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Table 2 Health utilities derived from a community sample by disease condition and age of patient

From: Health utilities and parental quality of life effects for three rare conditions tested in newborns

Health State Description Health Utility
N Mean 95% CIa
Krabbe Disease
 Early Stage Illness, 6 months 166 0.469 0.409–0.532
 Advanced Stage Illness, 6 months 167 0.374 0.316–0.430
 Early Stage Illness, 8 years 168 0.548 0.490–0.606
 Advanced Stage Illness,8 yr 168 0.440 0.380–0.500
 Early Stage Illness,≥18 yr 167 0.806 0.757–0.847
 Advanced Stage Illness,≥18 yr 167 0.554 0.497–0.612
Phenylketonuria
 Less Adherent to Diet, 8 yr 170 0.564 0.506–0.623
 More Adherent to Diet,8 yr 171 0.639 0.581–0.696
 Less Adherent to Diet, ≥18 yr 171 0.679 0.628–0.730
 More Adherent to Diet,≥18 yr 171 0.808 0.762–0.852
Pompe Disease
 Severe Symptoms, 6 months 170 0.399 0.341–0.457
 Mild Symptoms, 8 yr 171 0.799 0.750–0.844
 Moderate Symptoms, 8 yr 169 0.414 0.355–0.475
 Severe Symptoms, 8 yr 169 0.466 0.407–0.525
 ERTb Treatment, 8 yr 170 0.475 0.417–0.534
 Mild Symptoms, ≥18 yr 170 0.853 0.811–0.892
 Moderate Symptoms, ≥18 yr 170 0.683 0.634–0.729
 Severe Symptoms, ≥18 yr 171 0.536 0.480–0.594
 ERTb Treatment, ≥18 yr 169 0.673 0.621–0.723
  1. Disease health states listed ≥18 yr. of age are derived from Frame 1 TTO: Adult health state questions
  2. Disease health states listed for ages < 18 yr. are derived from Frame 2 TTO: Child health state questions
  3. aBootstrapped
  4. bEnzyme replacement therapy