Table 2.
Mean point estimates, based on 2000 simulated datasets generated under the ‘baseline’ scenario with n= 5000, for the proposed finite interval illness-death model with and without frailty (FI-IDγ, FI-ID); the cure fraction illness-death model of Conlon et al.31 with and without a frailty (Conlonγ, Conlon); the illness-death model with frailty (IDγ); the cure fraction model for T1; and a multi-state illness-death model (MSM), which aligns with the results of a univariate Cox model for acute GVHD on the 2 → 3 transition.
| Truth | FI-IDγ | FI-ID | Conlonγ | Conlon | IDγ | Cure | MSM | |
|---|---|---|---|---|---|---|---|---|
| Non-susceptible fraction | ||||||||
| βs,0 | −0.41 | −0.41 | −0.31 | −0.55 | −0.40 | 0.14 | ||
| βs,x | 0.50 | 0.50 | 0.51 | 0.53 | 0.54 | 0.51 | ||
| Non-susceptible: 1 → 4 | ||||||||
| log(κ14) | −8.52 | −8.51 | −7.06 | −9.06 | −7.41 | |||
| log(α14) | 0.34 | 0.33 | 0.14 | 0.38 | 0.18 | |||
| β14,x | 0.25 | 0.25 | 0.20 | 0.33 | 0.23 | |||
| Susceptible: 2 → 4 | ||||||||
| log(κ24) | −5.30 | −5.30 | −5.17 | −5.27 | −5.09 | −5.35 | ||
| log(α24) | 0.34 | 0.34 | 0.23 | 0.30 | 0.25 | 0.08 | ||
| β24,x | 0.50 | 0.50 | 0.41 | 0.52 | 0.45 | −0.08 | 0.08 | |
| Susceptible: 2 → 3 | ||||||||
| log(κ23) | −2.16 | −2.17 | −2.31 | −1.81 | −1.77 | −1.98 | ||
| log(α23) | −0.69 | −0.69 | −0.75 | −0.63 | −0.64 | −1.10 | ||
| β23,x | 0.25 | 0.25 | 0.25 | 0.30 | 0.25 | −0.31 | 0.38 | −0.26 |
| Susceptible: 3 → 4 | ||||||||
| log(κ34) | −6.21 | −6.22 | −5.66 | −6.20 | −5.66 | −7.01 | ||
| log(α34) | 0.26 | 0.26 | 0.15 | 0.26 | 0.15 | 0.37 | ||
| β34,x | 0.15 | 0.15 | 0.14 | 0.16 | 0.14 | 0.11 | 0.14 | |
| log-frailty variance | ||||||||
| log(θ) | −1.71 | −1.73 | −2.00 | −0.49 | ||||
Note: See Section 5 for details.