#AAN2018 – Neuropathy-based Scale May Help Predict Mobility Needs in Children with CMT

Researchers found that a neuropathy-based scale may help predict the need for mobility assistance in children with Charcot-Marie-Tooth disease.

The results will be presented April 23 at the 2018 American Academy of Neurology (ANN) Annual Meeting, April 21-27 in Los Angeles in the session dedicated to Child Neurology and Developmental Neurology: Neuromuscular Disease.

The presentation is titled “Predicting Ambulatory Aid Needs with Disease Progression in Charcot-Marie-Tooth Disease.”

Progressive loss of strength is a feature that characterizes those affected by Charcot-Marie-Tooth disease (CMT), and as the disease progresses, patients require mobility aids, such as orthoses, canes, walkers, and wheelchairs.

The CMT neuropathy score version 2 (CMTNSv2) is a validated measure that assesses the degree of disease severity — categorized into mild, moderate, or severe — in CMT patients.

The CMTNS is a composite 36‐point scale based on pata ient’s symptoms, signs at neurological examination, and neurophysiological assessment.

But “the correlations between CMTNSv2 and ambulatory aid use in children has not been studied,” researchers said.

They set out to develop a predictive scale that would correlate CMTNSv2 scores with the specific requirements of mobility aids in children with CMT.

Investigators performed a retrospective cross-sectional analysis using data of children with CMT, collected from the Inherited Neuropathy Consortium, a multi-center platform dedicated to clinical research in different forms of CMT to improve the care of patients.

“Data collected included demographics, CMTNS, type of ambulatory aid used, foot surgery, difficulty with buttons, and with utensils,” researchers explained.

The final analysis included 539 children ages 8-18 (mean age 13) with CMT, equally distributed among genders — 270 boys and 269 girls.

The analysis showed a marked and significant correlation between the need for mobility aids and CMTNS score. A statistical significance was also found between the ambulatory aid used and foot surgery, difficulty with buttons, and eating with utensils.

Researchers saw an increased need for ambulatory assistance correlated with higher CMTNS scores. For example, they established that no need for aids or minimal aids, such as shoe inserts, custom shoes, or night-splints correlated with a mean CMTNS score of 8.4.

The CMTNS score increased to 10.5 when patients required the use of ankle foot orthosis, which support the foot just above the ankle bone.

When the mean CMTNS score jumped to 23, it correlated with the need for a wheelchair or scooter.

“We have developed predictive scales that correlate specific scores in CMTNS with progressive functional aid needs,” researchers said. They also recommended their predictive scales be validated in a group of children with Charcot-Marie-Tooth disease.