Motor function measure scale, steroid therapy and patients with Duchenne muscular dystrophy

Abstract

OBJECTIVE: To assess the evolution of motor function in patients with Duchenne muscular dystrophy (DMD) treated with steroids (prednisolone or deflazacort) through the Motor Function Measure (MFM), which evaluates three dimensions of motor performance (D1, D2, D3). METHODS: Thirty-three patients with DMD (22 ambulant, 6 non-ambulant and 5 who lost the capacity to walk during the period of the study) were assessed using the MFM scale six times over a period of 18 months. RESULTS: All the motor functions remained stable for 14 months in all patients, except D1 for those who lost their walking ability. In ambulant patients, D2 (axial and proximal motor capacities) motor functions improved during six months; an improvement in D3 (distal motor capacity) was noted during the total follow-up. D1 (standing posture and transfers) and total score were useful to predict the loss of the ability to walk. CONCLUSIONS: The use of the MFM in DMD patients confirms the benefits of the steroid treatment for slowing the progression of the disease.

Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials

“…Kinali et al had shown a well-tolerated treatment and efficacy in terms of functional scores in comparison to untreated patients, through a small DMD sample. 17,19 Recently, promising therapeutics such as antisense oligonucleotides have emerged in DMD, strengthening the need for validated tools monitoring longitudinal changes in neuromuscular status over time in these children. Considering that DMD causes rapid impairment of motor function, it is crucial to use a valid, reliable, feasible, and sensitive tool with established metrological properties.…”

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

“…Comparisons between the natural history studies in the pre-glucocorticoid era and those after glucocorticoid therapy have demonstrated benefits in the motor function, giving longer independent gait, better core stabilization and upper limb function, prevention of spine deformities, and delaying the settlement of lower limb deformities [32][33][34][35][36][37][38][39] . The use of glucocorticoids is also responsible for nonmotor benefits, particularly in preserving respiratory function, preventing cardiomyopathy, improving quality of life parameters and prolonging life itself 38,40,41 .…”

Predicting the loss of ambulation in Duchenne muscular dystrophy

“…The disease is caused by deficiency of the sarco lemmal protein dystrophin and results in weakness affecting the proximal portions of the limbs, usually starting during the third and fourth years of life, in association with progressive loss of functional abilities 1 . The unique medical treatment available is steroid therapy, which appears to prolong walking capacity by at least two years 2,3 . Strength loss, such as in hip extension and ankle dorsiflexion, is considered the primary predictor of loss of ambu lation in DMD 4 , leading to progressively reduced walking capacity 5,6 .…”

“…Future evaluations, including those with a higher number of patients, prospective studies, and studies with similar physical therapy protocols, are necessary to confirm the results presented in that study. In addition, it is important to compare these results with measurements using traditional motor scales, such as the Motor Function Measure (MFM),the 6minute walk test (6MWT), and the North Star Ambulatory Assessment (NSAA) 3,5,6 . Finally, patients' use of ste roids could be tested to determine the variation of compensa tory movements under the effects of medication.…”