Table 2. Efficacy Results in the PPS Population.
| Group | HD201 | Referent trastuzumab | Difference, % (95% CI)a | Ratio (95% CI) | ||
|---|---|---|---|---|---|---|
| Unadjusted | Adjustedb | Unadjusted | Adjustedb | |||
| PPS, No. | 238 | 236 | NA | NA | NA | NA |
| Locally assessed tpCR | ||||||
| Responders, No. (%) [95% CI] | 107 (45.0) [38.5 to 51.5] | 115 (48.7) [42.2 to 55.3] | −3.8 (−12.8 to 5.4) | −2.1 (−10.9 to 6.7) | 0.92 (0.76 to 1.12) | 0.90 (0.75 to 1.07) |
| Centrally assessed tpCR | ||||||
| Responders, No. (%) [95% CI] | 102 (49.8) [42.7 to 56.8] | 112 (51.9) [45.0 to 58.7] | −2.1 (−11.6 to 7.5) | −2.1 (−11.3 to 7.1) | 0.96 (0.80 to 1.16) | 0.95 (0.80 to 1.14) |
| Locally assessed bpCR | ||||||
| Responders, No. (%) [95% CI] | 126 (52.9) [46.4 to 59.4] | 127 (53.8) [47.2 to 60.3] | −0.9 (−10.0 to 8.2) | −0.4 (−9.2 to 8.5) | 0.98 (0.83 to 1.16) | 0.95 (0.81 to 1.12) |
| Centrally assessed bpCR | ||||||
| Responders, No. (%) [95% CI] | 129 (56.3) [49.6 to 62.9] | 123 (53.9) [47.2 to 60.5] | 2.4 (−7.0 to 11.6) | 2.8 (−6.2 to 11.7) | 1.04 (0.89 to 1.23) | 1.02 (0.87 to 1.19) |
| Overall response at the end of neoadjuvant treatment | ||||||
| Responders, No. (%) | 217 (91.2) | 210 (89.0) | 2.2 (−6.7 to 11.3) | 1.5 (−3.9 to 6.9) | 1.03 (0.97 to 1.09) | 1.02 (0.96 to 1.08) |
Abbreviations: bpCR, breast pathological complete response; NA, not applicable; PPS, per-protocol set; tpCR, total pathological complete response.
a
Primary end point. The confidence interval was contained between the prespecified margins of equivalence (−15% to 15%). All confidence intervals were contained between the prespecified margins defining equivalence for the primary end point.
b
Adjusted for stratification factors. The effect of the stratification factors was evaluated using an adapted logistic model comprising a combination of logistic, binomial, and log-binomial regression (referred to as adapted logistic regression).