Table 5.
Summary of published patient-derived and mixed patient–proxy-derived mean HSUVs for patients by SMA type (standard deviation)
| Health state | Patient-derived HSUVs | Mixed patient–proxy-derived HSUVs | ||||
|---|---|---|---|---|---|---|
| Reference | Binz et al. [52] | Love et al.a [58] | Peña-Longobardo et al. [55] | Belter et al. [51] | Chambers et al. [53] | McMillan et al. [54] |
| Publication type | Full-text publication | Abstract | Full-text publication | Full-text publication | Full-text publication | Full-text publication |
| PBM used | EQ-5D-5L | HUI3 | EQ-5D-3L and EQ-5D-5L | HUI3 | EQ-5D-Y | EQ-5D-5L |
| Overall Types 1–3 SMA | 0.46 (0.37)b | 0.33 (0.28) | 0.167 (0.277)c | – | 0.115 (0.227) | 0.49 (0.26) |
| Baseline/overall Type 1 SMA | – | 0.29 (0.36) | – | – | 0.104 (0.278) | 0.32 |
| Worsening | – | – | – | – | – | – |
| Improvement | – | – | – | – | – | – |
| None | – | – | – | – | – | – |
| Mild | – | – | – | – | – | – |
| Moderate | – | – | – | – | – | – |
| Permanent ventilation | – | – | – | − 0.05 (0.10) | – | – |
| Non-sitting | – | – | – | 0.06 (0.10) | – | – |
| Sitting (with support) | – | – | – | 0.11 (0.21)d | – | – |
| Sitting without support | – | – | – | – | – | – |
| Standing (with support) | – | – | – | – | – | – |
| Standing without support | – | – | – | – | – | – |
| Walking (with support) | – | – | – | – | – | – |
| Walking without support | – | – | – | – | – | – |
| Overall Type 2 SMA | – | 0.23 (0.16) | – | – | 0.067 (0.158) | 0.46 |
| Overall Type 3 SMA | – | 0.41 (0.02) | – | – | 0.252 (0.332) | 0.65 |
| Baseline | – | – | – | – | – | – |
| Worsening | – | – | – | – | – | – |
| Stabilisation of baseline function | – | – | – | – | – | – |
| Mild improvement | – | – | – | – | – | – |
| Moderate improvement | – | – | – | – | – | – |
| Permanent ventilation, Type 2 SMA | – | – | – | 0.10 (0.11) | – | – |
| Non-sitting | – | – | – |
Type 2 SMA: 0.12 (0.12) Type 3 SMA: 0.14 (0.13) |
– | – |
| Sitting (with support) | – | – | – |
Type 2 SMA: 0.26 (0.16)d Type 3 SMA: 0.23 (0.11)d |
– | – |
| Sitting without support | – | – | – | – | – | |
| Standing (with support) | – | – | – | – | – | – |
| Standing without support | – | – | – | – | – | – |
| Walking (with support) | – | – | – |
Type 2 SMA: 0.44 (0.12) Type 3 SMA: 0.35 (0.21) |
– | – |
| Walking without support | – | – | – |
Type 2 SMA: 0.58 (0.15) Type 3 SMA: 0.64 (0.24) |
– | – |
| Loss of ambulation/motor function (with/without assistance) | – | – | – | – | – | – |
| Disutilities | NR | NR | NR | NR | NR | NR |
| Contraception | – | – | – | – | – | – |
HSUV health state utility value, HUI2/3 Health Utilities Index Mark 2/3, NR not reported, PBM preference-based measure, SMA spinal muscular atrophy
– Indicates not assessed in publication
aHUI3 utility values only were extracted into current table; HUI2 values also reported in publication (not extracted)
bStudy sample included patients with the following SMA types: Type 2 (n = 6); Type 3 (n = 11); and Type 4 (n = 1)
cValue for UK; mean (standard deviation) values for France and Germany also reported
dOnly a single health state reported; no differentiation for with and without support