Table 1 Patient reported outcome measures assessing quality of life identified in published articles with samples of people with DMD
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Assessment System for Children (BASC) first edition [40] | Child self-report Parent report Teacher report | Unknown (withdrawn from use, superseded by BASC-II and BASC-III) | Unknown (withdrawn from use, superseded by BASC-II and BASC-III) | Unknown (withdrawn from use, superseded by BASC-II and BASC-III) | Unknown (withdrawn from use, superseded by BASC-II and BASC-III) | Unknown (withdrawn from use, superseded by BASC-II and BASC-III) | USA | No |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 |
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 |
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 |
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 |
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 | |
Adult self-report | Undefined/present time | 1 total score (5 items) | Life satisfaction | 1–7 rating scale | 5–35 (raw) | USA | Yes | |
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 |