Estimating health state utilities associated with a rare disease: familial chylomicronemia syndrome (FCS)

Figures & data

Table 1. Sample characteristics.

Characteristics	UK (N = 208)	Canada (N = 100)
Age (Mean, SD)	44.8 (15.23)	47.4 (17.35)
Gender (n, %)
Male	104 (50.0%)	50 (50.0%)
Female	104 (50.0%)	50 (50.0%)
Racial/Ethnic Background (n, %)
White	148 (71.2%)	88 (88.0%)
Mixed	13 (6.3%)	3 (3.0%)
Asian	18 (8.7%)	0 (0.0%)
Black	28 (13.5%)	3 (3.0%)
Other^a	1 (0.5%)	6 (6.0%)
Marital status (n, %)
Single	137 (65.9%)	57 (57.0%)
Married/cohabitating/living with partner	69 (33.2%)	38 (38.0%)
Other^b	2 (1.0%)	5 (5.0%)
Employment status (n, %)
Full-time work	83 (39.9%)	52 (52.0%)
Part-time work	57 (27.4%)	10 (10.0%)
Other^c	68 (32.7%)	38 (38.0%)
Education level (n, %)
University degree	95 (45.7%)	35 (35.0%)
No university degree	113 (54.3%)	65 (65.0%)

^aOther self-reported racial/ethnic background in the UK was Persian (n = 1). Other in Canada included Arab (n = 1), Haitian (n = 1), and unspecified (n = 4).

^bOther self-reported marital status in the UK was engaged (n = 2). Other in Canada included civil union (n = 1), couple (n = 1), and free union (n = 3).

^cOther self-reported employment status in the UK included apprentice (n = 11), business owner (n = 1), care for disabled child (n = 1), disabled (n = 4), ESA (n = 1), retired (n = 22), self-employed (n = 5), student (n = 18), unemployed (n = 14), and 0 h contract (n = 1). Other in Canada included autonomous (n = 1), business owner (n = 2), disabled (n = 1), homemaker (n = 2), retired (n = 25), student (n = 5), and unemployed (n = 2).

Table 2. Health state utilities^a.

	Mean (SD) utilities			Disutility of acute pancreatitis
FCS health states	UK (N = 208)	Canada (N = 100)	Difference between UK and Canada	UK	Canada
10-year health states
A: Low TG, no AP	0.80 (0.21)	0.83 (0.18)	–0.03 (0.20)
B: High TG, no AP	0.60 (0.33)	0.61 (0.33)	–0.02 (0.33)
C: Low TG, previous AP	0.74 (0.23)	0.78 (0.19)	–0.04 (0.22)
D: High TG, previous AP	0.46 (0.42)	0.47 (0.36)	–0.01 (0.40)
1-year health states
Subgroup rating E relative to C	N = 107	N = 48
C without E	0.71 (0.30)	0.80 (0.17)	–0.08 (0.27)
C with E (adding an AP event to health state C)	0.46 (0.40)	0.60 (0.28)	–0.14 (0.37)	–0.25 (0.28)	–0.20 (0.20)
Subgroup rating E relative to D	N = 101	N = 52
D without E	0.53 (0.38)	0.57 (0.35)	–0.04 (0.37)
D with E (adding an AP event to health state D)	0.33 (0.43)	0.40 (0.34)	–0.07 (0.40)	–0.20 (0.23)	–0.17 (0.16)

Abbreviations. AP, Acute pancreatitis; FCS, Familial chylomicronemia syndrome; SD, Standard deviation; TG, Triglycerides.

^aTTO utilities are on a scale anchored to 0 representing dead and 1 representing full health.