Reliability of home-based, motor function measure in hereditary neuromuscular diseases

Xiomara Ruiz-Cortes1, Fernando Ortiz-Corredor1,2, Camilo Mendoza-Pulido1

  • 11 Division of Rehabilitation Medicine, Instituto de Ortopedia Infantil Roosevelt, Bogota, Colombia.

Insights

The Motor Function Measure (MFM) scale reliably assesses hereditary neuromuscular disease severity at home. This tool shows excellent reliability and correlates well with pediatric outcome measures for mobility.

Area of Science:

  • Neurology
  • Pediatrics
  • Rehabilitation Medicine

Background:

  • Hereditary neuromuscular diseases (HNMDs) require reliable assessment tools for monitoring disease severity and progression.
  • The Motor Function Measure (MFM) scale is used to evaluate motor function in various pediatric conditions.
  • Assessing disease progression in HNMDs can be challenging, particularly in home-based settings.

Purpose of the Study:

  • To evaluate the reliability of the Motor Function Measure (MFM) scale when administered in both home and clinic settings for children with HNMDs.
  • To assess the correlation between the MFM scale and the Paediatric Outcomes Data Collection Instrument (PODCI).
  • To determine the MFM scale's utility for tracking disease severity and progression in HNMD patients.

Main Methods:

  • A prospective study involving two assessors rating children with HNMDs using the MFM scale.
  • Assessments were conducted at the clinic and again at the patients' homes two weeks later.
  • Intraclass correlation coefficient (ICC) and weighted kappa were calculated to determine reliability; correlation with PODCI domains was also evaluated.

Main Results:

  • The MFM scale demonstrated excellent inter-rater reliability (ICC, 0.98) across both settings.
  • Home-based MFM showed excellent correlation with the PODCI 'basic mobility and transfers' domain and moderate correlation with the 'upper extremity' domain.
  • Weighted kappa for individual items ranged from excellent to poor, indicating variability in item-level reliability.

Conclusions:

  • The Motor Function Measure (MFM) scale is a reliable tool for assessing patients with HNMDs, even when administered in a home-based setting.
  • Home-based MFM assessments provide valuable data for tracking motor function and disease progression in pediatric neuromuscular conditions.
  • The MFM scale's correlation with specific PODCI domains highlights its relevance for evaluating functional outcomes in children with HNMDs.

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