Background: Scoliosis due to progressive muscle weakness occurs in
almost all males with Duchenne muscular dystrophy, and it progresses
relentlessly. Previous studies have shown that corticosteroid treatment slows
the decline in muscle strength and stabilizes muscle strength in patients with
this disease. We hypothesized that steroids may also attenuate the development
of scoliosis. The purpose of this study was to compare the prevalence of
scoliosis in male patients with Duchenne muscular dystrophy who received
steroids with a control group of such patients who did not.
Methods: A group of seven to ten-year-old boys with Duchenne
muscular dystrophy who were able to walk were enrolled in a nonrandomized
comparative study to determine the effect of deflazacort (a derivative of
prednisone) on muscle strength and pulmonary function. Thirty patients were
treated with deflazacort (treatment group), and twenty-four were not (control
group). The patients were matched for age and pulmonary function at baseline.
To assess the development of scoliosis, the patients in each group were
followed for at least five years. Survival curves were plotted to determine
the chance of scoliosis of =20° developing. The difference between the
groups with respect to the chance of scoliosis developing was determined with
Results: A curve of =20° developed during the follow-up
period in sixteen (67%) of the twenty-four patients in the control group but
in only five (17%) of the thirty patients in the treatment group. Fifteen of
the twenty-four patients in the control group underwent spine surgery, at a
mean age of thirteen years, whereas only five of the thirty patients in the
treatment group underwent spine surgery, at a mean age of fifteen years.
Kaplan-Meier analysis demonstrated a significant difference between the two
groups with regard to development of scoliosis of =20° (p < 0.001).
Cataracts developed in ten patients in the treatment group, and stress
fractures developed in three patients in the treatment group. Patients in the
treatment group weighed a mean of 3.7 kg more than did those in the control
Conclusions: Steroid treatment slows the progression of scoliosis in
males with Duchenne muscular dystrophy; however, longer-term evaluation will
be necessary to determine if the treatment prevents the development of
scoliosis or just delays its onset. At the very least, steroid treatment
delays the need for spinal surgery.
Level of Evidence: Therapeutic study, Level II-1
(prospective cohort study). See Instructions to Authors for a complete
description of levels of evidence.