Skip to main content

Table 1 Mean health utility by disease state

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

Disease state

N*

Health utility

    
  

UK

95% LCL

95% UCL

NL

95% LCL

95% UCL

Asymptomatic

**

0.93

0.8900

0.9700

0.93

0.8900

0.9700

Symptoms/recovery

17

0.8716

0.8177

0.9225

0.8897

0.8410

0.9349

Splenectomy

4

0.7532

0.6768

0.8215

0.7781

0.6990

0.8626

Bone complication

6

0.8614

0.7530

0.9685

0.8882

0.8027

0.9707

Multiple complications

13

0.7323

0.6601

0.8202

0.7981

0.7430

0.8638

Malignancy

1#

0.15

  

0.364

  
  1. LCL: lower limit of the confidence interval, UCL: upper limit of the confidence interval.
  2. *Patients may contribute to more than one disease state.
  3. **We used a foreign estimate of 0.93 (95% CI: 0.89-0.97) for healthy persons reported by Clarke et al. [16]. This estimate too was based on time trade-off based elicitation techniques.
  4. #The observed estimate for the stage of malignancy was based on just 1 person, but well within the range of values expected for end-stage malignant disease (e.g. Brown et al. [17]).
Back to article page

Orphanet Journal of Rare Diseases

ISSN: 1750-1172