Use of assistive technology devices in children and adolescents with Charcot-Marie-Tooth disease: feasibility study and proof-of-concept

• Grant number: 22/10786-0
• Support Opportunities: Scholarships in Brazil - Doctorate
• Start date: April 01, 2024
• End date: February 28, 2027
• Field of knowledge: Health Sciences - Physiotherapy and Occupational Therapy
• Principal Investigator: Ana Cláudia Mattiello-Sverzut
• Grantee: Juliana Cardoso
• Host Institution: Faculdade de Medicina de Ribeirão Preto (FMRP). Universidade de São Paulo (USP). Ribeirão Preto , SP, Brazil
• Associated scholarship(s): 24/21026-2 - PRESCRIPTION OF LOWER LIMB ORTHOSES IN CHILDREN AND ADOLESCENTS WITH CHARCOT-MARIE-TOOTH DISEASE AND THE PATHWAYS FOR THE PROVISION OF THESE DEVICES IN AN AUSTRALIAN PUBLIC HOSPITAL, BE.EP.DR

Abstract

Despite the data available in the literature on Charcot-Marie-Tooth disease, there is still a need for more information to guide the prescription of assistive technology devices, their manufacture and their effect, especially after prolonged use on physical function and gait biomechanics. Currently, well-designed randomised clinical trials (RCTs) provide the most reliable evidence on the effect of an intervention. However, before conducting a definitive RCT with a large sample size, feasibility and proof-of-concept studies are recommended. Therefore, the primary objective of this study is to investigate the feasibility of inserting personalised assistive technology devices in children and adolescents with CMT who have distal lower limb impairment and secondarily, by means of proof of concept, to assess the potential effectiveness of these devices on the parameters obtained in functional tests and biomechanical gait analysis. This research project will be divided into two studies: (1) a feasibility study and (2) a proof-of-concept study. It will include 30 children and adolescents with CMT, who will use a personalised assistive technology device for a period of six months. The feasibility of the intervention will be checked using measures of contact rate, contact success, recruitment success, inclusion success, dropout rate, satisfaction with using the device, adherence to treatment and safety of using the device. For the proof-of-concept study, patients will undergo four evaluations, with clinical impairment tests (Charcot-Marie-Tooth Paediatric Scale), dynamic plantar pressure distribution analysis (Pedar®-X equipment) and gait biomechanics analysis. A descriptive analysis will be carried out, according to the distribution of the data. Frequency measures, mean, median, minimum and maximum values observed will be presented in tables and/or figures.