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Abstract: FR-PO0016

Comparison of Causal Machine Learning (ML) Models on Predicting Survival Benefit with Dialysis in Patients with ESKD: A Retrospective Cohort Study

Session Information

Category: Artificial Intelligence, Digital Health, and Data Science

  • 300 Artificial Intelligence, Digital Health, and Data Science

Authors

  • Leung, Ka Chun, Tuen Mun Hospital, Hong Kong, Hong Kong
  • Yap, Yat Hin Desmond, The University of Hong Kong, Hong Kong, Hong Kong
Background

The choice to start dialysis at the bedside in advanced CKD is highly individualized, but average-effect trial evidence (e.g., IDEAL) cannot tell patient-specific treatment benefit. Causal ML methods offer a potential solution for quantifying this heterogeneity from observational data, but clinicians lack clear evidence on which method to trust.

Methods

Eight causal-ML models were trained on a Hong Kong CKD cohort (n = 20184) and validated on a external cohort (n = 6040). Primary outcome: IPCW-weighted mortality at 1 year. Model evaluation included discrimination, calibration, permutation null test and transportability across cohorts.

Results

The permutation null test rejected no informative HTE on every model (p ≤ 0.025) except the IPCW_DML_Pseudo model. All models preserved C-for-benefit 0.53 – 0.55 (risk-proportional baseline 0.513), proving causal ML edge over a clinician's heuristic. Treating the top 30 % by predicted CATE rank recovered 75 % (95 % CI 59 – 98 %) of the treat-all mortality benefit on the external test set, while sparing 70 % of patients from early dialysis.

Conclusion

Causal-ML models produce population-level treatment effects and detect treatment-effect heterogeneity (p < 0.025), while preserve their patient-level ranking across different cohorts. Calibration and transportability are the dimensions that distinguish outstanding models at the bedside.

Per-method summary on external test set 1-year mortality
MethodC-for-benefitΔ vs no-HTE nullCalibration slopeGlobal O:EQ1–Q5 spreadRecovery at K = 30 %Transport ΔC, spatial → external
Stacked ensemble0.554 (0.528-0.564)+0.036 (+0.015-+0.064)0.77 (0.52-0.95)0.560.184 (0.117-0.258)75 (59-98)-0.013 (-0.050-+0.011)
S-learner0.543 (0.519-0.561)+0.028 (+0.009-+0.059)0.62 (0.41-0.82)0.570.218 (0.144-0.296)73 (59-93)-0.025 (-0.050-+0.022)
X-learner0.541 (0.525-0.559)+0.026 (+0.007-+0.059)0.72 (0.50-0.92)0.550.189 (0.116-0.256)73 (59-89)-0.033 (-0.065-+0.005)
IPTW-S-learner0.545 (0.527-0.560)+0.033 (+0.009-+0.060)1.03 (0.70-1.40)0.710.166 (0.106-0.230)69 (54-89)-0.027 (-0.062-+0.002)
Survival-DML-Cox0.540 (0.523-0.558)+0.026 (+0.007-+0.058)0.52 (0.31-0.73)0.640.190 (0.108-0.282)65 (51-85)-0.019 (-0.057-+0.021)
T-learner0.547 (0.524-0.557)+0.038 (+0.008-+0.056)0.47 (0.28-0.64)0.570.158 (0.102-0.227)64 (51-79)-0.016 (-0.064-+0.009)
Kennedy-DR-learner0.533 (0.514-0.548)+0.017 (-0.002-+0.050)0.46 (0.26-0.67)0.550.138 (0.048-0.203)54 (37-68)-0.015 (-0.056-+0.018)
IPCW-DML pseudo-outcome0.506 (0.494-0.531) -0.005 (-0.020-+0.027) 0.23 (0.05-0.40)0.540.095 (0.035-0.180)47 (28-69)-0.049 (-0.086--0.017)

Discrimination per method on external validation

Recovery curves: fraction of treat-all mortality benefit recovered when treating only the top K % of patients by predicted CATE rank