Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a randomised, double-blind, placebo-controlled phase II trial.
Oral high dose ascorbic acid treatment for one year in young CMT1A patients: a randomised, double-blind, placebo-controlled phase II trial.
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DOI：
10.1186/1741-7015-7-70
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发表时间：
2009-11-12
期刊：
影响因子：
9.3
通讯作者：
van Schaik IN
中科院分区：
文献类型：
--
作者：
Verhamme C;de Haan RJ;Vermeulen M;Baas F;de Visser M;van Schaik IN
高剂量口服抗坏血酸显著改善Charcot-Marie-Tooth 1A型小鼠模型的髓鞘形成和运动功能。一项II期研究被授权调查高剂量抗坏血酸是否也对Charcot-Marie-Tooth 1A型患者的髓鞘形成有如此实质性的影响，以及这种治疗是否安全。年龄在25岁以下的患者被随机分配接受安慰剂或抗坏血酸（一克，每日两次）在一年的双盲方式。主要结果测量是正中神经运动神经传导速度随时间的变化。次要结局指标包括最小F反应潜伏期、复合肌肉动作电位振幅、肌力、感觉功能、Charcot-Marie-Tooth神经病评分和残疾的变化。6个安慰剂治疗组之间无显著差异。（中位年龄16岁，范围13至24岁）和五个抗坏血酸治疗组正中神经运动神经传导速度改变（19，14 ~ 24）例（抗坏血酸与安慰剂治疗的平均差异为1.3 m/s，置信区间为-0.3至3.0 m/s，P = 0.11）或任何次要结局指标随时间的变化。抗坏血酸组1例患者发生皮疹，导致停用研究药物。口服高剂量抗坏血酸一年没有改善年轻Charcot-Marie-Tooth 1A型患者的正中神经髓鞘形成。治疗相对安全。当前对照试验ISRCTN 56968278，ClinicalTrials.gov NCT 00271635。
High dose oral ascorbic acid substantially improved myelination and locomotor function in a Charcot-Marie-Tooth type 1A mouse model. A phase II study was warranted to investigate whether high dose ascorbic acid also has such a substantial effect on myelination in Charcot-Marie-Tooth type 1A patients and whether this treatment is safe. Patients below age 25 years were randomly assigned to receive placebo or ascorbic acid (one gram twice daily) in a double-blind fashion during one year. The primary outcome measure was the change over time in motor nerve conduction velocity of the median nerve. Secondary outcome measures included changes in minimal F response latencies, compound muscle action potential amplitude, muscle strength, sensory function, Charcot-Marie-Tooth neuropathy score, and disability. There were no significant differences between the six placebo-treated (median age 16 years, range 13 to 24) and the five ascorbic acid-treated (19, 14 to 24) patients in change in motor nerve conduction velocity of the median nerve (mean difference ascorbic acid as opposed to placebo treatment of 1.3 m/s, confidence interval -0.3 to 3.0 m/s, P = 0.11) or in change of any of the secondary outcome measures over time. One patient in the ascorbic acid group developed a skin rash, which led to discontinuation of the study medication. Oral high dose ascorbic acid for one year did not improve myelination of the median nerve in young Charcot-Marie-Tooth type 1A patients. Treatment was relatively safe. Current Controlled Trials ISRCTN56968278, ClinicalTrials.gov NCT00271635.
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