Child Neurology and Psychiatry Unit, Azienda Ospedaliera Universitaria Senese/University of Siena, Policlinico Le Scotte, Siena, Italy.
Mov Disord. 2012 Sep 1;27(10):1312-6. doi: 10.1002/mds.25126. Epub 2012 Aug 23.
No controlled studies exist regarding the pharmaceutical reduction of ataxia symptoms in ataxia telangiectasia (A-T). In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS). In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31%, respectively). In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established. (Current Controlled Trials, number ISRCTN08774933.)
尚无关于降低共济失调毛细血管扩张症（A-T）患者共济失调症状的药物治疗的对照研究。在一项多中心、双盲、随机、安慰剂对照交叉试验中，使用国际共济失调协作评定量表（ICARS）评估，比较了口服倍他米松（BETA）和安慰剂对共济失调症状的改善作用。在这项针对 13 名 A-T 患儿的研究中，在意向治疗人群中，倍他米松使 ICARS 总分中位数降低了 13 分，在符合方案人群中降低了 16 分（即，共济失调症状的中位数百分比分别降低了 28%和 31%）。总之，口服倍他米松可能是一种有前途的治疗方法，可以缓解 A-T 患者的共济失调症状；但是，必须确定其长期有效性和安全性。（正在进行的对照试验，注册号 ISRCTN08774933。）
