Table 1 Parameters Table
Parameter | Mean (95% Confidence Interval) | Reference |
|---|---|---|
Base case parameters | ||
Number of births per year | 400,308 | |
X-ALD incidence | 1 in 22,361 (15,083, 33,153) | |
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 | ᅟ ᅟ −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 | Â |
Specificity | 1 | Â |
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 | Â |