Abstract
ABSTRACTObjective:To determine the responsiveness of the domain climbing up and going down stairs of the Functional Evaluation Scale for Duchenne Muscular Dystrophy (FES-DMD-D3) in a one-year follow-up study.Method:The study included 26 patients with DMD. Effect Size (ES) and Standardized Response Mean (SRM) described the scale’s responsiveness.Results:For climbing up stairs, ES showed that responsiveness was low in the three-month assessments (0.26; 0.35; 0.13; 0.17), low to moderate in the six-month assessments (0.58; 0.48; 0.33), moderate in the nine-month assessments (0.70; 0.68), and high in the 12-month assessment (0.88). SRM showed that responsiveness was low in the three-month assessments (0.29; 0.38; 0.18; and 0.19), low to moderate in the six-month assessments (0.59; 0.51; 0.36), moderate in the nine-month assessments (0.74 and 0.70), and high in the 12-month assessment (0.89). For going down stairs, ES showed that responsiveness was low in the three- and six-month assessments (0.16; 0.25; 0.09; 0.08 and 0.48; 0.35; 0.18, respectively), low to moderate in the nine-month assessments (0.59; 0.44), and moderate in the 12-month assessment (0.71). SRM showed that responsiveness was low in the three- and six-month assessments (0.25; 0.35; 0.12; 0.09 and 0.47; 0.38; 0.21, respectively), low to moderate in the nine-month assessment (0.62; 0.49), and moderate in the 12-month assessment (0.74).Conclusion:Climbing up stairs should be assessed at intervals of nine months or longer, when responsiveness is moderate to high. Going down stairs should be assessed annually because moderate responsiveness was observed in this period.
Highlights
Duchenne muscular dystrophy (DMD) is a recessive genetic disorder, caused by the alteration of the gene that encodes dystrophin protein, which is essential for maintaining the muscle cell membrane
The analysis considered Cohen’s criteria, which established that coefficients >0.20 and 0.5 and 0.8 reflected high responsiveness[25]
At six-month intervals, responsiveness was moderate in two assessments (0.59 and 0.51) and low in the first assessment (0.36)
Summary
Duchenne muscular dystrophy (DMD) is a recessive genetic disorder, caused by the alteration of the gene that encodes dystrophin protein, which is essential for maintaining the muscle cell membrane. The affected gene is located on the short arm of chromosome X, locusXp211-3. DMD is characterized by generalized and irreversible progressive deterioration of muscular strength. Symptoms appear when children are three to five years old. Lower limbs are affected first, leading to difficulty climbing steps, running, and getting up from the floor, frequent falls, and pseudohypertrophy of the calf muscles. When children are 10 to 12 years old, patients lose gait and show upper limb and respiratory muscle weakness, spine deformities, and heart dysfunction[1,2,3]
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