Table 3.

Intracranial tumor assessment by IRF and by investigator.

All BCBMHER2-positiveHER2-negativeTNBCLC
Assessment(n = 60)(n = 29)(n = 31)(n = 13)a(n = 24)
Investigator
 CNS RECIST 1.1 best response, n (%)
  CR00000
  PRg9 (15%)6 (21%)3 (10%)1 (8%)7 (29%)
  Confirmed PRb5 (8%)3 (10%)2 (6%)04 (17%)
  SD32 (53%)18 (62%)14 (45%)5 (38%)9 (38%)
  PD19 (32%)5 (17%)14 (45%)7 (54%)8 (33%)
 Intracranial ORR, n (%)9 (15%)6 (21%)3 (10%)1 (8%)7 (29%)
 (95% CI)c(7.1–26.6)(8.0–39.7)(2.0–25.8)(0.2–36.0)(12.6–51.1)
 Overall intracranial CBR, n (%)41 (68%)24 (83%)17 (55%)6 (46%)16 (67%)
 (95% CI)c(55.0–79.7)(64.2–94.2)(36.0–72.7)(19.2–74.9)(44.7–84.4)
 Intracranial PFSd
  Median PFS, weeks (95% CI)e12.1 (9.3–18.3)14.1 (11.1–23.4)11.1 (6.0–16.6)12.4 (7.0–23.4)
  3-month PFS rate, % (95% CI)f52% (38.6%–64.0%)61% (40.6%–76.1%)44% (26.2%–60.5%)54% (32.7%–71.4%)
  6-month PFS rate, % (95% CI)f18.7% (9.5%–30.2%)27.0% (12.0%–44.6%)11.2% (2.9%–25.8%)25.5% (9.5–45.2)
IRF
 CNS RECIST 1.1 best response, n (%)
  CR00000
  PRb5 (8%)4 (14%)1 (3%)1 (8%)4 (17%)
  SD41 (68%)20 (69%)21 (68%)7 (54%)15 (62%)
  PD12 (20%)5 (17%)7 (23%)4 (31%)4 (17%)
  Missing2 (3%)02 (6%)1 (8%)1 (4%)
 Intracranial ORR, n (%)5 (8%)4 (14%)1 (3%)1 (8%)4 (17%)
 (95% CI)c(2.8–18.4)(3.9–31.7)(0.1–16.7)(0.2–36.0)(4.7–37.4)
 Overall intracranial CBR, n (%)46 (77%)24 (83%)22 (71%)8 (62%)19 (79%)
 (95% CI)c(64.0–86.6)(64.2–94.2)(52.0–85.8)(31.6–86.1)(57.8–92.9)
 Intracranial PFSd
  Median PFS, weeks (95% CI)e16.6 (12.7–20.6)20.1 (11.9–25.0)15.3 (11.1–18.3)14.9 (12.7–23.4)
  3-month PFS rate, % (95% CI)f67% (52.5%–77.8%)71% (49.9%–84.3%)63% (41.3%–78.2%)83% (60.3%–93.2%)
  6-month PFS rate, % (95% CI)f23% (10.5%–37.8%)31% (12.6%–51.3%)16% (3.2%–36.6%)25% (6.7%–49.2%)
  • Abbreviations: CBR, clinical benefit rate (CR + PR + SD); LC, leptomeningeal carcinomatosis; ORR, objective response rate (CR + PR).

  • aOnly ORR and iCBR analyses stratified for TNBC.

  • bTo confirm PR, it is required that the response is sustained for at least 4 weeks. All PR reported by IRF were already confirmed.

  • c95% CI for the frequency distribution is Clopper–Pearson exact CI.

  • dKaplan–Meier methodology is used to estimate PFS.

  • e95% CIs for median are computed using Brookmeyer and Crowley method.

  • f95% CIs for rate are computed using Greenwood's formula.

  • gInvestigators reported PR and confirmed PR separately.

  • – analysis not done.