Table 3.
Characteristics of studies identified in the systematic literature review
| Publication | Study design | Study location | Method of elicitation | Valuation methoda | Population for whom utility is being measured | Respondent | Sample size and patient demographics |
|---|---|---|---|---|---|---|---|
| Belter et al. [51] | Survey | Global | HUI3 | NR | Patients with SMA |
Patients with SMA (aged ≥ 18 years) Proxy (parent/caregiver) for pts aged < 18 years |
Responses represented n = 478 patients with SMA: Type 1 (n = 121), Type 2 (n = 225), Type 3 (n = 132) Female (n = 284), male (n = 194) Mean ± SD age 17.1 ± 16.8 years |
| Binz et al. [52] | Prospective longitudinal study | Germany | EQ-5D-5L | Value set for EQ-5D-5L based on preferences of German population [92] | Adult patients with SMA | Adult patients with SMA (aged ≥ 18 years) | n = 18 patients with SMA: Type 2 (n = 6), Type 3 (n = 11), Type 4 (n = 1) |
| Chambers et al. [53] | Cross-sectional study | Australia | EQ-5D-Y | EQ-5D-3L Australian value sets used as a proxy as EQ-5D-Y value set unavailable | Patients with Type 1, 2 or 3 SMA | Patients with SMA (aged 0–21 years) and caregiver pairs |
n = 40 patientt–caregiver pairs Children with SMA: Type 1 (n = 4), Type 2 (n = 26), Type 3 (n = 10). Female (n = 21), male (n = 19). Mean age 9.5 years (range 1–23) |
| Lloyd et al. [46] | Cross-sectional survey | UK | Clinical experts assessed Types 1 and 2 SMA case studies using EQ-5D-Y and PedsQL-NMM (baseline states only) | EQ-5D-Y data were scored using UK EQ-5D-3L tariff | Patients with SMA | Clinical experts in SMA | n = 5. No additional information on patient demographics |
| Lo et al. [57] | Survey | UK |
DCE Caregiver HRQoL and disutilities: Preference-based measure: EQ-5D-5L (caregiver HRQoL and disutilities) |
DCE choice data analysed by a conditional logit model EQ-5D-5L utility scores were calculated using EQ-5D-5L crosswalk index values [64] |
Patients with Type 2 SMA and non-ambulatory Type 3 SMA and their caregivers |
Adult patients with SMA and caregivers as proxy for paediatric patients with SMA Caregiver HRQoL: self-reporting by caregivers |
Patients (n = 84), caregivers (n = 83) Sex: Adult patient survey: female (n = 61), male (n = 23) Sex of patient of caregiver survey respondents: female (n = 8), male (n = 75) Mean ± SD age: Adult patient survey respondents: 33.9 ± 11.5 years Age of patient of caregiver survey respondents: 8.4 ± 3.4 years |
| Lo et al. [56] | Survey | UK | DCE | UK societal preferences (trade-off) | Patients with SMA | UK general public aged ≥ 18 years |
n = 506 members of UK general public Female (n = 258), male (n = 248) Mean ± SD age 49 ± 17 years (range 18–82) |
| López-Bastida et al. [47] | Cross-sectional, retrospective study | Spain |
EQ-5D-3L (parents/caregivers as proxies) EQ-5D-5L (caregivers self-report) |
NR | Patients with SMA and their caregivers | Caregivers completed EQ-5D-3L on behalf of patients and self-completed EQ-5D-5L |
n = 81 patient–caregiver pairs Children with SMA Type 1 (n = 8), Type 2 (n = 60), Type 3 (n = 13) Sex of patients: female (n = 47), male (n = 34) Mean ± SD patient age 7.22 ± 5.47 years |
| Love et al. [58] | Cross-sectional survey | Canada | HUI3b | NR | Patients with Types 1, 2, and 3 SMA aged 0–18 years | Patients with SMA (aged 13–18 years) and caregivers of patients with SMA (aged 0–18 years) | n = 14 patients, n = 46 parents. No additional information on patient demographics |
| Malone et al. [48] | NA | Global | PedsQL data from CHERISH mapped to EQ-5D-Y using a published algorithm [36] | NA | Patients with Type 1 SMA | CHERISH trial participants were aged 2–12 years | NR |
| McMillan et al. [54] | Survey | Canada | EQ-5D-5L | NR | Patients with SMA and their caregivers | Surveys were self-completed by adult patients with SMA or proxy completed by caregivers (children with SMA or adults with SMA needing assistance) |
n = 965 patient and n = 962 caregiver responses met eligibility criteria Patient survey respondents with SMA: Type 1 (n = 241), Type 2 (n = 399), Type 3 (n = 283) Patients of caregiver survey respondents: Type 1 (n = 285), Type 2 (n = 423), Type 3 (n = 241), unknown (n = 9) Median age: Patient survey respondents: 8.50 (IQR 2.75–23.50) years Age of patient of caregiver survey respondents: 6.25 (IQR 2.00–11.42) years Sex: Patient survey respondents: female (n = 387), male (n = 576); non-respondents (n ≤ 5) Patients of caregiver survey respondents: female (n = 551), male (n = 406); non-respondents (n = 5) |
| Peña-Longobardo et al. [55] | Cross-sectional study | France, Germany, UK |
EQ-5D-3L (patients) EQ-5D-5L (caregivers) |
UK tariff | Patients with Types 1, 2, and 3 SMA and their caregivers | Children/adolescents with SMA and their caregivers |
n = 86 patients with SMA–caregiver pairs Patients with SMA: Type 1 (n = 23), Type 2 (n = 45), Type 3 (n = 18) Sex of patients: female (n = 51), male (n = 35) Mean ± SD patient age: France: 6.19 ± 6.13 years; Germany: 9.52 ± 6.19 years; UK: 5.55 ± 4.79 years |
| Rowell et al. [59] | Survey | UK | EQ-5D-3L (crosswalk) | UK tariff | HRQoL of caregivers of patients with SMA; HRQoL of patients was not measured | HRQoL: caregivers of patients with SMA |
n = 80 caregivers No additional information on patient demographics relevant to caregiver respondents |
| Sampson and Garau [49] | Review reporting data from a cross-sectional study [68] | France, Germany, Spain, UK | EQ-5D-3L | NR | Caregivers of patients with SMA | Caregivers of patients with SMA | NR |
| Thompson et al. [50] | Cross-sectional study | France, Germany, Spain, UK |
Three options: 1. Parent proxy using EQ-5D-3L 2. Case vignette study of physician-rated EQ-5D-5L and PedsQL (motor function health) 3. CHERISH mapped to EQ-5D using a published algorithm (unspecified) |
NR | Infants and young children with SMA |
Three options: 1. Parents of patients with SMA 2. Physicians 3. Parent-proxy assessments of CHERISH trial participants |
NR |
DCE discrete choice experiment, HRQoL health-related quality of life, HUI health utilities index, IQR interquartile range, NA not applicable, NMM neuromuscular module, NR not reported, PedsQL Pediatric Quality of Life Inventory, SD standard deviation, SMA spinal muscular atrophy
aDetails of how country-specific tariffs were derived are presented in Table 1 (question D).
bHUI2 values also reported in publication; HUI3 utility values only were extracted in this systematic literature review