Table 5 Summary of utility outcomes for included studies
| Study reference | Condition(s) of interest | Respondent type | PBM (s) | Other relevant outcome measures | Overall utility scores (mean ± SD) | Sub-group utility scores (mean ± SD) |
|---|---|---|---|---|---|---|
| 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) |