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
 Responders5 (38.5)3 (14.3)2 (20.0)10 (22.7)
  95% CI for response rate13.9–68.43.0–36.32.5–55.611.5–37.8
 Nonresponders8 (61.5)18 (85.7)8 (80.0)34 (77.3)
Complete response0000
 95% CI0.00–24.70.0–16.10.0–30.80.0–8.0
Partial response5 (38.5)3 (14.3)2 (20.0)10 (22.7)
 95% CI13.9–68.43.0–36.32.5–55.611.5–37.8
Clinical benefit rate5 (38.5)5 (23.8)3 (30.0)13 (29.5)
 95% CI13.9–68.48.2–47.26.7–65.216.8–45.2
Median duration of response, months8.818.530.519.6
 95% CI3.7–36.117.4–19.6NE8.8–31.4
Patients with disease progression2 (15.4)8 (38.1)2 (20.0)12 (27.3)
  • aOne patient had missing or unavailable response data; this patient died before receiving a postbaseline 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.