Glucocorticoid Treatment for the Prevention of Scoliosis in Children with Duchenne Muscular Dystrophy: Long-Term Follow-up David E. Lebel, MD, PhD, John A. Corston, Laura C. McAdam, MD, FRCPC, W. Douglas Biggar, MD, FRCPC, and Benjamin A. Alman, MD, FRCSC Investigation performed at the Holland Bloorview Kids Rehabilitation Hospital, The Hospital for Sick Children, and the University of Toronto, Toronto, Ontario, Canada Background: 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. We examined if glucocorticoid treatment has a long-term effect on the prevalence of scoliosis. Methods: Fifty-four boys who had been diagnosed with Duchenne muscular dystrophy while they were still walking were enrolled in a non-randomized comparative study of the glucocorticoid deflazacort. The families of thirty boys elected for them to use glucocorticoid treatment and the families of twenty-four boys elected for them not to have this treatment. The boys were matched for important baseline characteristics including age and pulmonary function. 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. Because surgery was recommended for spinal curves measuring >20° on sitting posteroanterior radiographs, a curve of this magnitude was used as the definition for a patient developing scoliosis. Results: 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%) in the glucocorticoid treatment group and twenty-two (92%) 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 27 ) 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. Conclusion: The long-term use of the glucocorticoid results in a substantial decreased need for spinal surgery to treat scoliosis. Level of Evidence: Therapeutic Level II. See Instructions for Authors for a complete description of levels of evidence. D uchenne muscular dystrophy is a recessive, X-linked, inherited disorder. It is characterized by a progressive decline in muscle strength and is generally diagnosed in the first five years of life. The progressive muscle weakness results in an inability to walk and the secondary development of musculoskeletal deformity. The majority (90%) of untreated boys with this disorder develop progressive scoliosis, secondary to muscle weakness after becoming full-time wheelchair users 1 . Disclosure: None of the authors received payments or services, either directly or indirectly (i.e., via his or her institution), from a third party in support of any aspect of this work. None of the authors, or their institu- tion(s), have had any financial relationship, in the thirty-six months prior to submission of this work, with any entity in the biomedical arena that could be perceived to influence or have the potential to influence what is written in this work. Also, no author has had any other relationships, or has engaged in any other activities, that could be perceived to influence or have the potential to influence what is written in this work. The com- plete Disclosures of Potential Conflicts of Interest submitted by authors are always provided with the online version of the article. A commentary by Anne M. Connolly, MD, Han Jo Kim, MD, and Keith H. Bridwell, MD, is linked to the online version of this article at jbjs.org. 1057 COPYRIGHT Ó 2013 BY THE J OURNAL OF BONE AND J OINT SURGERY,I NCORPORATED J Bone Joint Surg Am. 2013;95:1057-61 d http://dx.doi.org/10.2106/JBJS.L.01577