Bayesian Causal Forests for Zero-Inflated Continuous Outcomes
Organizations: University of São Paulo, Brazil. · Columbia University Irving Medical Center.
Abstract
Estimating (heterogeneous) treatment effects is still challenging when the outcome is semicontinuous (i.e., exhibiting both a discrete point mass at zero and a skewed continuous distribution for positive values), despite the strong presence of this data type in real world settings. Existing methods either ignore the zero mass, impose fragile parametric assumptions, or suffer from extreme computational costs and data augmentation noise. This paper proposes Zero-Inflated Continuous Bayesian Causal Forests with Duan's Smearing (ZIC-BCF-Smear), a decoupled two-part nonparametric estimator that fits a Probit BCF on the full sample to model participation and a Gaussian BCF strictly on the active subpopulation to model positive intensity. Subpopulation Propensity Adjustment (SPA) mitigates regularization-induced confounding within the active subpopulation, and Duan's non-parametric smearing estimator re-transforms predictions to the response scale, bypassing the fragile scale multipliers of parametric models. A comprehensive simulation benchmark shows that ZIC-BCF-Smear matches well-specified parametric models without their assumptions and remains stable and calibrated where competitors either diverge or pathologically over-cover for nonlinear data. The method is then applied to individual-participant data from the SPECTRUM randomized phase III head-and-neck cancer trial, estimating the causal effect of assignment to panitumumab plus chemotherapy on the number of days patients spend with severe toxicity, an outcome for which a quarter of patients contribute an exact zero. Panitumumab assignment raises that burden by about ten days per patient under the trial's own differential treatment duration, and the decoupled structure localizes the effect mainly in the positive-intensity part rather than in whether the reconstructed severe-AE burden is positive.
Figures & tables
| Feature | BCF-Linear | DPglm | EDP+ZI | Gamma Hurdle | Gamma + .01 | ZIC-BCF-Smear |
|---|---|---|---|---|---|---|
| Separates Zero-Inflation | No | Yes | Yes | Yes | No | Yes |
| Hurdle CATE Estimation | No | Yes | Yes | Yes | No | Yes |
| MCMC Augmentation Noise | None | Extreme | Extreme | None | None | None |
| Subpopulation Confounding | N/A | Adjusted | Adjusted | Adjusted | N/A | Adjusted (SPA) |
| Scale Re-transformation | None | Parametric | Parametric | Parametric | Parametric | Non-Parametric |
| DGP | Model | CATE RMSE | CATE Cov | CATE Corr | CATE CI Len | ATE RMSE | ATE Abs Bias |
|---|---|---|---|---|---|---|---|
| DGP A: Log-Normal | BCF-Linear | 3.662 | 93.2% | 0.184 | 6.315 | 0.849 | 0.282 |
| DPglm | 1.409 | 100.0% | 0.321 | 51.210 | 0.781 | 0.637 | |
| Gamma Hurdle | 1.810 | 94.9% | 0.419 | 4.641 | 0.585 | 0.029 | |
| Gamma +.01 | 2.946 | 91.7% | 0.267 | 6.215 | 0.765 | 0.212 | |
| ZIC-BCF-Smear | 1.526 | 94.9% | 0.397 | 3.819 | 0.614 | 0.406 | |
| DGP B: Gamma | BCF-Linear | 4.010 | 92.5% | 0.156 | 6.489 | 0.812 | 0.253 |
| DGP | Model | CATE RMSE | CATE Cov | CATE Corr | CATE CI Len | ATE RMSE | ATE Abs Bias |
|---|---|---|---|---|---|---|---|
| DGP A: Log-Normal | BCF-Linear | 5.812 | 88.1% | 0.347 | 6.385 | 1.127 | 0.172 |
| DPglm | 3.070 | 99.7% | 0.198 | 61.532 | 0.680 | 0.021 | |
| Gamma Hurdle | 69.2% | -0.039 | |||||
| Gamma +.01 | 69.5% | -0.081 | |||||
| ZIC-BCF-Smear | 1.734 | 89.7% | 0.686 | 4.422 | 0.537 | 0.119 | |
| DGP B: Gamma | BCF-Linear | 5.792 | 89.1% | 0.318 | 6.624 | 1.101 | 0.217 |
| Link-Linear | Link-Nonlinear | ||||
|---|---|---|---|---|---|
| DGP | Model | Hurdle RMSE | Coverage | Hurdle RMSE | Coverage |
| DGP A: Log-Normal | DPglm | 0.053 | 99.7% | 0.077 | 98.6% |
| Gamma Hurdle | 0.098 | 94.0% | 0.129 | 88.6% | |
| ZIC-BCF-Smear | 0.046 | 99.3% | 0.065 | 94.7% | |
| DGP B: Gamma | DPglm | 0.055 | 99.7% | 0.077 | 98.2% |
| Gamma Hurdle | 0.098 | 94.0% | 0.129 | 88.5% | |
| Estimand | Model | Estimate | 95% Credible Interval |
|---|---|---|---|
| Summed record duration (event-days) | ZIC-BCF-Smear | 14.27 | [4.69, 24.23] |
| Gamma Hurdle | 15.95 | [6.15, 26.30] | |
| Distinct calendar days | ZIC-BCF-Smear | 10.00 | [3.57, 16.62] |
| Gamma Hurdle | 12.20 | [5.56, 19.23] | |
| Participation margin (probability) | ZIC-BCF-Smear | 0.029 | [ , 0.102] |
| Gamma Hurdle | 0.017 | [ , 0.089] |
| Window | Chemotherapy | Panitumumab | Difference |
|---|---|---|---|
| Last contact capped | 19.18 | 30.90 | 11.73 |
| Day 84, all patients | 11.25 | 14.33 | 3.08 |
| Day 84, observed to day 84 | 11.25 | 14.93 | 3.68 |
| Day 120, all patients | 15.55 | 19.58 | 4.03 |
| Day 120, observed to day 120 | 16.12 | 21.25 | 5.13 |
| DGPs A & B (Hurdle) | DGP C (Tweedie) | ||||
|---|---|---|---|---|---|
| Configuration | Target Zeros | Link-Linear | Nonlinear | Link-Linear | Nonlinear |
| Level 1 (Severe zero-inflation) | |||||
| Level 2 (High zero-inflation) | |||||
| Level 3 (Standard configuration) | |||||
| Level 4 (Moderate zero-inflation) | |||||
| Level 5 (Low zero-inflation) | |||||
| Data-generating process | Monitored set | Median | 95th pct. | Max | Median ESS | Min ESS | Max |
|---|---|---|---|---|---|---|---|
| Linear DGP A (log-normal hurdle) | Global summaries | 1.004 | 1.014 | 1.017 | 928 | 449 | 4.05 |
| Unit-level CATEs | 1.005 | 1.012 | 1.048 | 1,125 | 508 | 3.68 | |
| Linear DGP B (Gamma hurdle) | Global summaries | 1.002 | 1.007 | 1.010 | 1,124 | 444 | 2.57 |
| Unit-level CATEs | 1.003 | 1.010 | 1.022 | 1,563 | 693 | 3.53 | |
| Linear DGP C (Tweedie) | Global summaries | 1.004 | 1.038 | 1.118 | 1,113 | 203 | 4.08 |
| Unit-level CATEs | 1.004 | 1.011 | 1.030 | 1,489 | 869 | 4.91 |
| Data-generating process | Median BP | Rejected at | Residual-scale ratio | Median relative change | Max relative change |
|---|---|---|---|---|---|
| Linear DGP A (log-normal hurdle) | 0.194 | 1 of 5 | 1.23 | ||
| Linear DGP B (Gamma hurdle) | 0.026 | 3 of 5 | 1.21 | ||
| Linear DGP C (Tweedie) | 5 of 5 | 2.12 | |||
| Nonlinear DGP A (log-normal hurdle) | 0.313 | 0 of 5 | 1.21 | ||
| Nonlinear DGP B (Gamma hurdle) | 0.242 | 1 of 5 | 1.27 | ||
| Nonlinear DGP C (Tweedie) | 5 of 5 | 1.54 |
| DGP | Model | Diverged Seeds | CATE RMSE (median) | ATE Abs Err (median) | CATE Corr |
|---|---|---|---|---|---|
| DGP A: Log-Normal | Gamma Hurdle | 4 / 100 | 3.960 | 0.759 | -0.039 |
| Gamma +.01 | 5 / 100 | 4.336 | 0.747 | -0.081 | |
| ZIC-BCF-Smear | 0 / 100 | 1.611 | 0.352 | 0.686 | |
| DGP B: Gamma | Gamma Hurdle | 5 / 100 | 3.704 | 0.784 | -0.040 |
| Gamma +.01 | 9 / 100 | 4.145 | 0.692 | -0.068 | |
| ZIC-BCF-Smear | 0 / 100 | 1.537 | 0.494 | 0.645 |
| DGP | Model | Parameter | ZI 85% (Level 1) | ||||
|---|---|---|---|---|---|---|---|
| DGP A: Log-Normal | DPglm | RMSE | 0.086 | 0.067 | 0.053 | 0.057 | 0.050 |
| Coverage | 98.0% | 99.5% | 99.7% | 99.7% | 86.6% | ||
| Gamma Hurdle | RMSE | 0.208 | 0.129 | 0.098 | 0.071 | 0.080 | |
| Coverage | 95.1% | 95.8% | 94.0% | 94.8% | 95.1% | ||
| ZIC-BCF-Smear | RMSE | 0.068 | 0.054 | 0.046 | 0.040 | 0.036 | |
| Coverage | 98.9% | 99.0% | 99.3% | 98.6% | 98.3% |
| DGP | Model | Parameter | ZI 85% (Level 1) | ||||
|---|---|---|---|---|---|---|---|
| DGP A: Log-Normal | DPglm | RMSE | 0.105 | 0.085 | 0.077 | 0.078 | 0.078 |
| Coverage | 98.2% | 98.8% | 98.6% | 96.1% | 96.9% | ||
| Gamma Hurdle | RMSE | 0.229 | 0.167 | 0.129 | 0.111 | 0.104 | |
| Coverage | 92.6% | 89.9% | 88.6% | 82.2% | 85.9% | ||
| ZIC-BCF-Smear | RMSE | 0.083 | 0.074 | 0.065 | 0.062 | 0.065 | |
| Coverage | 98.0% | 96.4% | 94.7% | 90.8% | 92.8% |
| Arm | Zero (%) | Mean | Positive mean | Positive median | Maximum | |
|---|---|---|---|---|---|---|
| Chemotherapy | 260 | 24.6 | 26.75 | 35.49 | 20.5 | 263 |
| Panitumumab + chemotherapy | 260 | 22.7 | 42.43 | 54.89 | 36.0 | 641 |
| Endpoint | Total days | Chemotherapy mean | Panitumumab + chemotherapy mean | Unadjusted difference | Zeros |
|---|---|---|---|---|---|
| Summed AE-record days | 17,988 | 26.75 | 42.43 | 15.68 | 123 |
| Distinct severe-AE calendar days | 13,107 | 19.18 | 31.23 | 12.06 | 123 |
| Arm | Mean | SD | Median | Maximum | |
|---|---|---|---|---|---|
| Chemotherapy | 260 | 83.72 | 50.14 | 89.0 | 276 |
| Panitumumab plus chemotherapy | 260 | 130.30 | 127.19 | 112.5 | 1,149 |
| Date variant and horizon | Chemotherapy | Panitumumab | Difference |
| Imputed start, imputed end (primary) | |||
| Uncapped | 19.18 | 31.23 | |
| 120 days | 15.55 | 19.65 | |
| 90 days | 11.96 | 15.33 | |
| 84 days | 11.25 | 14.39 | |
| Raw start, raw end | |||
| Arm | Total treatment days | Total severe-AE days | Patient-level mean rate | Aggregate rate |
|---|---|---|---|---|
| Chemotherapy | 21,766 | 4,986 | 53.72 | 22.91 |
| Panitumumab plus chemotherapy | 33,877 | 8,121 | 49.91 | 23.97 |
| Arm | Completed safety follow-up | Mean DSDY | Mean LASTCTDY | LASTCTDY | LASTCTDY | |
|---|---|---|---|---|---|---|
| Chemotherapy | 260 | 181 (69.6%) | 110.7 | 376.8 | 47 | 213 |
| Panitumumab plus chemotherapy | 260 | 173 (66.5%) | 159.5 | 403.5 | 41 | 219 |
| Model and estimand | Estimate | 95% credible interval | |
|---|---|---|---|
| ZIC-BCF-Smear, summed duration (event-days) | 14.27 | [4.69, 24.23] | 0.999 |
| Gamma Hurdle, summed duration (event-days) | 15.95 | [6.15, 26.30] | 0.999 |
| Unadjusted arm difference, summed duration | 15.68 | – | – |
| ZIC-BCF-Smear, distinct calendar days | 10.00 | [3.57, 16.62] | 0.999 |
| Gamma Hurdle, distinct calendar days | 12.20 | [5.56, 19.23] | 1.000 |
| Unadjusted arm difference, distinct calendar days | 12.06 | – | – |
| Covariate | Level | CATE | 95% credible interval | ||
|---|---|---|---|---|---|
| Overall | All | 520 | 14.27 | [4.69, 24.23] | 0.999 |
| Sex | Male | 455 | 14.20 | [4.53, 24.37] | 0.999 |
| Sex | Female | 65 | 14.76 | [2.28, 29.89] | 0.990 |
| ECOG status | ECOG 0 | 150 | 18.74 | [5.76, 35.51] | 1.000 |
| ECOG status | ECOG 1 | 370 | 12.46 | [3.27, 22.47] | 0.996 |
| Diagnosis site | Hypopharynx | 75 | 13.37 | [3.56, 24.47] | 0.997 |
| Covariate | Level | CATE | 95% credible interval | ||
|---|---|---|---|---|---|
| Overall | All | 520 | 10.00 | [3.57, 16.62] | 0.999 |
| Sex | Male | 455 | 10.12 | [3.55, 17.01] | 0.999 |
| Sex | Female | 65 | 9.10 | [0.40, 18.63] | 0.980 |
| ECOG status | ECOG 0 | 150 | 14.76 | [4.86, 28.25] | 1.000 |
| ECOG status | ECOG 1 | 370 | 8.07 | [1.85, 14.58] | 0.994 |
| Diagnosis site | Hypopharynx | 75 | 9.55 | [2.65, 17.22] | 0.997 |
| Domain | Scalar CATE ($) | Locally smeared | Local 95% CrI | Change | |
|---|---|---|---|---|---|
| Age group : 35-49 | 3468 | 175.27 | 183.95 | [150.47, 218.70] | 5.0% |
| Age group : 18-34 | 3352 | 169.06 | 176.81 | [142.45, 214.46] | 4.6% |
| Family income (weighted tertile) : High-income tertile | 5330 | 188.25 | 196.54 | [149.30, 242.36] | 4.4% |
| Marital status (detailed) : Never married | 4579 | 191.74 | 199.91 | [160.15, 246.77] | 4.3% |
| Region : Midwest | 3837 | 195.04 | 202.46 | [153.53, 259.09] | 3.8% |
| Poverty cat. : High-income | 7351 | 206.16 | 213.89 | [164.10, 260.06] | 3.7% |
| Covariate | Subgroup | Analytic-sample average | Population target | |
|---|---|---|---|---|
| Overall | All | 18,763 | 188.02 [156.95, 220.67] | 184.67 [154.63, 214.70] |
| Sex | Male | 8,928 | 185.86 [153.06, 220.98] | 182.56 [149.75, 215.38] |
| Sex | Female | 9,835 | 189.99 [149.59, 230.49] | 186.70 [148.40, 225.00] |
| Race and ethnicity | Hispanic | 4,165 | 144.82 [113.97, 178.04] | 146.57 [114.08, 179.06] |
| Race and ethnicity | White, non-Hispanic | 10,243 | 218.16 [176.61, 260.39] | 208.95 [170.75, 247.15] |
| Race and ethnicity | Black, non-Hispanic | 2,495 | 138.37 [84.47, 192.74] | 137.53 [84.85, 190.21] |
| Covariate | Subgroup | Population target | |
|---|---|---|---|
| Overall | All | 18,763 | 0.139 [0.123, 0.155] |
| Sex | Male | 8,928 | 0.143 [0.121, 0.165] |
| Sex | Female | 9,835 | 0.136 [0.113, 0.158] |
| Race and ethnicity | Hispanic | 4,165 | 0.129 [0.103, 0.155] |
| Race and ethnicity | White, non-Hispanic | 10,243 | 0.151 [0.130, 0.171] |
| Race and ethnicity | Black, non-Hispanic | 2,495 | 0.109 [0.063, 0.154] |
Causal Effect Estimation
Causal effect estimation measures how outcomes would change under interventions, using experiments or observational data and explicit assumptions.
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