Table 2 Cohort Definitions
Cohort | Model Input | Value/Definition | Assumption |
|---|---|---|---|
Poor Compliance | Mean age-at-KF (years) | 15.40 | Reported value in Nesterova, et al. (2015) [13] |
Composite Compliance Score | 21.93 | Calculated using Nesterova’s equation [13]: 𝑦 = 0.30𝑥 + 8.82. Where \(y = 15.40\) | |
Treatment duration prior to KF (years) | 13.40 | Assumption: equal to the mean age-at-KF minus 2 years | |
Annual Compliance Score | 1.62 | Calculated based on treatment duration and CCS: 21.93 ÷ 13.40 = 1.62 | |
Mean age-at-death (years) | 48.77 | Derived from survival analysis applied to Kaplan-Meier mortality data provided in Brodin-Sartorius et al. (2012) [8]; validated by clinical experts | |
Good Compliance | Mean age-at-KF (years) | 23.00 | Assumption: based on clinical expert guidance |
Composite Compliance Score | 47.27 | Calculated using Nesterova’s equation [13]: 𝑦 = 0.30𝑥 + 8.82. Where \(y = 23.00\) | |
Treatment duration prior to KF (years) | 21.00 | Assumption: equal to the mean age-at-KF minus 2 years | |
Annual compliance score | 2.25 | Calculated based on treatment duration and CCS: 47.27 ÷ 21.00 = 2.25 | |
Mean age-at-death (years) | 56.37 | Calculated by assuming the difference in mean age-at-KF between both cohorts is equal to the difference in mean age-at-death in both cohorts; validated by clinical experts |