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Association of Body Mass Index With Disease Progression in Children With Charcot-Marie-Tooth Disease
Gabrielle A Donlevy1, Kayla M D Cornett2, Sarah P Garnett2
1From the Faculty of Medicine and Health & Children's Hospital at Westmead (G.A.D., K.M.D.C., S.P.G., J.B., M.P.M.), University of Sydney, Australia; Department of Neurology (R.S., M.E.S.), and Department of Pediatrics (T.E., S.W.Y.), Carver College of Medicine, University of Iowa, Iowa City; Division of Neurology (T.E., S.W.Y.), The Children's Hospital of Philadelphia, PA; Department of Neurology (T.E., S.W.Y.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; Department of Occupational Therapy (K.A.), Children's Hospital of Philadelphia, PA; Fondazione IRCCS (D.P., I.M.), Istituto Neurologico Carlo Besta, Milan, Italy; University College London Institute of Child Health & Great Ormond Street Hospital (F.M.); Department of Neuromuscular Diseases (M.M.R.), University College London Institute of Neurology, Queen Square, United Kingdom; Center for Experimental Neurotherapeutics (Pediatrics) (R.S.F.), St. Jude Children's Research Hospital, Memphis, TN; and Department of Neurology (D.N.H., K.J.E.), University of Rochester, NY. gabrielle.donlevy@sydney.edu.au.
Background And Objectives:
The aim of this study was to evaluate the impact of body mass index (BMI) on disease progression over 2 years in children with Charcot-Marie-Tooth disease (CMT).
Methods:
BMI was classified in 242 participants aged 3-20 years with CMT enrolled in the Inherited Neuropathy Consortium, using the International Obesity Task Force (based on adult BMI values, kg/m2) criteria. Groups were categorized as severely underweight (BMI <17 kg/m2), underweight (BMI ≥17 to <18.5 kg/m2), healthy weight (BMI ≥18.5 to <25 kg/m2), overweight (BMI ≥25 to <30 kg/m2), and obese (BMI ≥30 kg/m2). Disease severity was assessed using the CMT Pediatric Scale (CMTPedS), a clinical outcome assessment of disability (0-44 points, mild to severe).
Results:
At baseline, compared with individuals being of a healthy weight (mean CMTPedS 15.48, SD 9.22), children who were severely underweight (mean CMTPedS difference 9.03, 95% CI 0.94-17.12; p = 0.02), underweight (mean CMTPedS difference 5.97, 95% CI 0.62-11.31; p = 0.02), or obese (mean CMTPedS difference 7.96, 95% CI 1.03-14.88; p = 0.015) exhibited greater disability. At 2 years, compared with individuals being of a healthy weight (mean CMTPedS 17.53, SD 9.41), children who were severely underweight exhibited greater disability (mean CMTPedS difference 9.27, 95% CI 0.90-17.64; p = 0.02). Over the 2-year periods, the mean CMTPedS for the whole sample deteriorated by 1.72 points (95% CI 1.09-2.38; p < 0.001), with severely underweight children progressing at the fastest rate (mean CMTPedS change of 2.3, 95% CI 1.53-6.13; p = 0.21). In children who did not have a change in BMI categories over 2 years (69% of sample), CMTPedS scores deteriorated faster in those who were severely underweight (mean CMTPedS change 6.40 points, 95% CI 2.42-10.38; p = 0.01) than those of healthy weight (mean CMTPedS change 1.79 points, 95% CI 0.93-2.69; p < 0.001). For children who changed BMI categories (31% of sample), CMTPedS scores deteriorated faster in children who became overweight/obese (mean CMTPedS change 2.76 points, 95% CI 0.11-5.41; p = 0.031).
Discussion:
Children with CMT who were severely underweight, underweight, or obese exhibited greater disability at baseline. Over the 2-year period in those whose BMI remained stable, severely underweight children deteriorated at the fastest rate. For children who changed BMI categories over the 2 years, CMTPedS scores deteriorated faster in children who became overweight/obese. Interventions that maintain or improve BMI toward healthy weight may reduce disability in children with CMT.
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