Responsiveness and Minimal Clinically Important Difference of the Chinese Version of the Motor Function Measure-32 in Children and Adolescents with Duchenne Muscular Dystrophy

ABSTRACT

Purpose: This study aimed to examine the responsiveness and minimal clinically important difference of the Motor Function Measure 32-Chinese version (MFM 32-CN) in children and adolescents with Duchenne muscular dystrophy (DMD). Methods: This observational, prospective cohort study assessed changes in motor function over 12 months using the MFM 32-CN in 108 pediatric patients with DMD. Results: Moderate to high internal responsiveness was found for MFM total, D1, and D2 scores (SRM = 0.46–0.83). Sufficient external responsiveness was observed in all MFM scores, as determined by a good correlation with the Patient Global Impression of Change (PGIC) ratings (r = 0.54–0.72, p < .01). The estimated MCID values derived from different anchor-based methods ranged from 4.7 to 6.0, with a median of 5.0 points (%). Conclusion: The MFM 32-CN demonstrates sufficient internal and external responsiveness as a measure of motor function in children and adolescents with DMD. The present study established the MCID of MFM-32 in pediatric patients with DMD.

KEYWORDS:

• Motor function measure
• Chinese version
• responsiveness
• minimal clinically important difference
• Duchene muscular dystrophy

Acknowledgments

The authors would like to show their gratitude to all the children and families who have participated in this study. The authors would like to acknowledge the NMD MDT Group in Shenzhen children’s hospital for their support and assistance throughout participant enrollment and data collection.

Disclosure statement

No potential conflict of interest was reported by the author(s).

Data availability statement

The data that support the findings of this study are available from the corresponding author upon reasonable request.

Additional information

Funding

This work was supported by the Shenzhen Disabled Persons’ Comprehensive Service Center under Grant [number JB2017-16-3] and Sanming Project of Medicine in Shenzhen under Grant [SZSM201612036].