Mandel Jonas, Bertrand Viviane, Lehert Philippe, Attarian Shahram, Magy Laurent, Micallef Joëlle, Chumakov Ilya, Scart-Grès Catherine, Guedj Mickael, Cohen Daniel
Pharnext SAS, Issy-les-Moulineaux, France.
Faculty of Medicine, University of Melbourne, Melbourne, Australia.
Orphanet J Rare Dis. 2015 Jun 13;10:74. doi: 10.1186/s13023-015-0293-y.
CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo. Patients undergoing PXT3003 treatment showed an improvement in CMTNS and ONLS, statistically significant versus Placebo and potentially precursory of a meaningful change in the disease course.
遗传性运动感觉神经病1型（CMT1A）是最常见的遗传性周围神经病。目前尚无获批的治疗方法。我们进行了一项荟萃分析，纳入了四项随机、双盲、安慰剂对照临床试验，以评估在接受安慰剂、抗坏血酸（AA）或PXT3003（三种重新利用药物的组合）治疗一年后的疾病进展情况。我们观察到接受安慰剂治疗的患者病情有轻微恶化，远低于所报道的自然疾病进展。接受AA治疗的患者在一年后病情稳定，但与安慰剂组无显著差异。接受PXT3003治疗的患者在遗传性运动感觉神经病神经病变评分量表（CMTNS）和神经病变损害量表（ONLS）方面有所改善，与安慰剂相比具有统计学意义，并且可能预示着疾病进程将发生有意义的变化。
