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Table 1 Patient reported outcome measures assessing quality of life identified in published articles with samples of people with DMD

From: Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments

PROM Respondent type Recall Period N dimensions (items) Dimensions of quality of life assessed Response options Total score range Origin Validated English copy and development papers freely available for review?
36-item Short-Form Health Survey (SF-36) v1.0 [30,31,32,33,34,35,36,37] Adult self-report Varies by dimension 8 + a single item of perceived change in health (36 items) Physical functioning; bodily pain; role limitations due to physical health problems; role limitations due to personal or emotional problems; emotional well-being; social functioning; energy/fatigue; general health perceptions Varies by dimension No total score calculated USA Yes
SF-36 Health Survey v2.0 [38] Adult self-report Varies by dimension 8 + a single item of perceived change in health (36 items) Assumed same as SF-36 v1.0 Varies by dimension No total score calculated USA No
Autoquestionnaire Qualité de vie Enfant Imagé (AUQEI) [39] Child self-report Unknown/undefined 4 + a total score (26 items) Autonomy; leisure; functioning; family; total 0–3 rating scale (with pictures) 0–78 (raw) France No
Beck Depression Inventory I (BDI) [34] Adult interview or self-report Present/today 1 total score (21 items) Depression 0–3 rating scale 0–63 (raw) USA Yes
Behavior Assessment System for Children (BASC) first edition [40] Child self-report
Parent report
Teacher report
Unknown (withdrawn from use, superseded by BASC-II and BASC-III) Unknown (withdrawn from use, superseded by BASC-II and BASC-III) Unknown (withdrawn from use, superseded by BASC-II and BASC-III) Unknown (withdrawn from use, superseded by BASC-II and BASC-III) Unknown (withdrawn from use, superseded by BASC-II and BASC-III) USA No
Child Activity Limitations Interview (CALI) [40] Child interview or self-report Last 4 weeks 1 total score (8 items chosen from a set of 21) Activity limitations 0–4 rating scale 0–32 (raw) USA Yes
Child Health Questionnaire - Parent Form 50 (CHQ-PF50) [41, 42] Parent self-report Last 4 weeks (past year for change in health) 14 (50 items) Physical functioning; role/social limitations – physical; role/social limitations – emotional; role/social limitations – behavioral; general health perceptions; bodily pain/discomfort; family activities; parent impact – time; parent impact – emotion; self-esteem; mental health; behaviour; family cohesion; change in health Varies by dimension No total score calculated USA No
Children’s Assessment of Participation and Enjoyment (CAPE) [43,44,45] Child and young adults self-report or interview Unknown/undefined 5 + a total score (55 items) Diversity of activities; intensity of activities (frequency of participation); enjoyment of activities; with whom; where; total participation Varies by dimension 0–55 (raw) USA No
Depressions-Inventar für Kinder und Jugendliche (DIKJ) 2nd edition [34] Child self-report Unknown/undefined 1 total score (26 items) Depression Unknown/undefined 0–46 (raw) Germany No
DISABKIDS generic module (DCGM-37) [34] Child self-report
Proxy report
Past 4 weeks 6 + a total score (37 items) Independence; emotion; social inclusion; social exclusion; limitation; treatment; total 1–4 rating scale 37–148 (raw) Multi-country Yes
DISABKIDS – Smileys [34] Child self-report
Proxy report
Assumed same as DCGM-37 Assumed same as DCGM-37 (12 items) Assumed same as DCGM-37 Assumed same as DCGM-37 (with pictures) 12–48 (raw) Multi-country No
Dutch Children AZL/TNO Questionnaire Quality of Life Short Form (DUC-25) [45] Child self-report Unknown/undefined 4 + a total score
(25 items)
Physical; emotional; social; home functioning; total 0–4 rating scale 0–100 (raw) Netherlands No
EuroQoL 5-domain 3-Level (EQ-5D-3L) [46, 47] Adult self-report
Proxy report
Present/today 5 (5 items) + self-rated health VAS Mobility; self-care; usual activities; pain/discomfort; anxiety/depression 1–3 rating scale −0.594 – 1 (utility scores) Multi-country Yes
Fatigue Severity Scale (FSS) [36] Adult self-report Within last week 1 total score (9 items) + global fatigue VAS Fatigue severity 1–7 rating scale 1–63 (raw) USA Yes
Generalized Anxiety Disorder Scale 7-item (GAD-7) [48] Adult self-report 2 weeks 1 total score (7 items) Anxiety 0–3 rating scale 0–21 (raw) USA Yes
Health Utilities Index Questionnaire mark 2 (HUI-2) 15Q [49, 50] Child self-report
Adult self-report
Proxy report
During past 4 weeks 7 (15 items) Sensation; mobility; emotion; cognition; self-care; pain; fertility Varies by dimension −0.03 – 1
(utility scores)
Canada Yes
Health Utilities Index Questionnaire mark 3 (HUI-3) 15Q [49, 50] Child self-report
Adult self-report
Proxy report
During past 4 weeks 8 (15 items) Vision; hearing; speech; ambulation; dexterity; emotion; cognition; pain Varies by dimension −0.36 – 1
(utility scores)
Canada Yes
Hospital Anxiety and Depression Scale (HADS) [36, 47] Adult self-report Last week 2 (14 items) Anxiety; depression 0–3 rating scale No total score calculated UK Yes
Individualized Neuromuscular Quality of Life Questionnaire (INQOL) [38] Adult self-report At the moment 10 + a total score (45 items) Weakness; locking; pain; fatigue; activities; independence; social relationships; emotions; body image; treatment; total 7-point rating scale, varies by dimension Scoring unclear UK Yes
KIDSCREEN-10 [51] Child self-report
Proxy report
Last week 1 total score (10 items) Health-related quality of life 1–5 rating scale 10–50 (raw) Multi-country Yes
KIDSCREEN-27 [52] Child self-report
Proxy report
Last week 5 (27 items) Physical well-being; psychological well-being; autonomy and parent relation; social support and peers; school environment 1–5 rating scale No total score calculated Multi-country Yes
KIDSCREEN-52 [53] Child self-report
Proxy report
Last week 10 (52 items) Physical well-being; psychological well-being; moods and emotions; self-perception; autonomy; parent relation and home life; financial resources; social support and peers; school environment; social acceptance (bullying) 1–5 rating scale No total score calculated Multi-country Yes
Life Satisfaction Index for Adolescents (LSIA) [54,55,56,57] Child and young adults self-report At present 5 + a total score (45 items) General well-being; interpersonal relationships; personal development; personal fulfilment; leisure and recreation; total 1–5 rating scale (plus 0 = N/A) 0–225 (raw) Canada Yes
Muscular Dystrophy Child Health Index of Life with Disabilities (MDCHILD) [8] Child self-report Past 4 weeks 7 + a total score (47 items) Activities of daily living & independence; positioning, transferring, & mobility; comfort & endurance; emotions & behaviour; social interaction & school; health; your overall quality of life; total Varies by dimension 0–100 (transformed) Canada Yes
Neurological Disorders Quality of Life Questionnaire (Neuro-QoL) [35] Adult self-report Varies by dimension Up to 16 (up to 564 items in item banks) Ability to participate in social roles and activities; anxiety; bowel function; cognitive function; communication; depression; emotional and behavioral dyscontrol; fatigue; lower extremity function – mobility; positive affect and well-being; satisfaction with social roles and activities; sleep disturbance; sexual function; stigma; upper extremity function – fine motor, ADL; urinary/bladder function 1–5 rating scale No total score calculated USA Yes
Offer Self-Image Questionnaire for Adolescents (OSIQ) [54, 57] Child and young adult self-report or interview Unknown/undefined 11 + a total score (130 items) Impulse control; emotional tone; body and self-image; social relationships; morals; vocational and educational goals; family relationships; mastery of the external world; psychopathology; superior adjustment; total 1–6 rating scale 130–780 (raw) USA No
Patient Health Questionnaire 9-item (PHQ-9) [48] Adult self-report 2 weeks 1 total score (9 items) Depression 0–3 rating scale 0–27 (raw) USA Yes
Pediatric Neurological Disorders Quality of Life Questionnaire (Pediatric Neuro-QoL) [35] Child self-report Varies by dimension Up to 11 (up to 161 items in item banks) Anger; anxiety; cognitive function; depression; fatigue; lower extremity – mobility; pain; social relations – interaction with adults; social relations – interaction with peers; stigma; upper extremity – fine motor, ADL 1–5 rating scale No total score calculated USA Yes
Pediatric Outcomes Data Collection Instrument (PODCI) [58,59,60,61] Child self-report
Proxy report
Varies by dimension 7 (86 items) Global function & comfort; upper extremity function; physical function and sport; transfers and mobility; comfort; POSNA happy and satisfied; POSNA expectations Varies by dimension No total score calculated USA Yes
Pediatric Quality of Life Inventory (PedsQL) 3.0 DMD module [62, 63] Child and young adult self-report
Proxy report
Past month or past 7 days (acute version) 4 (18 items) Daily activities; treatment barriers; worry; communication 0–4 rating scale No total score calculated USA Yes
PedsQL 3.0 Multidimensional fatigue scale (MFS) [63, 64] Adult self-report
Child and young adult self-report
Proxy report
Past month or past 7 days (acute version) 3 + a total score (18 items) General fatigue; sleep/rest fatigue; cognitive fatigue; total fatigue 0–4 rating scale 0–100 (transformed) USA Yes
PedsQL 3.0 Neuromuscular module (NMM) [35, 50, 51, 63,64,65,66,67,68,69] Child and young adult self-report
Proxy report
Past month or past 7 days (acute version) 3 + a total score (25 items) About my/my child’s neuromuscular disease; communication; about our family resources; total 0–4 rating scale 0–100 (transformed) USA Yes
Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales (GCS) [35, 43, 51, 58, 61,62,63,64,65, 67, 70,71,72,73,74,75,76,77] Adult self-report
Child and young adult self-report
Proxy report
Past month or past 7 days (acute version) 5 + a total score (23 items) Physical health; psychosocial health; emotional functioning; social functioning; school functioning; total 0–4 rating scale 0–100 (transformed) USA Yes
PedsQL 4.0 Generic Short-form (SF-15) [78] Adult self-report
Child and young adult self-report
Proxy report
Past month or past 7 days (acute version) 5 + a total score (15 items) Physical health; psychosocial health; emotional functioning; social functioning; school functioning; total 0–4 rating scale 0–100 (transformed) USA Yes
Pittsburgh Sleep Quality Index (PSQI) [35, 74] Adult self-report Past month 8 + a total score (10 items) Subjective sleep quality; sleep latency; sleep duration; sleep efficiency; sleep disturbance; use of sleep medication; daytime dysfunction; total Varies by dimension 0–21 (raw) USA Yes
Satisfaction with Life Scale (SWLS) [48, 54] Adult self-report Undefined/present time 1 total score (5 items) Life satisfaction 1–7 rating scale 5–35 (raw) USA Yes
Strength and Difficulties Questionnaire (SDQ) [79,80,81] Child self-report
Proxy report
Last 6 months 5 + a total score (25 items) + an impact supplement Emotional symptoms; conduct problems; hyperactivity/inattention; peer relationship problems; total difficulties; prosocial behaviour 0–2 rating scale 0–40 (raw) UK Yes
Strips of Life with Emoticons Questionnaire (SOLE) [82] Child self-report Specific scenarios 1 total score (33 items) Quality of life 0–2 rating scale (with pictures) 0–66 (raw) Italy No
TNO-AZL Children’s Quality of Life questionnaire (TACQoL) [83] Child self-report
Proxy report
The last few weeks 7 (56 items) Physical functioning; motor functioning; independent daily functioning; cognitive functioning and school performance; social contacts; positive moods; negative moods Varies by dimension No total score calculated Netherlands No
TNO-AZL
Adult Quality of Life questionnaire (TAAQoL) [83]
Adult self-report In the last month 12 (45 items) Gross motor functioning; fine motor functioning; cognition; sleep; pain; social contacts; daily activities; sex; vitality; happiness; depressive mood; anger Varies by dimension No total score calculated Netherlands No
World Health Organisation Quality of Life Scale-Brief Version (WHOQOL-BREF) [33, 35,36,37] Adult self-report or interview
Proxy report
2 weeks 4 (26 items, 24 items make up domain scores) Physical health; psychological; social relationships; environment 1–5 rating scale No total score calculated Multi-country Yes
  1. References next to PROM names represent published studies where the PROM has been used in a sample of people with DMD. PROM = patient reported outcome measure