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

被引：82

作者：

Merlini, Luciano ^{[1
]}

Gennari, Monia ^{[2
]}

Malaspina, Elisabetta ^{[2
]}

Cecconi, Ilaria ^{[2
]}

Armaroli, Annarita ^{[3
]}

Gnudi, Saverio ^{[4
]}

Talim, Beril ^{[5
]}

Ferlini, Alessandra ^{[3
]}

Cicognani, Alessandro ^{[2
]}

Franzoni, Emilio ^{[2
]}

机构：

[1] Rizzoli Orthoped Inst, Lab Musculoskeletal Cell Biol, Bologna, Italy

[2] Univ Bologna, Dept Pediat, Bologna, Italy

[3] Univ Ferrara, Dept Expt & Diagnost Med, Med Genet Sect, I-44100 Ferrara, Italy

[4] Rizzoli Orthoped Inst, Dept Med, Bologna, Italy

[5] Hacettepe Univ, Dept Pediat, Ankara, Turkey

来源：

MUSCLE & NERVE | 2012年 / 45卷 / 06期

关键词：

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;

D O I：

10.1002/mus.23272

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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

引用

页码：796 / 802

页数：7

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