Table 4.
Summary of published proxy-derived mean HSUVs for patients with SMA by type (standard deviation) [standard error] [95% confidence interval]
| Health state | Proxy-derived HSUVs | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Reference | NICE TA588 ERG-preferred valuesa [65] | NICE TA588 ACM1 [65] | NICE TA588 ACM2 [65] | NICE TA588 ACM3 [65] | Lloyd et al. [46] | Lo et al. [56] | López-Bastida et al. [47] | Love et al. [58] | Malone et al. [48] | Sampson and Garau [49] | Thompson et al. [50] |
| Publication description | NICE report | NICE report | NICE report | NICE report | Full-text publication | Abstract and associated poster | Full-text publication | Abstract | Full-text publication | Full-text publication | Abstract and associated poster |
| PBM used | EQ-5D-Y vignette [46, 70] | PedsQL mapped to EQ-5D | EQ-5D-Y vignette [46, 70] | Clinical experts | Clinical experts assessed Types 1 and 2 SMA case studies using EQ-5D-Y and PedsQL-NMM (baseline states only) | DCE survey of UK general population | EQ-5D-3L (caregivers as proxies) | HUI3b | PedsQL data from CHERISH mapped to EQ-5D-Y using a published algorithm [36] | EQ-5D-3L |
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) |
| Overall Types 1–3 SMA | – | – | – | – | – | – | 0.158 (0.44) | 0.31 (0.27) | – | 0.22c | UKd 0.167 |
| Type 1 SMA (early onset) | |||||||||||
| Baseline/overall Type 1 SMA | – | – | – | – | − 0.12 (0.19) | – | – | 0.14 (0.19) | – | – | – |
| Worsening | – | – | – | – | − 0.24 (0.14) | – | – | – | – | – | – |
| Improvement | – | – | – | – | − 0.17 (0.17) | – | – | – | – | – | – |
| None | − 0.240 | 0.733 | − 0.240 | − 0.020 | – | – | – | – | – | – | – |
| Mild | − 0.120 | 0.752 | − 0.120 | 0.100 | – | – | – | – | – | – | – |
| Moderate | − 0.170 | 0.752 | − 0.170 | 0.200 | – | – | – | – | – | – | – |
| Permanent ventilation | – | – | – | – | – | – | – | – | 0.730 | – | – |
| Non-sitting | – | – | – | – | – | – | – | – | 0.756 | – | – |
| Sitting with support | – | – | − 0.040 | 0.400 | – | – | – | – | 0.764e.f | – | – |
| Sitting without support | − 0.040 | 0.780 | – | – | − 0.04 (0.09) | – | – | – | 0.764e.f | – | – |
| Standing with support | 0.040 | 0.807 | 0.040 | 0.650 | – | – | – | – | – | – | – |
| Standing without support | 0.710 | 0.878 | 0.710 | 0.850 | 0.71 (0.14) | – | – | – | – | – | – |
| Walking with support | 0.520 | 0.807 | 0.520 | 0.750 | 0.52 (0.22) | – | – | – | 0.878e,g | – | – |
| Walking without support | 0.710 | 0.878 | 0.710 | 0.850 | 0.71 (0.14) | – | – | – | 0.878e,g | – | – |
| Types 2/3 SMA (late onset) | |||||||||||
| Overall Type 2 SMA | – | – | – | – | – | – | − 0.012 (0.347) | 0.24 (0.12) | – | – | – |
| Overall Type 3 SMA | – | – | – | – | – | – | 0.62 (0.27) | – | – | – | |
| Baseline Type 2 SMA | – | – | – | – | 0.04 (0.10) | – | – | – | – | – | |
| Worsening | – | – | – | – | − 0.13 (0.06) | – | – | – | – | – | 0.730 [0.0132] |
| Stabilisation of baseline function | – | – | – | – | – | – | – | – | – | – | 0.756 [0.0188] |
| Mild improvement | – | – | – | – | 0.04 (0.11) | – | – | – | – | – | 0.716 [0.0174] |
| Moderate improvement | – | – | – | – | 0.10 (0.09) | – | – | – | – | – | 0.764 [0.0142] |
| Permanent ventilation, Type 2 SMA | – | – | – | – | – | – | – | – | – | – | – |
| Cannot sit | – | – | – | – | – | − 0.408 [CI − 0.440 to − 0.337] (cannot sit, disutility) | – | – | – | – | – |
| Sitting with support | – | – | – | – | – | − 0.068 [CI − 0.083 to − 0.053] (sit with assistance, disutility) | – | – | – | – | – |
| Sitting without support | 0.040 | 0.733 | 0.040 | 0.400 | – | − 0.222 [CI − 0.242 to − 0.201] (sit but cannot stand, disutility) | – | – | – | – | – |
| Sits and rolls | 0.040 | 0.752 | 0.040 | 0.450 | – | – | – | – | – | – | – |
| Sits and crawls | 0.100 | 0.780 | 0.100 | 0.500 | – | – | – | – | – | – | – |
| Standing with support | 0.390 | 0.807 | 0.390 | 0.700 | 0.39 (0.29) | –0.068 [CI − 0.083 to − 0.053] (stand with assistance, disutility) | – | – | – | – | 0.807 [0.0182] |
| Standing without support | 0.720 | 0.807 | 0.720 | 0.850 | 0.72 (0.12) | – | – | – | – | – | 0.805 [0.0256] |
| Walking with support | 0.390 | 0.807 | 0.390 | 0.700 | 0.39 (0.29) | − 0.068 [CI − 0.083 to − 0.053] (walk with assistance, disutility) | – | – | – | – | 0.807 [0.0182] |
| Walking without support | 0.720 | 0.878 | 0.720 | 0.850 | 0.72 (0.12) | – | – | – | – | – | 0.878 [0.0297] |
| Loss of ambulation/motor function (with/without assistance) | – | – | – | – | − 0.12 (0.16) | – | – | – | – | – | 0.774 [0.0303] |
| Disutilities (non-motor function specific) | |||||||||||
| Respiratory support (any) | – | – | – | – | − 0.33 (0.27) | – | – | – | – | – | – |
| Respiratory support (< 16 h/day) | – | – | – | – | – | − 0.159 [CI − 0.174 to − 0.143] | – | – | – | – | – |
| Respiratory support (> 16 h/day) | – | – | – | – | – | − 0.304 [CI − 0.328 to − 0.281] | – | – | – | – | – |
| Oral vs. intrathecal drug administration | – | – | – | – | – | − 0.071 [CI − 0.085 to − 0.057] | – | – | – | – | – |
| Treatment reactions, 12 h/4 mo | – | – | – | – | – | − 0.057 [CI − 0.071 to − 0.042] | – | – | – | – | – |
| Treatment reactions, 1–2 days/4 mo | – | – | – | – | – | − 0.060 [CI − 0.078 to − 0.042] | – | – | – | – | – |
| Treatment reactions, 3–4 days/4 mo | – | – | – | – | – | − 0.087 [CI − 0.103 to − 0.071] | – | – | – | – | – |
| Ophthalmological monitoring before/during treatment if symptoms present | – | – | – | – | – | − 0.024 [CI − 0.036 to − 0.012] | – | – | – | – | – |
| Ophthalmological monitoring before/during treatment 2 ×/year for 2 years | – | – | – | – | – | − 0.023 [CI − 0.037 to − 0.009] | – | – | – | – | – |
| SMA after scoliosis surgery | – | – | – | – | − 0.22 (0.22) | – | – | – | – | – | – |
| Gastric/NG tube | – | – | – | – | − 0.17 (0.17) | – | – | – | – | – | – |
| Contraception | – | – | – | – | – | − 0.012 [CI − 0.021 to − 0.002] | – | – | – | – | – |
ACM appraisal committee meeting, CI confidence interval, DCE discrete choice experiment, ERG evidence review group, HSUV health state utility value, HUI2/3 Health Utilities Index Mark 2/3, mo months, NG nasogastric, NICE National Institute for Health and Care Excellence, NMM neuromuscular module, PBM preference-based measure, PedsQL Pediatric Quality of Life Inventory, SD standard deviation, SMA spinal muscular atrophy, TA technology assessment
– Indicates not assessed in publication
aERG-preferred (dis)utility values are from Lloyd et al. [46, 70]
bHUI3 utility values only were extracted into current table; HUI2 values also reported in publication (not extracted)
cData referenced from López-Bastida et al. [68]; however, utility data for SMA are not reported in López-Bastida et al.; therefore, the review by Sampson and Garau is included as a primary source
dValue for UK; mean (SD) values for France, Germany and Spain also reported in publication
eOnly a single health state reported; no differentiation for with and without support
fHealth state functionally equivalent to Type 2 SMA
gHealth state functionally equivalent to Type 3 SMA