Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease

Table 5 Mean yearly numbers and costs of out-of-hospital consultations^§ by disease state

Disease state*	Volumes	95% LCL	95% UCL	Costs in €	95% LCL	95% UCL
(No) signs/symptoms (N = 10)**	3.6	1.1	7.8	121	31	272
Recovery (N = 9)	16.8	6.3	28.3	641	229	1,097
Splenectomy (N = 4)	6.0	0.3	11.6	299	8	589
Bone complication (N = 6)	12.7	1.6	26.4	449	51	939
Multiple complications (N = 13)	6.8	2.9	11.2	245	97	421
Malignancy (N = 1)	2			56

LCL: lower limit of the confidence interval, UCL: upper limit of the confidence interval.
*Patients may contribute to more than one disease state. **Data from two asymptomatic patients have been added to data from eight symptomatic patients to improve precision; the estimates were applied to both, the asymptomatic as well as symptomatic Markov disease state. § Patients in the asymptomatic and symptomatic disease states reported visiting the general physician, physiotherapist, company physician, and alternative healer. Patients in recovery reported visiting the general physician, physiotherapist, social worker, and alternative healer. Patients with a splenectomy reported visiting the general physician, social worker, and alternative healer. Patients with a bone complication reported visiting the general physician, physiotherapist, and company physician. Patients with multiple complications reported visiting the general physician, physiotherapist, psychologist/psychiatrist, company physician, social worker, and alternative healer. The patient with a malignancy reported visiting the general physician.

ISSN: 1750-1172