioration in pulmonary function, and decreases the need for scoliosis surgery. This excellent study offers long-term data that physicians can use to encourage more families and their physicians to try corticosteroids in patients with Duchenne muscular dystrophy.
