Table 1: Ipilimumab efficacy in phase II and III trials [1621].

StudyPhasePopulation ( )TreatmentORR (%)PFSMedian OS, months (95% CI)

CA184-022 [18]IIPretreated
( = 217)
Ipi 10 mg/kg
Ipi 3 mg/kg
Ipi 0.3 mg/kg
11.1%
4.2%
0.0%
24-week: 18.9%
24-week: 12.9%
24-week: 2.7%
11.4 (6.9–16.1)
8.7 (6.9–12.1)
8.6 (7.7–12.7)

CA184-008 [22]IIHeavily pretreated, progressed on prior therapy ( = 155) Ipi 10 mg/kg 5.8% NR10.2 (7.6–16.3)

CA184-007 [23]IITreatment naïve and previously treated ( = 115) Ipi 10 mg/kg
Ipi 10 mg/kg + budesonide
15.8%
12.1%
NR19.3 (12.0–34.5)
17.7 (6.8–45.0)

MDX010-08 [10]IITreatment naïve ( = 72)Ipi 3 mg/kg* 
Ipi 3 mg/kg* + DTIC
5.4%
14.3%
NR14.3 (10.2–18.8)
11.4 (6.1–15.6)

MDX010-20 [16]IIIPretreated, progressed on prior therapy ( = 676)Ipi 3 mg/kg + gp100
Ipi 3 mg/kg
Gp100
5.7%
10.9%
1.5%
Median: 2.76 months
Median: 2.86 months
Median: 2.76 months
10.0
10.1
6.4

CA184-024 [17]IIITreatment naïve ( = 502)Ipi 10 mg/kg + DTIC
DTIC
15.2%
10.3%
Median PFS similar in both arms, but overall 24% reduction in risk of progression for ipi + DTIC versus DTIC
(HR 0.76; = 0.006)
11.2 (9.4–13.6)
9.1 (7.8–10.5)

Once every 4 weeks × 4 cycles (induction).
Prophylactic budesonide was added to determine if the rate of grade ≥2 diarrhea was reduced.
CI: confidence interval; DTIC: dacarbazine; HR: hazard ratio; NR: not reported; ORR: objective response rate; PFS: progression-free survival; OS: overall survival; WHO: World Health Organization.