SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
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DOI：
10.1002/mus.23904
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发表时间：
2014-02
期刊：
影响因子：
3.4
通讯作者：
Swoboda, Kathryn J.
中科院分区：
文献类型：
--
作者：
Kissel, John T.;Elsheikh, Bakri;King, Wendy M.;Freimer, Miriam;Scott, Charles B.;Kolb, Stephen J.;Reyna, Sandra P.;Crawford, Thomas O.;Simard, Louise R.;Krosschell, Kristin J.;Acsadi, Gyula;Schroth, Mary K.;D'Anjou, Guy;LaSalle, Bernard;Prior, Thomas W.;Sorenson, Susan;Maczulski, Jo Anne;Swoboda, Kathryn J.
一项开放标签试验表明，丙戊酸（VPA）可改善脊髓性肌萎缩症（SMA）成人患者的力量。我们报告了一项在非卧床SMA成人中开展的为期12个月的VPA双盲交叉研究。本研究纳入了33例受试者，年龄为20-55岁。基线评估后，受试者随机接受VPA（10-20 mg/kg/天）或安慰剂。6个月后，患者被转移到另一组。在3、6和12个月时进行评估。主要结局是最大自主等长收缩试验的6个月变化，以及肺、电生理和功能次要结局。30例受试者完成了研究。VPA耐受性良好，依从性良好。在6个月或12个月时，主要或次要结局没有变化。VPA未改善SMA成人的力量或功能。使用的结局是可行和可靠的，可用于SMA成人的未来试验。
An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults. There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.
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