TY - JOUR
T1 - Trajectories of motor function in children with Duchenne muscular dystrophy
T2 - A longitudinal study on a Colombian population
AU - Ortiz-Corredor, Fernando
AU - Sandoval-Salcedo, Andres
AU - Castellar-Leones, Sandra
AU - Soto-Pena, Diana
AU - Ruíz-Ospina, Edicson
AU - Suarez-Obando, Fernando
N1 - Publisher Copyright:
© 2023 European Paediatric Neurology Society
PY - 2023/11
Y1 - 2023/11
N2 - Duchenne Muscular Dystrophy (DMD) is characterized by an initial increase in motor function followed by a plateau phase and then entering a phase of steady decline. However, motor evolution of DMD have not been evaluated in developing countries. Therefore, this study aims to evaluate the trajectory of motor function in a sample of Colombian children with DMD. We included 119 children with DMD aged 4.8–19.3 years (mean follow-up = 1.7 years). A linear mixed model was used with age as the time scale and adjusted for covariates using a stepwise regression. Participants showed a progressive decline in motor skills from the age of 5 years with a decrease in speed around the age of 11 years (p < 0.001). After age 11, the decline in motor function was observed to continue until age 20 but at a slower rate (βAge = −9.64. and βAge2 = 0.18, p < 0.001 for both). Educational inclusion, glucocorticoid treatment and the number of mutated exons were shown to be associated with the motor performance. These findings may indicate that the evolution of DMD maintains similar patterns between high income countries and the Colombian population. They allow us to adapt and develop treatments that impact the population with DMD in Colombia, based in international evidence.
AB - Duchenne Muscular Dystrophy (DMD) is characterized by an initial increase in motor function followed by a plateau phase and then entering a phase of steady decline. However, motor evolution of DMD have not been evaluated in developing countries. Therefore, this study aims to evaluate the trajectory of motor function in a sample of Colombian children with DMD. We included 119 children with DMD aged 4.8–19.3 years (mean follow-up = 1.7 years). A linear mixed model was used with age as the time scale and adjusted for covariates using a stepwise regression. Participants showed a progressive decline in motor skills from the age of 5 years with a decrease in speed around the age of 11 years (p < 0.001). After age 11, the decline in motor function was observed to continue until age 20 but at a slower rate (βAge = −9.64. and βAge2 = 0.18, p < 0.001 for both). Educational inclusion, glucocorticoid treatment and the number of mutated exons were shown to be associated with the motor performance. These findings may indicate that the evolution of DMD maintains similar patterns between high income countries and the Colombian population. They allow us to adapt and develop treatments that impact the population with DMD in Colombia, based in international evidence.
KW - Disability
KW - Duchenne syndrome
KW - motor function
KW - Neuromuscular disease
KW - Trajectories
UR - http://www.scopus.com/inward/record.url?scp=85174177237&partnerID=8YFLogxK
U2 - 10.1016/j.ejpn.2023.10.002
DO - 10.1016/j.ejpn.2023.10.002
M3 - Article
C2 - 37856935
AN - SCOPUS:85174177237
SN - 1090-3798
VL - 47
SP - 105
EP - 109
JO - European Journal of Paediatric Neurology
JF - European Journal of Paediatric Neurology
ER -