Scientists discovered that percentile curves in the Motor Function Measure (MFM) scoring system can facilitate the interpretation of Duchenne muscular dystrophy (DMD) disease progression, as published in the European Journal of Pediatric Neurology.
“Being able to compare the development of individual motor function within a historical group of glucocorticoid treated and untreated patients of different ages would greatly help to find subtle changes from a trajectory line,” Hafner et al wrote.
The MFM is a test that is used to assess both ambulant and nonambulant patients aged between 6 and 60 years. The authors of this study chose this clinical scoring system for assessing DMD motor function as it is widely accepted in both clinical studies and practice.
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They decided to investigate whether the generation of MFM percentile curves would better equip caregivers and researchers to evaluate the progression of motor function in patients with DMD. They obtained data of 819 patients between the ages of 6 and 15 years from the international MFM database.
“Age-dependent percentile curves were estimated using a ‘Generalized additive model for location, scale and shape’ as suggested by the World Health Organization Multicentre Growth Reference Study Group,” the researchers wrote.
In addition, they presented percentile curves and subscores for the MFM total score for patients who were on glucocorticoids and those who were not.
The results demonstrated that mean scores declined with age. With regards to glucocorticoid therapy, patients treated with glucocorticoids demonstrated higher mean values when compared to patients of the same age who were glucocorticoid-naive.
“With emerging new treatment options, the percentile curves will be an important aid to select patients with similar curves of progression for clinical trials and to monitor treatment effects on estimated evaluation of motor function,” the authors concluded.
Hafner P, Schmidt S, Schädelin S. Implementation of Motor Function Measure score percentile curves – predicting motor function loss in Duchenne muscular dystrophy. Eur J Paediatr Neurol. Published online December 14, 2021. doi:10.1016/j.ejpn.2021.11.004