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. 2020 Oct 1;14(5):421-432.
doi: 10.1302/1863-2548.14.200156.

Efficacy of corticosteroid in decreasing scoliosis and extending time to loss of ambulation in a single clinic: an effectiveness trial

Michael D Sussman  1 , Susan E Sienko  2 , Cathleen E Buckon  2 , Coleman Hilton  2 , Camila Bedeschi De Mattos  3 , Charles d'Amato  1
Affiliations

Efficacy of corticosteroid in decreasing scoliosis and extending time to loss of ambulation in a single clinic: an effectiveness trial

Michael D Sussman et al. J Child Orthop. .

Abstract

Purpose: Pharmacologic doses of corticosteroid (CS) have been shown to ameliorate the progression of Duchenne muscular dystrophy (DMD) preserving strength, pulmonary function and ambulation as well as reducing the incidence of scoliosis. However, there are serious side effects of CS, which may impact dose tolerance. The purpose of this study was to compare the magnitude of positive CS effects on patients in our clinic to those reported in the literature.

Methods: We retrospectively reviewed medical records and radiographs of 142 DMD patients who were seen between 1st January 1991 and 31st December 2017.

Results: In total, 101 boys met study inclusion criteria. Of these 32 were steroid naïve, 37 took the recommended dose (standard of care, SOC) of Prednisone or Deflazacort, and 32 took a lower dose (LD). Following initiation of CS, both treatment groups showed an increase in weight velocity and decrease in linear growth velocity. Although there was a trend to later loss of ambulation (LOA) in the SOC group relative to the naïve group by one year, this was not significant, however, a small subgroup of boys on Deflazacort showed a 3.4 year later LOA than the naïve group. The incidence of scoliosis was reduced from 69% in the naïve, to 41% in the LD and 47% in the SOC group.

Conclusions: Although there was a reduction in the incidence of scoliosis, it was not as robust as seen elsewhere. Many published studies have inadequate data on scoliosis probably due to the lack of inclusion of orthopaedists in the study group.

Level of evidence: IV.

Keywords: Deflazacort; Duchenne muscular dystrophy; corticosteroids; efficacy; scoliosis.

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Figures

Fig. 1
Fig. 1
Historical age of steroid prescription.
Fig. 2
Fig. 2
Height velocity.
Fig. 3
Fig. 3
Weight velocity.
Fig. 4
Fig. 4
Length of steroid use and age at loss of ambulation.
Fig. 5
Fig. 5
This patient walked until the age of 13.5 years. (a) Growth chart showing almost complete cessation of linear growth following administration of Deflazacort at 30 mg/day. (b) Radiograph of same boy at age 15 showing a 37.2° scoliosis, which was first ascertained six months earlier when it was 28°. His FVC at this time was 60% of predicted.
Fig. 6
Fig. 6
Recommendations for studies of scoliosis in patients with Duchenne muscular dystrophy.
See this image and copyright information in PMC

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