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Table 1 Parameters Table

From: Economic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme

Parameter Mean (95% Confidence Interval) Reference
Base case parameters
Number of births per year 400,308 [22,23,24]
X-ALD incidence 1 in 22,361 (15,083, 33,153) [3, 29,30,31,32]
Proportion
CCALD, AMN, Addison’s/Asymptomatic
0.53 (0.36, 0.69), 0.32 (0.18, 0.49), 0.15 (0.05, 0.28) [14]
Non-X-ALD peroxisomal incidence 1 in 63,000 (33,897, 117,090) [32]
Age at presentation CCALD 7 (6.76, 7.24) [11]
Survival from onset CCALD
Weibull distribution
- shape parameter
- scale parameter
- correlation


−2.970
0.162
−0.8994
[11]
Time to CCALD progression (years) 1.6 (1.34, 1.86) [11]
Mortality risk HSCT 0.08 (0.01, 0.21) [10]
Proportion of CCALD currently undergoing early transplant (Family history) 0.33 (0.23, 0.43) [10]
Proportion ALD-DRS 0, ALD-DRS1, ALD-DRS2, ALD-DRS 3–4, after HSCT 0.62 (0.35, 0.85), 0.23 (0.05, 0.48), 0.08 (0.002, 0.26), 0.08 (0.002, 0.26) [10]
Proportion successful HSCT develop AMN 0.6 (0.19, 0.93) [65]
Sensitivity 0.995  
Specificity 1  
Proportion of AMN mild 0.51 (0.38, 0.64) [44]
Proportion of AMN developing adult onset cerebral X-ALD 0.63 (0.44, 0.8) [40]
Age at presentation AMN (years) 35.3 (26.7, 43.9) [40]
Time to development of adult onset cerebral X-ALD (years) 10.2 (3.3, 17.1) [40]
Survival adult onset cerebral X-ALD (years) 3.4 (0.5, 6.3) [40]
QALYs See Additional file 2  
Costs See Additional file 3