Abstract
Objective: To develop predictive scales that correlate Charcot-Marie-Tooth Neuropathy Score version 2 (CMTNSv2) scores with specific ambulatory aid use in pediatric patients with Charcot Marie Tooth disease (CMT).
Background: CMT, the most common genetic neuromuscular disease, causes progressive decline in strength with increased use of ambulatory aids such as orthoses, canes, walkers and wheelchairs as the disease progresses. The CMT neuropathy score (CMTNSv2) is a validated outcome measure that categorizes the severity of neuropathy into mild, moderate or severe neuropathy in patients with CMT. The correlations between CMTNSv2 and ambulatory aid use in children has not been studied.
Design/Methods: Pediatric patients consented through the Inherited Neuropathy Consortium for the natural history clinical registry trial were assessed in this retrospective cross-sectional study. Data collected included demographics, CMTNS, type of ambulatory aid used, foot surgery, difficulty with buttons, and with utensils. Mean scores and standard deviations for the CMTNS were calculated.
Results: Of 625 eligible patients, 539 had completed data and were included in the analysis. Mean age was 13 (range 8–18, SD 2.9); Gender: 270 M, 269 F; Race: 84% Caucasian. Ambulatory aid used correlated strongly with CMTNS score (0.63, p < 0.0001). Smaller correlations were seen between ambulatory aid used and foot surgery (0.16, p =0.007), buttons (0.31, p < 0.0001) and eating utensils (0.33, p < 0.0001). Mean CMTNS for using no aids or minimal aids (shoe inserts, custom shoes, night-splints) were equivalent at 8.4. Mean CMTNS for using supramalleolar orthosis (SMO), supramalleolar ankle foot orthosis (SMAFO), molded/ankle foot orthosis (MAFO/AFO), and Arizona brace were equivalent at 10.5. Mean CMTNS for using wheelchair or scooter was 23.
Conclusions: We have developed predictive scales that correlate specific scores in CMTNS with progressive functional aid needs. We recommend prospective validation of our predictive scales in a CMT population.
Disclosure: Dr. Ramchandren has nothing to disclose. Dr. Moore has nothing to disclose. Dr. Hornyak has nothing to disclose.
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