Nerve growth factor for the treatment of spinocerebellar ataxia type 3: an open-label study.
Nerve growth factor for the treatment of spinocerebellar ataxia type 3: an open-label study.
复制标题
神经生长因子治疗 3 型脊髓小脑共济失调：一项开放标签研究
DOI：
10.4103/0366-6999.150087
复制
发表时间：
2015-02-05
影响因子：
6.1
通讯作者：
Xu YM
中科院分区：
文献类型：
--
作者：
Tan S;Wang RH;Niu HX;Shi CH;Mao CY;Zhang R;Song B;Sun SL;Liu XJ;Hou HM;Liu YT;Gao Y;Fang H;Kong XD;Xu YM
背景资料：脊髓小脑性共济失调3型（SCA 3）是世界范围内最常见的SCA亚型，并且具有缓慢进展和持续的病程。目前没有可治愈的治疗方法。越来越多的证据表明，神经生长因子（NGF）可能对神经退行性疾病有治疗作用，也可能对SCA 3有治疗作用。本研究的目的是测试神经生长因子在SCA 3患者中的疗效。方法：我们在经遗传学证实的成人（>18岁）SCA 3患者中进行了一项开放标签前瞻性研究。通过肌内注射施用NGF（18 g，每日一次），连续28天。采用中文版共济失调评定量表（SARA）对所有患者进行基线、治疗后2周和4周的评定。结果如下：21例SCA 3患者（10例男性和11例女性，平均年龄39.14 ± 7.81岁，平均病程4.14 ± 1.90年，平均CAG重复次数77.57 ± 2.27）入组。治疗28天后，平均总SARA评分从基线的8.48 ± 2.40降至6.30 ± 1.87（P < 0.001）。亚部分SARA评分也显示站立（P = 0.003）、言语（P = 0.023）、手指追逐（P = 0.015）、快速交替手部运动（P = 0.009）和脚跟-胫骨滑动（P = 0.001）显著改善。结论：我们的初步数据表明，神经生长因子可能是有效的治疗SCA 3患者。
Background: Spinocerebellar ataxia type 3 (SCA3) is the most common subtype of SCA worldwide, and runs a slowly progressive and unremitting disease course. There is currently no curable treatment available. Growing evidence has suggested that nerve growth factor (NGF) may have therapeutic effects in neurodegenerative diseases, and possibly also in SCA3. The objective of this study was to test the efficacy of NGF in SCA3 patients. Methods: We performed an open-label prospective study in genetically confirmed adult (>18 years old) SCA3 patients. NGF was administered by intramuscular injection (18 &mgr;g once daily) for 28 days consecutively. All the patients were evaluated at baseline and 2 and 4 weeks after treatment using the Chinese version of the scale for assessment and rating of ataxia (SARA). Results: Twenty-one SCA3 patients (10 men and 11 women, mean age 39.14 ± 7.81 years, mean disease duration 4.14 ± 1.90 years, mean CAG repeats number 77.57 ± 2.27) were enrolled. After 28 days of NGF treatment, the mean total SARA score decreased significantly from a baseline of 8.48 ± 2.40 to 6.30 ± 1.87 (P < 0.001). Subsections SARA scores also showed significant improvements in stance (P = 0.003), speech (P = 0.023), finger chase (P = 0.015), fast alternating hand movements (P = 0.009), and heel-shin slide (P = 0.001). Conclusions: Our preliminary data suggest that NGF may be effective in treating patients with SCA3.