CMT 1A: Randomized trial of botulinum toxin to prevent pes cavus progression in

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Muscle Nerve. 2010 May 18

Randomized trial of botulinum toxin to prevent pes cavus progression in

pediatric charcot-marie-tooth disease type 1A.

Burns J, Scheinberg A, MM, Rose KJ, Ouvrier RA.

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

Australia.

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.