INTRODUCTION Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD). METHODS In this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety. RESULTS Change in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD]: 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects. DISCUSSION In boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth.

中文翻译：

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6个月的重组人胰岛素样生长因子-1疗法可改善患有杜兴氏肌营养不良症的男孩的生长，但不能改善其运动功能。

引言重组人胰岛素样生长因子1（rhIGF-1）是一种生长因子，对肌肉具有合成代谢作用。我们调查了rhIGF-1治疗是否：1）改善或保留了肌肉功能；2）改善患有Duchenne肌肉营养不良（DMD）的男孩的生长。方法在这项研究中，我们比较了一项为期6个月的前瞻性，随机，对照的皮下注射rhIGF-D试验，该研究对青春期前，非卧床，糖皮质激素治疗的DMD男孩（n = 17）与对照组（仅糖皮质激素治疗，n = 21）进行了比较。 1疗法。主要结果是步行6分钟即可到达（6MWD）。次要结果包括身高速度（HV），身高标准偏差评分（ΔHtSDS）变化，运动功能，心肺功能，身体成分，胰岛素敏感性，生活质量和安全性。结果各组之间6MWD的变化相似（rhIGF-1与对照组[平均值±SD]：3.4±32.4 vs -5.1±50.2米，P = .53）。接受治疗的受试者的生长快于对照组（HV：6.5±1.7 vs / 3.3±1.3 cm /年，P <.0001; 6个月ΔHtSDS：0.25，P <.0001）。接受治疗的受试者的瘦体重和胰岛素敏感性增加。讨论在患有DMD的男孩中，rhIGF-1治疗6个月没有改变运动功能，但改善了线性生长。