TREAT-NMD Core Datasets

Patient-reported outcome measures

Important note: All registries must collect a minimum of one patient-reported outcome measure for each individual. The list of outcome measures included in the item Patient-reported outcome measure below represents several measures known to be used for LGMD. Registries are not expected to collect the full list. Selection of appropriate outcome measure(s) is at the discretion of the clinician and/or preference of the individual

Items in this group

Patient-reported outcome measure CR PR longitudinal

Patient-reported outcome measure CR PR

Patient-reported outcome measure score CR PR

Record

Patient-reported outcome measure CR PR longitudinal

This record is longitudinal: For each record instance, a datestamp with month and year must be captured. See the introduction for more information.

Patient-reported registries should add the questionnaires of the outcome measures they intend to collect to their registry and calculate the score from the replies. However, for TREAT-NMD, only the score is relevant.

Changes:

In version 1.0, it was clarified that patient-reported registries should add the questionnaires of the outcome measures they intend to collect to their registry and calculate the score from the replies.

Patient-reported outcome measure CR PR

Item type:	single selection
Usage in other datasets:	SMA DMD

Value ID	Description
ACTIVLIM	ACTIVLIM (Measurement of Activity Limitations), provide score between 0 and 36
EQ-5D-5L state	EQ-5D-5L health state, provide 5-digit code
EQ-5D-5L VAS	EQ-5D-5L VAS, provide score between 0 and 100
INQoL	INQoL (Individualised Neuromuscular Quality of Life Questionnaires) 2.0, provide score between 0 and 274
IPAQ category	IPAQ (International Physical Activity Questionnaire), provide 1 for low, 2 for moderate, or 3 for high level of physical activity
IPAQ score	IPAQ (International Physical Activity Questionnaire), provide MET-minutes per week
PedsQL 3.0 Core Module	PedsQL 3.0 Core Module, provide score between 0 and 92
PedsQL 3.0 Neurosmuscular Module	PedsQL 3.0 Neurosmuscular Module, provide score between 0 and 100
PedsQL Multidimensional Fatigue Scale	PedsQL Multidimensional Fatigue Scale, provide score between 0 and 72
PODCI	PODCI (Pediatric Outcomes Data Collection Instrument), provide score between 0 and 100
PROMIS mobility adult	PROMIS (Patient-Reported Outcomes Measurement Information System) mobility measure for adults, provide T-score
PROMIS mobility child	PROMIS (Patient-Reported Outcomes Measurement Information System) mobility measure for children, provide T-score
PROMIS upper extremity adult	PROMIS (Patient-Reported Outcomes Measurement Information System) upper extremity measure for adults, provide T-score
PROMIS upper extremity child	PROMIS (Patient-Reported Outcomes Measurement Information System) upper extremity measure for children, provide T-score
SF-36v2	SF-36v2 Health Survey, provide score between 0 and 100
QoL-gNMD	QoL-gNMD (Quality of Life in Genetic Neuromuscular Diseases), provide score between 0 and 78

Changes:

Before version 1.0, this item was erroneously marked as non-mandatory.

Patient-reported outcome measure score CR PR

The Patient-reported outcome measure value description specifies which type of score must be provided.

Item type:	decimal
Usage in other datasets:	SMA DMD

Changes:

Before version 1.0, this item was erroneously marked as non-mandatory.

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