Early corticosteroid treatment in 4 duchenne muscular dystrophy patients: 14-year follow-up

Merlini L., Gennari M., Malaspina E., Cecconi I., Armaroli A., Gnudi S., ...More

MUSCLE & NERVE, vol.45, no.6, pp.796-802, 2012 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 45 Issue: 6
  • Publication Date: 2012
  • Doi Number: 10.1002/mus.23272
  • Journal Name: MUSCLE & NERVE
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.796-802
  • Keywords: alternate-day regimen, corticosteroid therapy, Duchenne muscular dystrophy, outcome, prospective study, PREDNISONE THERAPY, DEFLAZACORT TREATMENT, RESPIRATORY-FUNCTION, CONTROLLED-TRIAL, DOUBLE-BLIND, BOYS, DYSFUNCTION, STRENGTH, CHILDREN, QUALITY
  • Hacettepe University Affiliated: Yes

Abstract

Introduction: Corticosteroid treatment is the standard of care in Duchenne muscular dystrophy (DMD), but the optimal age to initiate treatment and dosage pattern remain a matter of discussion. Methods: We performed a long-term study of alternate-day corticosteroids in five 2- to 4-year-old DMD patients. The primary outcome measure was prolongation of the ability to walk. Results: One patient lost ambulation at age 10. Four patients, aged 16 to 18 were fully ambulant, and 3 of them could still climb stairs. Respiratory function was moderately reduced in 2. Left ventricular ejection fraction was > 45%. Short stature and delayed puberty were the most relevant side effects. Although the negative impact of corticosteroid treatment on growth rate remained their major concern, parents and patients stated that they preferred corticosteroid therapy. Conclusions: Long-term corticosteroid treatment is effective in prolonging function but not in recovering lost function, and its early use seems appropriate. Muscle Nerve 45: 796802, 2012