Table 4.
Clinical activity in patients with measurable disease at baseline
| n (%) |
PIK3CA-mutated (n = 13) |
PIK3CA-MND (n = 21) |
PIK3CA mutation status unknowna (n = 10) |
All patients (N = 44) |
|---|---|---|---|---|
| Best confirmed response | ||||
| Responders | 5 (38.5) | 3 (14.3) | 2 (20.0) | 10 (22.7) |
| 95% CI for response rate | 13.9–68.4 | 3.0–36.3 | 2.5–55.6 | 11.5–37.8 |
| Non-responders | 8 (61.5) | 18 (85.7) | 8 (80.0) | 34 (77.3) |
| Complete response | 0 | 0 | 0 | 0 |
| 95% CI | 0.00–24.7 | 0.0–16.1 | 0.0–30.8 | 0.0–8.0 |
| Partial response | 5 (38.5) | 3 (14.3) | 2 (20.0) | 10 (22.7) |
| 95% CI | 13.9–68.4 | 3.0–36.3 | 2.5–55.6 | 11.5–37.8 |
| Clinical benefit rate | 5 (38.5) | 5 (23.8) | 3 (30.0) | 13 (29.5) |
| 95% CI | 13.9–68.4 | 8.2–47.2 | 6.7–65.2 | 16.8–45.2 |
| Median duration of response, months | 8.8 | 18.5 | 30.5 | 19.6 |
| 95% CI | 3.7–36.1 | 17.4–19.6 | NE | 8.8–31.4 |
| Patients with disease progression | 2 (15.4) | 8 (38.1) | 2 (20.0) | 12 (27.3) |
a
One patient had missing or unavailable response data; they died prior to receiving a post-baseline tumor assessment, as a result of pericardial effusion related to study disease and device-related infection. 95% CI for median duration of response was calculated using the method of Brookmeyer and Crowley; all others used the Clopper–Pearson method. Patients were classified as missing or NE if no post–baseline response assessments were available or all post–baseline response assessments were unevaluable. Clinical benefit was defined as an objective response or stable disease lasting for ≥24 weeks since first study treatment.