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.
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 DMDpatients. 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.
Authors: Rebecca J Willcocks; William T Triplett; Donovan J Lott; Sean C Forbes; Abhinandan Batra; H Lee Sweeney; Jerry R Mendell; Krista Vandenborne; Glenn A Walter Journal: Muscle Nerve Date: 2018-01-24 Impact factor: 3.217
Authors: David J Birnkrant; Katharine Bushby; Carla M Bann; Susan D Apkon; Angela Blackwell; David Brumbaugh; Laura E Case; Paula R Clemens; Stasia Hadjiyannakis; Shree Pandya; Natalie Street; Jean Tomezsko; Kathryn R Wagner; Leanne M Ward; David R Weber Journal: Lancet Neurol Date: 2018-02-03 Impact factor: 44.182
Authors: Jennifer M Kwon; Hoda Z Abdel-Hamid; Samiah A Al-Zaidy; Jerry R Mendell; Annie Kennedy; Kathi Kinnett; Valerie A Cwik; Natalie Street; Julie Bolen; John W Day; Anne M Connolly Journal: Muscle Nerve Date: 2016-06-13 Impact factor: 3.217
Authors: Qing Ke; Zheng-Yan Zhao; Robert Griggs; Veronica Wiley; Anne Connolly; Jennifer Kwon; Ming Qi; Daniel Sheehan; Emma Ciafaloni; R Rodney Howell; Petra Furu; Peter Sazani; Arvind Narayana; Michele Gatheridge Journal: World J Pediatr Date: 2017-05-17 Impact factor: 2.764