Instruments to assess upper-limb function in children and adolescents with neuromuscular diseases: a systematic review

Authors:

Davoli, G. B. Q., Cardoso, J., Silva, G. C., Moreira, R. F. C., and Mattiello-Sverzut, A. C.

Abstract:

AIM: To synthesize clinical and scientific evidence regarding the instruments available to assess upper-limb function in paediatric patients with neuromuscular disease (NMD).

METHOD: This systematic review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses and COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines (Prospective Registry of Systematic Reviews no. CRD42020140343). Two independent reviewers searched the PubMed/MEDLINE, LILACS, Embase, and Scopus databases. Inclusion criteria were cross-sectional or longitudinal studies or randomized controlled trials that used scales or questionnaires to assess upper-limb function in paediatric patients with NMDs. The COSMIN Risk of Bias checklist and criteria for good measurement properties were applied to assess the methodological quality of the instruments.

RESULTS: In total, 34 articles and 12 instruments were included. The Brooke Upper Extremity (n=16) and Performance of Upper Limb (PUL) (n=12) instruments were the most used tools. The PUL and Duchenne muscular dystrophy (DMD) Upper Limb patient-reported outcome measures (PROMs) tested more measurement properties and provided higher methodological quality scores for patients with DMD. Likewise, the Revised Upper Limb Module (RULM) was the most suitable instrument for patients with spinal muscular atrophy. No instrument has been devised to assess upper-limb function in patients with Charcot-Marie-Tooth disease and no other disease-specific instruments were found.

INTERPRETATION: The PUL, DMD Upper Limb PROM, and RULM are the most suitable instruments to assess upper-limb function in the two most prevalent paediatric NMDs. The identified gaps and methodological flaws of the available instruments indicate a need to develop high-quality instruments to assess other types of paediatric NMDs.

DOI:

10.1111/dmcn.14887

Journal:

Dev Med Child Neurol

issn:

0012-1622

Publication year:

2021

pages:

Functional status:

Physical functioning

Age:

Children (0-18)

Disease:

Diseases of and symptoms related to the musculoskeletal system and connective tissue
Diseases of and symptoms related to the nervous system

PRO / non-PRO:

Non-patient Reported Outcome
Patient Reported Outcome

Type of measurement instrument:

1 - Questionnaires
4 - Performance-based tests
6 - Clinical rating scales
7 - Observations

Instrument:

Brooke Upper Extremity Scale
CUE - Capabilities of Upper Extremity instrument
DMD Upper Limb PROM - Duchenne Muscular Dystrophy Upper Limb Patient Reported Outcome Measure
Duchenne Muscular Dystrophy Functional Ability Self-Assessment Tool
EEFES - Escala de Evaluación Funcional de Extremidades Superiores
MFM - Motor Function Measure (AND MFM-20 - 20 item version)
Paediatric Outcomes Data Collection Instrument
PUL - Performance of the upper limb
QUEST - Quality of Upper Extremity Skills Test
ULM - Upper Limb Module (versions: RULM - Revised ULM)
WBQ - Web-based questionnaire