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

Journal of Patient-Reported Outcomes

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

Health State Description	Health Utility
Health State Description	N	Mean	95% CI^a
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
ERT^b 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
ERT^b Treatment, ≥18 yr	169	0.673	0.621–0.723

Disease health states listed ≥18 yr. of age are derived from Frame 1 TTO: Adult health state questions
Disease health states listed for ages < 18 yr. are derived from Frame 2 TTO: Child health state questions
^aBootstrapped
^bEnzyme replacement therapy