Table 5 Summary of utility outcomes for included studies

Bartlett et al. (2010) [33]

Adolescents with cerebral palsy

Proxy (parent)

HUI3

Gross Motor Function Measure 66 Items (GMFM-66)

Spinal Alignment and Range of Motion Measure (SAROMM)

NA

Overall utility scores not reported, only vision, cognition and pain dimensions used - ambulation, hearing, speech, dexterity and emotion dimensions all excluded due to conceptual overlap with other measures

NA

Bray et al. (2017) [15]

Children and adolescents with impaired mobility (relevant conditions: cerebral palsy, hemiplegia, muscular dystrophy)

Self-reported and proxy (parent); matched-pairs

HUI2, HUI3 and EQ-5D-Y

Visual analogue scale (VAS)

Child self-reported

HUI2: 0.53 (±0.07)

HUI3: 0.22 (±0.09)

EQ-5D-Y: 0.24 (±0.30)

Parent proxy

HUI2: 0.49 (±0.09)

HUI3: 0.16 (±0.10)

EQ-5D-Y: 0.01 (±0.14)

NA

Burstrom et al. (2014) [11]

Children and adolescents with functional motor, orthopaedic and medical disabilities (relevant conditions: artrogryposis multiple congenital, myelomeningocele, cerebral palsy, orthopaedic lower limb deformities, achondroplasia)

Self-reported

EQ-5D-Y

VAS

KIDSCREEN-27

KIDSCREEN-10

Self-rated health (SRH)

Life satisfaction ladder (LSL)

NA

Overall utility scores not reported, only dimension scores reported

NA

Cavazza et al. (2016) [34]

Adolescents and adults with Duchenne muscular dystrophy

Self-reported

EQ-5D-Y

VAS

Barthel Index

Mean 0.24

Varied by country, ranging from − 0.71 (±0.41) in Sweden to 0.66 (±0.08) in Bulgaria

NA

Christensen et al. (2016) [35]

Children and adolescents with cerebral palsy

Proxy (caregiver)

HUI3

Wong-Baker FACES Pain Rating Scale

NA

Overall utility scores not reported, only HUI3 pain dimension measured

NA

Findlay et al. (2015) [36]

Children with cerebral palsy

Self-reported and proxy (caregiver); proportion of proxy data not reported

HUI3

DISABKIDS Chronic Generic module (DCGM-37)

DISABKIDS Smiley measure (DSM)

Wong-Baker FACES Pain Rating Scale

NA

Overall utility scores not reported, only HUI3 pain dimension measured

NA

Hendriksz et al. (2014) [37]

Children and adults with Morquio A syndrome

Self-reported

EQ-5D-5 L

Brief Pain Inventory Short Form (BPI-SF)

Adolescent Pediatric Pain Tool (APPT)

NA

Overall utility scores not reported

Child group: utility score by wheelchair use frequency

No use: 0.534

Occasional use: 0.664

Full-time use: − 0.180

Adult group: utility score by wheelchair use frequency

No use: 0.846

Occasional use: 0.582

Full-time use: 0.057

Karmur and Kulkarni (2018) [38]

Children and adolescents with spina bifida (myelomeningocele) and shunted hydrocephalus

Proxy (caregiver)

HUI2 and HUI3 (version used to calculate utility scores not stated)

Hydrocephalus Outcome Questionnaire (HOQ)

0.51 (±0.28)

NA

Kennes et al. (2002) [39]

Children with cerebral palsy

Proxy (caregiver)

HUI3

Functional Independence Measure for Children (WeeFIM)

Strength and Difficulties Questionnaire (SDQ)

Wide Range Achievement Test (WRAT)

NA

Overall utility scores not reported, only dimension scores reported

NA

Kulkarni et al. (2004) [40]

Children with hydrocephalus

Proxy (parent)

HUI2

HOQ

NA

Overall utility scores not reported

NA

Kulkarni et al. (2006) [41]

Children with hydrocephalus

Proxy (parent)

HUI2

HOQ

NA

Overall utility scores not reported

NA

Kulkarni et al. (2008) [42]

Children with hydrocephalus

Proxy (caregiver)

HUI3

HOQ

0.58 (±0.32)

NA

Kulkarni et al. (2008) [43]

Children with hydrocephalus

Self-reported and proxy (caregiver); proxy-reported HUI3 and HOQ, self-reported cHOQ

HUI3

Child-completed version of the HOQ (cHOQ)

0.71 (±0.27)

NA

Landfeldt et al. (2016) [44]

Children and adolescents with Duchenne muscular dystrophy

Proxy (caregiver); although patients included in data collection, only proxy utility data reported

HUI (version not stated)

Pediatric Quality of Life Inventory (PedsQL) neuromuscular module 3.0

NA

Overall utility scores not reported

Utility score by ambulatory status

Early ambulatory: 0.75

Late non-ambulatory: 0.15

Lindquist et al. (2014) [45]

Adults who experienced hydrocephalus in infancy

Self-reported

15D

NA

Study group: 0.92

Control group: 0.95

Livingston and Rosenbaum (2008) [46]

Adolescents with cerebral palsy

Proxy (parent)

HUI3

Quality of Life Instrument for People with Developmental Disabilities (QOL Instrument)

NA

Overall utility scores not reported

NA

Lopez-Bastida et al. (2017) [47]

Children with spinal muscular atrophy

Proxy (caregiver)

EQ-5D-3 L

VAS

Barthel Index

0.16 (±0.44)

Utility score by spinal muscular atrophy type

Type II spinal muscular atrophy sub-group: − 0.01 (±0.35)

Scores for Type I and III not reported

Morrow et al. (2011) [48]

Children with chronic conditions (relevant condition: cerebral palsy)

Self-reported and proxy (parent and doctor); matched groups

HUI2 and HUI3

NA

NA

Overall utility scores not reported for relevant sub-group

NA

Penner et al. (2013) [49]

Children and adolescents with cerebral palsy

Self-reported and proxy (parent and physician); proxy-reported HUI3 pain score, self-reported Wong-Baker FACES Pain Scale

HUI3

Wong-Baker FACES Pain Rating Scale

NA

Overall utility scores not reported, only HUI3 pain dimension measured

NA

Perez Sousa et al. (2017) [50]

Children and adolescents with cerebral palsy

Self-reported and proxy (mother and father); matched groups

EQ-5D-Y

VAS

NA

Overall utility scores not reported, only dimension scores reported

NA

Petrou and Kupek (2009) [51]

Children with childhood conditions (relevant conditions: microcephaly, cerebral palsy, spinal muscular atrophy, muscular dystrophy, spina bifida)

Proxy (caregiver)

HUI3

NA

Mean utility by condition

Microcephaly: 0.141

Cerebral palsy: 0.276

Muscular dystrophy or spinal muscular atrophy: 0.386

Spina bifida: 0.452

NA

Rocque et al. (2015) [52]

Children and adolescents with spina bifida

Self-reported and proxy (caregiver); predominantly proxy (11% self-reported)

HUI3

NA

NA

Overall utility scores not reported

Utility score by type of spina bifida

Myelomeningocele: 0.51

Closed spinal dysraphism: 0.77

Utility score by treatment history

No history of shunt or CM-II decompression: 0.74

Shunt but no CM-II decompression: 0.49

Shunt and CM-II decompression: 0.29

Rosenbaum et al. (2007) [53]

Adolescents with cerebral palsy

Self-reported and proxy (parent); proxy-reported HUI3, 34% self-reported QOL Instrument

HUI3

QOL Instrument

0.42 (±0.41)

Utility score by GMFCS level

GMFCS I: 0.84 (±0.20)

GMFCS II: 0.50 (±0.31)

GMFCS III: 0.39 (±0.31)

GMFCS IV: 0.16 (±0.26)

GMFCS V: − 0.08 (±0.23)

Sims-Williams et al. (2017) [54]

Children with spina bifida

Self-reported and proxy (caregiver); matched pairs

HUI3

VAS

Child self-reported

0.58 (95% CI 0.49–0.66)

Caregiver proxy

0.55 (95% CI 0.47–0.63)

NA

Slaman et al. (2015) [55]

Adolescents and young adults with cerebral palsy

Self-reported

SF-6D

36-Item Short Form Survey (SF-36) - SF-6D utility outcomes derived from SF-36

Baseline

Control: 0.74 (±0.12)

Intervention: 0.75 (±0.10)

6 months post intervention

Control: 0.77 (±0.12)

Intervention: 0.80 (±0.03)

NA

Tilford et al. (2005) [56]

Children with spina bifida

Proxy (caregiver)

HUI2

Quality of Well-Being scale (QWB)

Case group: 0.55 (±0.24)

Utility by case group lesion level

Sacral lesion: 0.61 (±0.26)

Lower lumbar lesion: 0.54 (±0.19)

Thoracic lesion: 0.45 (±0.25)

General population control group: 0.93 (±0.11)

Usuba et al. (2014) [57]

Adolescents and adults with cerebral palsy

Self-reported and proxy (undefined); predominantly proxy (40% self-reported)

HUI3 and AQoL

SRH

Baseline (both groups)

HUI3: 0.29 (±0.39)

AQoL: 0.35 (±0.33)

8-year follow-up (both groups)

HUI3: 0.29 (±0.38)

AQoL: 0.35 (±0.32)

NA

Vitale et al. (2001) [58]

Adolescents with orthopaedic problems (relevant condition: cerebral palsy)

Self-reported

EQ-5D (version not stated)

SF-36

Cerebral palsy sub-group: 0.922

NA

Wallander et al. (2009) [59]

Adults treated for CTEV in infancy

Self-reported

EQ-5D (version not stated)

SF-36

VAS

American Academy of Orthopaedic Surgeons Foot and Ankle Questionnaire

NA

Overall utility scores not reported

NA

Young et al. (2010) [22]

Adolescents and young adults with cerebral palsy

Self-reported and proxy (caregiver); predominantly self-reported (45% proxy)

HUI3 and AQoL

SRH

Health Assessment Questionnaire (HAQ)

Combined age groups

HUI3: 0.30 (±0.42)

AQoL: 0.28 (±0.33)

Utility score by age group (HUI3 / AQoL)

‘Youth’ group: 0.30 (±0.43) / 0.28 (±0.34)

‘Adult’ group: 0.31 (±0.40) / 0.28 (±0.314)

Youth group: utility score by GMFCS level (HUI3 / AQoL)

GMFCS I: 0.67 (±0.32) / 0.58 (±0.31)

GMFCS II: 0.59 (±0.35) / 0.53 (±0.34)

GMFCS III: 0.43 (±0.39) / 0.31 (±0.32)

GMFCS IV: 0.08 (±0.25) / 0.06 (±0.12)

GMFCS V: − 0.13 (±0.19) / 0.01 (±0.07)

Adult group: utility score by GMFCS level (HUI3 / AQoL)GMFCS I: 0.64 (±0.30) / 0.52 (±0.32)

GMFCS II: 0.50 (±0.39) / 0.33 (±0.24)

GMFCS III: 0.53 (±0.27) / 0.39 (±0.27)

GMFCS IV: 0.06 (±0.21) / 0.10 (±0.20)

GMFCS V: − 0.14 (±0.20) / 0.02 (±0.06)

Young et al. (2013) [21]

Adolescents and young adults with spina bifida

Self-reported and proxy (caregiver); predominantly self-reported (15% proxy)

HUI3 and AQoL

SRHHAQ

Combined age groups

HUI3: 0.52 (±0.28)

AQoL: 0.34 (±0.24)

Utility score by age group (HUI3 / AQoL)

‘Youth’ group: 0.58 (±0.27) / 0.37 (±0.26)

‘Adult’ group: 0.36 (±0.27) / 0.25 (±0.17)

Utility score by lesion level (HUI3 / AQoL)

Thoracic: 0.29 (±0.14) / 0.22 (±0.14)

High-lumbar: 0.44 (±0.31) / 0.28 (±0.23)

Low-lumbar: 0.63 (±0.23) / 0.39 (±0.21)

Sacral: 0.76 (±0.20) / 0.51 (±0.33)

Unknown: 0.22 (±0.02) / 0.09 (±0.04)