Review of patient-reported outcome measures for use in myotonic dystrophy type 1 patients
- Abstract:
- INTRODUCTION: The heterogeneity of symptoms experienced by myotonic dystrophy type 1 (DM1) patients means patient-reported outcome (PRO) assessments are uniquely suited to address this through questionnaires.
METHODS: A structured literature review of PRO measures used in DM1 populations, comparing psychometric data from this population was undertaken.
RESULTS: One health status measure, 3 activities of daily living (ADL) scales, 3 health-related quality of life (HRQOL) assessments, and 5 sleep and fatigue measures have validity and reliability information from DM1 populations. The Myotonic Dystrophy Health Index and DM1 Activity and Participation Scale (DM1-Activ) have the strongest validity and reliability evidence. The DM1-Activ(c) has been published recently and builds on the DM1-Activ by adding more relevant items.
CONCLUSIONS: The PRO instruments we identified have varying psychometric evidence in DM1 populations; all require further testing to be confident of their ability to make accurate and valid measurements of symptoms, HRQOL, and ADL in a DM1 population. Muscle Nerve 56: 86-92, 2017. - DOI:
- 10.1002/mus.25469
- URL:
- https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.25469
- Journal:
- Muscle Nerve
- issn:
- 0148-639x
- Publication year:
- 2017
- pages:
- 86-92
- Symptom status:
- Physical state
- Functional status:
- Physical functioning
Social functioning - General health perceptions / HRQoL:
- Health-related quality of life
- Age:
- Adults (18-65)
- Disease:
- Congenital malformations, deformations and chromosomal abnormalities and related symptoms
Diseases of and symptoms related to the musculoskeletal system and connective tissue - PRO / non-PRO:
- Patient Reported Outcome
- Type of measurement instrument:
- 1 - Questionnaires
- Instrument:
- CFS - Chalder Fatigue Scale [alias: FQ - Fatigue Questionnaire][alias: FRS - Fatigue Rating Scale]
DM1 Activ-c - DM1 Activity and Participation Scale - c
DM1-activ - DM1 Activity and Participation Scale
DSS - Daytime Sleepiness Scale
ESS - Epworth Sleepiness Scale
FDSS - Fatigue and Daytime Sleepiness Scale
INQol - Individualised Neuromuscular Quality of Life
LIFE-H - Assessment of Life Habits [see: ZUYD (NL)]
MDHI - Myotonic Dystrophy Health Index