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随机试验:肉毒杆菌毒素预防小儿 1A 型遗传性运动感觉神经病足弓畸形进展

Randomized trial of botulinum toxin to prevent pes cavus progression in pediatric Charcot-Marie-Tooth disease type 1A.

机构信息

Faculty of Health Sciences, University of Sydney, Sydney, New South Wales, Australia.

出版信息

Muscle Nerve. 2010 Aug;42(2):262-7. doi: 10.1002/mus.21685.

DOI:10.1002/mus.21685
PMID:20544937
Abstract

Pes cavus in Charcot-Marie-Tooth disease type 1A (CMT1A) is thought to be due to muscle imbalance of the lower leg. Botulinum toxin type A (BoNT-A) can modify foot deformity in other conditions of muscle imbalance. We tested the safety and effectiveness of BoNT-A on pes cavus progression in pediatric CMT1A. A 24-month, randomized, single-blind trial of BoNT-A was undertaken in 10 affected children (20 legs), aged 3-14 years. The treated leg received intramuscular BoNT-A injections at 6-month intervals in the tibialis posterior and peroneus longus. The control leg received no injections. Primary outcome was radiographic alignment at 24 months. Secondary outcomes were foot posture, ankle flexibility, and strength, assessed every 6 months. Radiographically, BoNT-A produced a small non-significant reduction in cavus progression. There was no effect of BoNT-A on secondary outcomes. There were no serious adverse events. At 24 months, the intramuscular BoNT-A injections proved safe and well-tolerated but did not affect the progression of pes cavus in CMT1A.

摘要

Charcot-Marie-Tooth 病 1A 型(CMT1A)中的高弓足被认为是由于小腿肌肉失衡引起的。肉毒杆菌毒素 A 型(BoNT-A)可以改变其他肌肉失衡情况下的足畸形。我们测试了 BoNT-A 对小儿 CMT1A 中高弓足进展的安全性和有效性。对 10 名受累儿童(20 条腿)进行了为期 24 个月的随机、单盲、BoNT-A 试验,年龄为 3-14 岁。治疗腿每 6 个月接受胫后肌和腓骨长肌的肌内 BoNT-A 注射,对照腿不接受注射。主要结果是 24 个月时的影像学对线。次要结果是足部姿势、踝关节灵活性和力量,每 6 个月评估一次。影像学上,BoNT-A 使高弓足的进展略有但无统计学意义的减少。BoNT-A 对次要结果没有影响。没有严重不良事件。24 个月时,肌内 BoNT-A 注射证明是安全且耐受良好的,但不能影响 CMT1A 中高弓足的进展。

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