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Table 2 Utility values of patients treated with cerliponase alfa using the UK EQ-5D-5L value set

From: Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Disease stage Cerliponase alfa
Mean value Standard error Median value Minimum value Maximum value
6 0.990 0.010 1.000 0.924 1.000
5 0.850 0.008 0.846 0.825 0.901
4 0.745 0.019 0.761 0.642 0.801
3 0.502 0.061 0.539 0.302 0.666
2 0.474 0.060 0.436 0.267 0.666
1 0.338 0.053 0.317 0.167 0.652
0 0.129 0.057 0.179  − 0.213 0.282
0 + VL 0.119 0.065 0.186  − 0.281 0.282
0 + VL + PC 0.104 0.065 0.174  − 0.281 0.268
  1. Disease progression increases with decreasing disease stage. Utility values are given on a scale where 1 is equivalent to perfect health, and 0 equivalent to death. UK value set
  2. PC, palliative care; VL, vision loss