Research Article

A Simulation Study Comparing Different Statistical Approaches for the Identification of Predictive Biomarkers

Table 2

Estimated power with exact confidence intervals (in brackets) for Scenarios 3 and 4 for all investigated methods.

n = 250n = 500n = 1000
Low cens.High cens.Low cens.High cens.Low cens.High cens.

Scenario 3Median split23.5%14.1%40.8%25.1%70.2%40.9%
(20.9–26.3%)(12.0–16.4%)(37.7–43.9%)(22.4–27.9%)(67.3–73.0%)(37.8–44.0%)
Quartile split19.2%12.4%36.4%17.7%66.0%31.2%
(16.8–21.8%)(10.4–14.6%)(33.4–39.5%)(15.4–20.2%)(63.0–68.9%)(28.3–34.2%)
Optimal split71.4%57.6%86.4%71.7%97.1%84.6%
(68.5–74.2%)(54.5–60.7%)(84.1–88.5%)(68.8–74.5%)(95.9–98.0%)(82.2–86.8%)
STEPP13.4%10.5%29.2%15.5%55.1%26.4%
(11.3–15.7%)(8.7–12.6%)(26.4–32.1%)(13.3–17.9%)(52.0–58.2%)(23.7–29.2%)
Cox (linear int.)29.9%15.2%54.2%31.6%83.8%51.9%
(27.1–32.8%)(13–17.6%)(51.1–57.3%)(28.7–34.6%)(81.4–86.0%)(48.8–55.0%)
MFPI (FP1-flex3)30.2%18.2%54.2%32.5%82.8%51.1%
(27.4–33.2%)(15.9–20.7%)(51.1–57.3%)(29.6–35.5%)(80.3–85.1%)(48–54.2%)
MFPI (FP2-flex1)20.4%12.7%42.9%21.0%74.7%41.2%
(17.9–23.0%)(10.7–14.9%)(39.8–46.0%)(18.5–23.7%)(71.9–77.4%)(38.1–44.3%)
LPLB15.2%9.1%32.7%16.2%61.2%30.9%
(13.0–17.6%)(7.4–11.1%)(29.8–35.7%)(14.0–18.6%)(58.1–64.2%)(28.0–33.9%)

Scenario 4Median split26.8%14.1%52.3%24.4%78.7%38.7%
(24.1–29.7%)(12.0–16.4%)(49.2–55.4%)(21.8–27.2%)(76–81.2%)(35.7–41.8%)
Quartile split22.8%14.2%48.6%24.2%79.4%37.6%
(20.2–25.5%)(12.1–16.5%)(45.5–51.7%)(21.6–27.0%)(76.8–81.9%)(34.6–40.7%)
Optimal split77.6%57.8%92.3%72.6%99.1%88.8%
(74.9–80.1%)(54.7–60.9%)(90.5–93.9%)(69.7–75.3%)(98.3–99.6%)(86.7–90.7%)
STEPP14.8%8.3%36.8%17.6%68.9%29.9%
(12.7–17.2%)(6.7–10.2%)(33.8–39.9%)(15.3–20.1%)(65.9–71.8%)(27.1–32.8%)
Cox (linear int.)33.9%17.7%64.4%32.4%89.8%49.2%
(31.0–36.9%)(15.4–20.2%)(61.3–67.4%)(29.5–35.4%)(87.8–91.6%)(46.1–52.3%)
MFPI (FP1-flex3)40.4%25.8%72.0%38.8%92.4%56.3%
(37.3–43.5%)(23.1–28.6%)(69.1–74.8%)(35.8–41.9%)(90.6–94.0%)(53.2–59.4%)
MFPI (FP2-flex1)22.3%14.5%51.2%25.6%84.7%41.3%
(19.8–25.0%)(12.4–16.8%)(48.1–54.3%)(22.9–28.4%)(82.3–86.9%)(38.2–44.4%)
LPLB19.4%8.4%44.1%20.1%75.2%33.8%
(17.0–22.0%)(6.8–10.3%)(41.0–47.2%)(17.7–22.7%)(72.4–77.8%)(30.9–36.8%)

Due to increased type I error probabilities, results for Optimal split and MFPI (FP1-flex3) are presented in italics.