Efficacy of corticosteroid in decreasing scoliosis and extending time to loss of ambulation in a single clinic: An effectiveness trial
| Autor: | Susan Sienko, Michael D. Sussman, Charles d’Amato, Cathleen E. Buckon, Coleman Hilton, Camila Bedeschi De Mattos |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
Duchenne muscular dystrophy
scoliosis medicine.drug_class business.industry Deflazacort efficacy Scoliosis medicine.disease corticosteroids Pulmonary function testing 03 medical and health sciences 0302 clinical medicine Anesthesia Pediatrics Perinatology and Child Health Original Clinical Article medicine Corticosteroid Orthopedics and Sports Medicine 030212 general & internal medicine business 030217 neurology & neurosurgery medicine.drug |
| Zdroj: | Journal of Children's Orthopaedics |
| ISSN: | 1863-2548 1863-2521 |
| Popis: | 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 |
| Databáze: | OpenAIRE |
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