The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.

CMT is an inherited nerve condition that can cause muscle weakness, loss of sensation, and foot deformities. These changes often make walking difficult and can reduce independence and quality of life. Treatment options commonly include physical therapy alone or surgery to correct foot alignment followed by rehabilitation. However, it is not clear whether one approach leads to better long-term walking outcomes.

The main question this study aims to answer is whether individuals who undergo functional foot surgery followed by rehabilitation experience different changes in walking ability over time compared with those who receive structured physical therapy alone.

Researchers will compare walking performance between these two treatment groups over a period of up to two years. Walking ability will be evaluated using standardized walking tests and patient questionnaires.

Participants included in this study are individuals with CMT-related foot deformities that affect walking and who received either surgery followed by rehabilitation or physical therapy alone. Researchers will analyze changes in walking ability over time and determine how many participants achieve meaningful improvement.

The findings from this study may help clinicians and individuals with CMT better understand how different treatment strategies influence walking function over time.

Charcot-Marie-Tooth disease (CMT) is a genetically heterogeneous inherited peripheral neuropathy characterized by progressive distal muscle weakness, sensory impairment, and neuromuscular foot deformities. These impairments frequently alter gait mechanics and reduce walking capacity. In the absence of disease-modifying therapies, clinical management focuses on optimizing functional mobility and mitigating secondary musculoskeletal consequences.

Both conservative physical therapy and functional surgical correction followed by rehabilitation are widely used to address gait-limiting deformities in CMT. Physical therapy primarily targets strength, balance, and motor control, whereas surgical intervention aims to improve structural alignment and rebalance muscle-tendon forces. Despite routine implementation of both approaches, comparative evidence regarding their longitudinal effects on objective walking performance remains limited.

This study uses a bidirectional observational cohort design integrating retrospectively ascertained treatment exposure with prospectively collected longitudinal outcomes. Individuals with CMT-related foot deformities affecting walking who were treated between January 2017 and January 2024 were identified from an institutional clinical database. Participants were classified according to treatment strategy: (1) functional surgical correction followed by standardized postoperative rehabilitation, or (2) structured physical therapy alone. Treatment allocation was determined by routine clinical decision-making rather than randomization.

To address confounding inherent in nonrandomized comparisons, propensity score matching was applied to balance baseline demographic and disease-related characteristics between treatment groups. Covariates included factors such as age at presentation, genetic subtype when available, and baseline disease severity. Balance diagnostics were assessed to confirm adequate group comparability following matching.

The primary focus of the study is longitudinal walking performance. Standardized performance-based assessments of habitual walking speed and functional walking capacity were administered at baseline and during follow-up. A multidimensional framework was used to capture impairment-level measures, task-specific walking performance, and patient-reported health status, enabling evaluation of both functional change and broader clinical impact.

Follow-up assessments were conducted at prespecified intervals up to 2 years after treatment initiation. Baseline data were extracted from electronic medical records, and follow-up data were collected prospectively through structured outpatient assessments and standardized remote evaluations when necessary. Consistent assessment procedures were implemented across time points to enhance reproducibility.

Longitudinal analyses were performed using repeated-measures modeling to evaluate within- and between-group changes over time, with particular emphasis on group-by-time interactions. Multivariable regression analyses were conducted to explore associations between treatment strategy and changes in walking performance while adjusting for relevant clinical covariates. Clinically meaningful improvement was evaluated using established minimal clinically important difference thresholds for walking-related outcomes. Missing data were handled using multiple imputation techniques.

Quality assurance procedures included standardized evaluator training, predefined analytic plans, and structured data management processes to minimize bias and enhance methodological rigor.

Through this longitudinal and performance-centered design, the study aims to clarify the functional consequences of surgical and conservative management strategies for gait-limiting CMT-related foot deformities and to better characterize the trajectory and clinical relevance of walking outcomes over time.

Inclusion Criteria:

• Participants aged 12 years or older at the time of enrollment
• Genetically or clinically confirmed CMT, based on established diagnostic criteria
• Presence of foot deformity and/or gait impairment attributable to CMT, as determined by a treating clinician
• Ability to ambulate at least 10 meters, with or without assistive devices
• Medically eligible for either functional surgical intervention or structured physical therapy, as determined by the treating team
• Willingness and ability to participate in longitudinal follow-up assessments
• Provision of written informed consent

Exclusion Criteria:

• Previous major foot or ankle surgery that substantially altered lower-limb biomechanics
• Presence of non-CMT-related neurological disorders affecting gait (eg, stroke, Parkinson disease, multiple sclerosis)
• Severe musculoskeletal conditions unrelated to CMT that limit walking ability (eg, advanced hip or knee osteoarthritis)
• Active lower-limb infection, ulceration, or acute injury at the time of enrollment
• Severe cognitive impairment or psychiatric condition precluding reliable participation
• Medical contraindications to surgery or exercise-based rehabilitation, when relevant
• Inability to complete baseline functional assessments or anticipated inability to complete follow-up

The study population comprises adults with Charcot-Marie-Tooth disease (CMT) evaluated and treated at a tertiary referral center specializing in neuromuscular and foot and ankle disorders. Participants were identified from consecutive clinical encounters beginning in February 2017, incorporating both retrospectively and prospectively collected data. Eligible individuals had a genetically or clinically confirmed diagnosis of CMT and presented with foot deformity and gait impairment attributable to peripheral neuropathy. All participants were ambulatory, with or without assistive devices, at the time of enrollment. Management strategies included functional surgical intervention followed by rehabilitation or structured physical therapy alone, reflecting routine clinical practice. Longitudinal outcome data were obtained through standardized assessments conducted at baseline and predefined follow-up time points throughout the study period.