Cost-effectiveness of enzyme replacement therapy for Fabry disease

Table 1 Mean health utilities per year by disease state cluster and 95% confidence intervals after boots-trapping

	N*	Mean health utility	95% LCL	95% UCL
Asymptomatic	19	0.874	0.804	0.934
Acroparesthesia/Symptomatic	55	0.762	0.699	0.822
Single complication	18	0.744	0.658	0.821
Multiple complications	5	0.584	0.378	0.790
Total	97	0.772	0.729	0.815

*Patients may contribute to more than one disease state.
Given low patient numbers in the more progressive disease states, results were clustered by four states: asymptomatic, acroparesthesia/symptomatic, single complication, multiple complications. LCL: lower confidence limit. UCL: upper confidence limit.

ISSN: 1750-1172