Effect of recombinant human insulin-like growth factor-I on progression of ALS
Effect of recombinant human insulin-like growth factor-I on progression of ALS
复制标题
重组人胰岛素样生长因子-I对ALS进展的影响
作者：
E. Lai;K. Felice;B. Festoff;M. Gawel;D. Gelinas;R. Kratz;M. Murphy;H. Natter;F. Norris;S. Rudnicki
本研究的目的是探讨重组人胰岛素样生长因子- i （rhIGF-I）治疗散发性肌萎缩侧索硬化症的安全性和有效性。一项双盲、安慰剂对照、随机研究在北美的八个中心进行，共有266名患者。安慰剂或rhigf - 1 （0.05 mg/kg/天或0.10 mg/kg/天）治疗9个月。主要结局指标是疾病症状进展，通过Appel ALS评分量表总分的变化率（每位患者斜率）进行评估。疾病影响概况（SIP）是患者感知的与健康相关的生活质量评估，是次要结果变量。接受高剂量（0.10 mg/kg/天）rhIGF-I治疗的患者的功能损害进展比接受安慰剂治疗的患者慢26% （p = 0.01）。高剂量治疗组终止研究的可能性较小，因为方案中定义了疾病症状进展的标志物，并且根据SIP评估，该组成员表现出较慢的生活质量下降。接受0.05 mg/kg/天rhIGF-I治疗的患者表现出与高剂量治疗相关的趋势相似，表明存在剂量依赖性反应。临床显著不良经历的发生率在三个治疗组之间具有可比性。重组人胰岛素样生长因子- 1减缓了ALS患者功能障碍的进展和健康相关生活质量的下降，没有医学上重要的不良反应。
The objective of this study was to investigate the safety and efficacy of recombinant human insulinlike growth factor-I (rhIGF-I) in the treatment of sporadic ALS. A double-blind, placebo-controlled, randomized study of 266 patients was conducted at eight centers in North America. Placebo or rhIGF-I(0.05 mg/kg/day or 0.10 mg/kg/day) was administered for 9 months. The primary outcome measure was disease symptom progression, assessed by the rate of change (per patient slope) in the Appel ALS rating scale total score. The Sickness Impact Profile (SIP), a patient-perceived, health-related quality of life assessment, was a secondary outcome variable. Progression of functional impairment in patients receiving high-dose (0.10 mg/kg/day) rhIGF-I was 26% slower than in patients receiving placebo (p = 0.01). The high-dose treatment group was less likely to terminate the study due to protocol-defined markers of disease symptom progression, and members in this group exhibited a slower decline in quality of life, as assessed by the SIP. Patients receiving 0.05 mg/kg/day of rhIGF-I exhibited trends similar to those associated with high-dose treatment, suggesting a dose-dependent response. The incidence of clinically significant adverse experiences was comparable among the three treatment groups. Recombinant human insulin-like growth factor-I slowed the progression of functional impairment and the decline in health-related quality of life in patients with ALS with no medically important adverse effects.
Manduca sexta 蛾变态过程中已识别神经元的树突重组：与周围相互作用的影响。
DOI：
10.1002/neu.480240102
发表时间：
1993
期刊：
Journal of neurobiology
影响因子：
--
作者：
Kent,KS;Levine,RB
通讯作者：
Levine,RB