Research Article
A Simulation Study Comparing Different Statistical Approaches for the Identification of Predictive Biomarkers
Table 1
Estimated type I error probabilities with exact
confidence intervals (in brackets) for Scenarios 1 and 2 for all investigated methods.
| | n = 250 | n = 500 | n = 1000 | Low cens. | High cens. | Low cens. | High cens. | Low cens. | High cens. |
| Scenario 1 | Median split | 4.7% | 5.3% | 4.9% | 4.4% | 5.0% | 6.4% | (3.5–6.2%) | (4.0–6.9%) | (3.6–6.4%) | (3.2–5.9%) | (3.7–6.5%) | (5.0–8.1%) | Quartile split | 6.3% | 5.3% | 4.2% | 5.1% | 3.5% | 5.7% | (4.9–8.0%) | (4.0–6.9%) | (3.0–5.6%) | (3.8–6.7%) | (2.4–4.8%) | (4.3–7.3%) | Optimal split | 43.6% | 39.9% | 45.8% | 40.9% | 45.5% | 46.6% | (40.5–46.7%) | (36.8–43.0%) | (42.7–48.9%) | (37.8–44.0%) | (42.4–48.6%) | (43.5–49.7%) | STEPP | 4.8% | 5.9% | 5.1% | 4.2% | 4.0% | 6.3% | (3.6–6.3%) | (4.5–7.5%) | (3.8–6.7%) | (3.0–5.6%) | (2.9–5.4%) | (4.9–8.0%) | Cox (linear int.) | 4.9% | 5.2% | 4.8% | 4.4% | 4.8% | 5.8% | (3.6–6.4%) | (3.9–6.8%) | (3.6–6.3%) | (3.2–5.9%) | (3.6–6.3%) | (4.4–7.4%) | MFPI (FP1-flex3) | 10.1% | 10.6% | 10.4% | 12.0% | 10.3% | 13.5% | (8.3–12.1%) | (8.8–12.7%) | (8.6–12.5%) | (10.1–14.2%) | (8.5–12.4%) | (11.4–15.8%) | MFPI (FP2-flex1) | 4.9% | 5.6% | 4.9% | 4.7% | 6.0% | 7.0% | (3.6–6.4%) | (4.3–7.2%) | (3.6–6.4%) | (3.5–6.2%) | (4.6–7.7%) | (5.5–8.8%) | LPLB | 4.6% | 4.5% | 4.2% | 4.4% | 3.9% | 5.8% | (3.4–6.1%) | (3.3–6.0%) | (3.0–5.6%) | (3.2–5.9%) | (2.8–5.3%) | (4.4–7.4%) |
| Scenario 2 | Median split | 4.2% | 6.0% | 5.7% | 4.2% | 5.7% | 4.3% | (3.0–5.6%) | (4.6–7.7%) | (4.3–7.3%) | (3.0–5.6%) | (4.3–7.3%) | (3.1–5.7%) | Quartile split | 5.3% | 6.7% | 5.1% | 4.5% | 5.0% | 5.4% | (4.0–6.9%) | (5.2–8.4%) | (3.8–6.7%) | (3.3–6.0%) | (3.7–6.5%) | (4.1–7.0%) | Optimal split | 50.8% | 42.8% | 53.9% | 45.9% | 52.0% | 47.4% | (47.7–53.9%) | (39.7–45.9%) | (50.8–57.0%) | (42.8–49.0%) | (48.9–55.1%) | (44.3–50.5%) | STEPP | 5.4% | 8.2% | 6.8% | 6.8% | 7.8% | 6.9% | (4.1–7.0%) | (6.6–10.1%) | (5.3–8.5%) | (5.3–8.5%) | (6.2–9.6%) | (5.4–8.7%) | Cox (linear int.) | 4.5% | 8.2% | 4.8% | 6.4% | 5.0% | 5.2% | (3.3–6.0%) | (6.6–10.1%) | (3.6–6.3%) | (5.0–8.1%) | (3.7–6.5%) | (3.9–6.8%) | MFPI (FP1-flex3) | 8.1% | 12.6% | 9.1% | 10.3% | 8.1% | 7.8% | (6.5–10.0%) | (10.6–14.8%) | (7.4–11.1%) | (8.5–12.4%) | (6.5–10.0%) | (6.2–9.6%) | MFPI (FP2-flex1) | 5.5% | 6.5% | 6.5% | 5.6% | 4.1% | 5.9% | (4.2–7.1%) | (5.1–8.2%) | (5.1–8.2%) | (4.3–7.2%) | (3.0–5.5%) | (4.5–7.5%) | LPLB | 6.2% | 5.8% | 7.3% | 4.8% | 7.7% | 6.1% | (4.8–7.9%) | (4.4–7.4%) | (5.8–9.1%) | (3.6–6.3%) | (6.1–9.5%) | (4.7–7.8%) |
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