Natural history of CMT1A including QoL: A 2-year prospective study

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Volume 18, Issue 3, Pages 199-203 (March 2008)

Neuromuscular Disorders

Natural history of CMT1A including QoL: A 2-year prospective study

L. Paduaab, D. Pareysonc, I. Aprileb, T. Cavallarod, A. Quattronee,

N. Rizzutof, G. Vitag, P. Tonaliab, A. Schenoneh

Abstract

The Italian CMT study group performed a multicentre,

multidimensional, longitudinal 2-year follow-up study using validated

measurements of neurological impairment, disability and quality of

life. The aim of the study was to evaluate the natural history of

clinical features, disability and QoL in patients with CMT1A.

On clinical examination, CMT1A patients showed a significant

reduction in muscle strength and sensory function during the 2-year

follow-up period. However, there was no worsening of QoL or

disability, nor was depression observed.

The discrepancy between the evolution of clinical features and the

evolution of QoL and disability may be due to the development of

compensatory strategies that help patients cope with the slow

progression of the disease. Our observations provide information

which may be useful when designing clinical trials in CMT.

a Institute of Neurology, Università Cattolica del Sacro Cuore, Largo

Francesco Vito 1, Rome, Italy

b Fondazione Don Carlo Gnocchi, Rome, Italy

c Fondazione IRCCS, Istituto Neurologico " Carlo Besta " , Milan, Italy

d Department of Neurological and Visual Sciences, Section of Clinical

Neurology, University of Verona, Italy

e Azienda O.U. Policlinico di Catanzaro, University of Catanzaro,

Italy

f Policlinico GB Rossi, University of Verona, Italy

g Department of Neuroscience, Psichiatry and Anaesthesiology,

Messina, Italy

h Department of Neurological and Vision Sciences, Genova, Italy