Variables associated with upper extremity function in patients with Duchenne muscular dystrophy.

Area of Science:

• Neurology
• Rehabilitation Medicine
• Pediatric Neuromuscular Disorders

Background:

• Preserving upper extremity (UE) function is vital for independence and quality of life in Duchenne muscular dystrophy (DMD) patients.
• Limited knowledge exists regarding factors influencing UE function in DMD, hindering effective clinical decision-making.
• Understanding these variables is essential for developing targeted interventions and management strategies.

Purpose of the Study:

• To investigate variables associated with UE function in individuals with DMD.
• To identify factors that positively and negatively impact UE function in this population.
• To provide insights for clinical management and improve patient outcomes.

Main Methods:

• Utilized data from an international web-based questionnaire completed by 213 DMD patients.
• Employed multivariable linear regression analyses with six dependent UE function variables.
• Examined 26 independent variables including patient characteristics, medication, therapy, pain, stiffness, and participation.

Main Results:

• Identified twelve independent variables significantly related to UE function in DMD.
• Negative associations with UE function included later disease stage, scoliosis, higher age, UE splint use, stiffness, and elbow pain.
• Positive associations included school/work participation, corticosteroid use, higher BMI, and earlier age at diagnosis.

Conclusions:

• Corticosteroid use and participation in school/work activities are positively linked to UE function in DMD.
• Managing pain, stiffness, and preventing scoliosis are crucial for maintaining UE function.
• These identified variables collectively explain a substantial portion (56-81%) of the variation in UE function.