Treatment for Charcot-Marie-Tooth disease

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Cochrane Database Syst Rev. 2008 Jan 23;(1):CD006052.

Treatment for Charcot-Marie-Tooth disease.Young P, De Jonghe P,

Stögbauer F, Butterfass-Bahloul T.

http://www.ncbi.nlm.nih.gov/pubmed/18254090?

ordinalpos=1 & itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pu

bmed_RVDocSum

BACKGROUND: Charcot-Marie-Tooth disease (CMT) comprises a large

variety of different forms of motor and sensory neuropathies. The

most frequent are demyelinating forms (CMT1) and axonal forms (CMT2).

The molecular basis of several CMT forms has been clarified during

the last 15 years. Since muscle wasting and sensory disturbance are

the main features of these syndromes, treatments aim to improve motor

impairment and sensory disturbances. Specific treatment trials are

rare.

OBJECTIVES: The objective was to review systematically all randomised

and quasi-randomised studies of any treatment for CMT.

SEARCH STRATEGY: We searched the Cochrane Neuromuscular Disease Group

Trials Register, MEDLINE (January 1966 to August 2007), EMBASE

(January 1980 to August 2007), LILACS (January 1982 to August 2007)

for randomised controlled trials of treatment for CMT. SELECTION

CRITERIA: We included randomised and quasi-randomised trials of any

treatment for people with CMT. Where a study aimed to evaluate the

treatment of general neuromuscular symptoms of people with peripheral

neuropathy including CMT, we included the study if we were able to

identify the effect of treatment in the CMT group. Observational

studies and case reports on the treatment of people with CMT were not

included.

DATA COLLECTION AND ANALYSIS: Two review authors (PY and TBB)

extracted the data, assessed study quality and performed data

extraction independently. MAIN RESULTS: Only one trial with only

eight participants met all the inclusion criteria and provided the

primary outcome measure for this review. In this trial, four

participants treated with neurotrophin-3 had more improvement after

six months on the Neuropathy Impairment Score, mean difference -9.50

(95% CI -13.77 to -5.23), than those four treated with placebo. Small

trials of exercise training, creatine monohydrate, orthoses and

purified bovine brain ganglioside injections (Cronassial) showed no

significant benefit in people with genetically undefined CMT1 or

CMT2.

AUTHORS' CONCLUSIONS: Small trials of exercise, creatine, purified

brain gangliosides, and orthoses have been performed. None showed

significant benefit. A very small trial of Neurotrophin-3 showed

possible minor benefit which needs to be replicated in a larger

trial. None of the two trials were large enough to detect moderate

benefit or harm. Larger RCTs are needed for any form of

pharmacological intervention as well as as for any form of physical

intervention. Outcome measures should include a validated composite

scale such as the Charcot-Marie-Tooth neuropathy scale.