Table 1.
Baseline characteristics of eligible phase 3 trials.
| Study | Underlying malignancy | Masking | Line of treatment | Treatment agents | No. of patients | Median age (range), years | Sex, m/f | Median follow-up, months |
|---|---|---|---|---|---|---|---|---|
| A367100930 | Melanoma | Open label | 1 | Tremelimumab | 328 | 57 (22–90) | 190/138 | >40.0 |
| Chemotherapy | 327 | 56 (22–90) | 182/145 | |||||
| ARCTIC31 | Lung cancer | Open label | 3+ | Durvalumab | 62 | 64 (35–79) | 42/20 | >18.0 |
| Chemotherapy | 64 | 62 (41–81) | 48/16 | |||||
| Durvalumab + tremelimumab | 174 | 63 (26–81) | 115/59 | |||||
| Chemotherapy | 118 | 65 (42–83) | 81/37 | |||||
| ATTRACTION-432,33 | GC/GEJC | Double blind | 1 | Nivolumab + chemotherapy | 362 | 64 (25–86) | 253/109 | 26.6 |
| Placebo + chemotherapy | 362 | 65 (27–89) | 270/92 | |||||
| CA184-02434,35 | Melanoma | Double blind | 1 | Ipilimumab + dacarbazine | 252 | 58 (33–87) | 152/98 | 36.6 |
| Placebo + dacarbazine | 250 | 61 (31–76) | 149/103 | |||||
| CA184-09536 | Prostate Cancer | Double blind | 1 | Ipilimumab | 400 | 70 (44–91) | 400/0 | <54.0 |
| Placebo | 202 | 69 (42–92) | 202/0 | |||||
| CA184-10437 | Lung cancer | Double blind | 1 | Ipilimumab + chemotherapy | 388 | 64 (28–84) | 326/62 | 12.5 |
| Placebo + chemotherapy | 361 | 64 (28–85) | 309/52 | 11.8 | ||||
| CAPSTONE-138 | Lung cancer | Double blind | 1 | Adebrelimab + chemotherapy | 230 | 62 (55–66) | 184/46 | 14.4 |
| Placebo + chemotherapy | 232 | 62 (56–67) | 188/44 | 12.8 | ||||
| CASPIAN39, 40, 41 | Lung cancer | Open label | 1 | Durvalumab + tremelimumab + chemotherapy | 268 | 63 (58–68) | 202/66 | 25.1 |
| Durvalumab + chemotherapy | 268 | 62 (58–68) | 190/78 | |||||
| Placebo + chemotherapy | 269 | 63 (57–68) | 184/85 | |||||
| CheckMate 9LA42,43 | Lung cancer | Open label | 1 | Nivolumab + ipilimumab + chemotherapy | 361 | 65 (59–70) | 252/109 | 30.7 |
| Chemotherapy | 358 | 65 (58–70) | 252/106 | |||||
| CheckMate 01744, 45, 46, 47 | Lung cancer | Open label | 2 | Nivolumab | 135 | 62 (39–85) | 111/24 | 69.5 |
| Docetaxel | 137 | 64 (42–84) | 97/40 | |||||
| CheckMate 02548, 49, 50 | Renal cancer | Open label | 2+ | Nivolumab | 410 | 62 (23–88) | 315/95 | 72.0 |
| Everolimus | 411 | 62 (18–86) | 304/107 | |||||
| CheckMate 03751,52 | Melanoma | Open label | 2+ | Nivolumab | 272 | 59 (23–88) | 176/96 | 24.0 |
| Chemotherapy | 133 | 62 (29–85) | 85/48 | |||||
| CheckMate 05744,53 | Lung cancer | Open label | 2 | Nivolumab | 292 | 61 (37–84) | 151/141 | 69.4 |
| Docetaxel | 290 | 64 (21–85) | 168/122 | |||||
| CheckMate 06654, 55, 56, 57 | Melanoma | Open label | 1 | Nivolumab | 210 | 64 (18–86) | 121/89 | 32 |
| Dacarbazine | 208 | 66 (26–87) | 125/83 | 10.9 | ||||
| CheckMate 07858, 59, 60 | Lung cancer | Open label | 2+ | Nivolumab | 338 | 60 (27–78) | 263/75 | >37.3 |
| Docetaxel | 166 | 60 (38–78) | 134/32 | |||||
| CheckMate 14161, 62, 63 | Head and neck cancer | Open label | 2 | Nivolumab | 240 | 59 (29–83) | 197/43 | >24.2 |
| Chemotherapy | 121 | 61 (28–78) | 103/18 | |||||
| CheckMate 21464, 65, 66, 67 | Renal cancer | Open label | 1 | Nivolumab + ipilimumab | 550 | 62 (26–85) | 413/137 | 43.6 |
| Sunitinib | 546 | 62 (21–85) | 395/151 | 32.3 | ||||
| CheckMate 22768, 69, 70 | Lung cancer | Open label | 1 | Nivolumab + ipilimumab | 396 | 64 (26–84) | 255/141 | 54.8 |
| Nivolumab | 396 | 64 (27–85) | 272/124 | |||||
| Chemotherapy | 397 | 64 (29–87) | 260/137 | |||||
| CheckMate 45971 | HCC | Open label | 1 | Nivolumab | 371 | 65 (57–71) | 314/57 | 15.2 |
| Sorafenib | 372 | 65 (58–72) | 317/55 | 13.4 | ||||
| CheckMate 49872 | Glioblastoma | Open label | 1 | Nivolumab + radiotherapy | 280 | 60 (18–83) | 190/90 | 13.0 |
| Temozolomide + radiotherapy | 280 | 56 (23–81) | 175/105 | 14.2 | ||||
| CheckMate 54873 | Glioblastoma | Double blind | 1 | Nivolumab + radiotherapy + temozolomide | 358 | 60 (24–79) | 205/153 | >12.5 |
| Placebo + radiotherapy + temozolomide | 358 | 60 (18–81) | 197/161 | >19.5 | ||||
| CheckMate 64874 | EC | Open label | 1 | Nivolumab + chemotherapy | 321 | 64 (40–90) | 253/68 | >13.0 |
| Nivolumab + ipilimumab | 325 | 63 (28–81) | 269/56 | |||||
| Chemotherapy | 324 | 64 (26–81) | 275/49 | |||||
| CheckMate 64975,76 | GC/GEJC/EC | Open label | 1 | Nivolumab + chemotherapy | 473 | 63 (54–69) | 331/142 | >24.0 |
| Chemotherapy | 482 | 62 (54–68) | 349/133 | |||||
| CheckMate 64975,76 | GC/GEJC/EC | Open label | 1 | Nivolumab + ipilimumab | 234 | 62 (22–84) | NR | >35.7 |
| Chemotherapy | 239 | 61 (23–90) | NR | |||||
| CheckMate 74377,78 | Mesothelioma | Open label | 1 | Nivolumab + ipilimumab | 303 | 69 (65–75) | 234/69 | 29.7 |
| Chemotherapy | 302 | 69 (62–75) | 233/69 | |||||
| CONTACT-0379 | Renal cancer | Open label | 2+ | Atezolizumab + Cabozantinib | 263 | 62 (20–85) | 204/59 | 15.2 |
| Cabozantinib | 259 | 63 (18–89) | 197/62 | |||||
| COSMIC-31280 | HCC | Open label | 1 | Cabozantinib + atezolizumab | 432 | 64 (57–71) | 360/72 | 13.3 |
| Sorafenib | 217 | 67 (60–73) | 186/31 | |||||
| DANUBE81 | Urothelial cancer | Open label | 1 | Durvalumab | 346 | 67 (60–73) | 249/97 | 41.2 |
| Durvalumab + tremelimumab | 342 | 68 (60–73) | 256/86 | |||||
| Chemotherapy | 344 | 68 (60–73) | 274/70 | |||||
| EMPOWER-Cervical 182 | Cervical cancer | Open label | 2 | Cemiplimab | 304 | 51 (22–81) | 0/304 | 18.2 |
| Chemotherapy | 304 | 50 (24–87) | 0/304 | |||||
| EMPOWER-Lung 383 | Lung cancer | Double blind | 1 | Cemiplimab + chemotherapy | 312 | 63 (57–68) | 268/44 | 16.3 |
| Chemotherapy | 154 | 63 (57–68) | 123/31 | 16.7 | ||||
| ESCORT-1st84 | EC | Double blind | 1 | Camrelizumab + chemotherapy | 298 | 62 (56–66) | 260/38 | 10.8 |
| Placebo + chemotherapy | 298 | 62 (56–67) | 362/35 | |||||
| IMagyn05085 | Ovarian cancer | Double blind | 1 | Atezolizumab + bevacizumab + chemotherapy | 651 | 60 (29–84) | 0/651 | 19.9 |
| Placebo + bevacizumab + chemotherapy | 650 | 59 (18–83) | 0/650 | 19.8 | ||||
| IMbassador25086 | Prostate cancer | Open label | 2 | Atezolizumab + enzalutamide | 379 | 70 (51–91) | 379/0 | 15.2 |
| Enzalutamide | 380 | 70 (40–92) | 380/0 | 16.6 | ||||
| IMbrave15087,88 | HCC | Open label | 1 | Atezolizumab + Bevacizumab | 336 | 64 (56–71) | 277/59 | 15.6 |
| Sorafenib | 165 | 66 (59–71) | 137/28 | |||||
| IMmotion15189,90 | Renal cancer | Open label | 1 | Atezolizumab + Bevacizumab | 454 | 62 (56–69) | 317/137 | >40.0 |
| Sunitinib | 461 | 60 (54–66) | 352/109 | |||||
| IMpassion13091, 92, 93 | Breast cancer | Double blind | 1 | Atezolizumab + Nab-Paclitaxel | 451 | 55 (20–82) | 3/448 | 18.8 |
| Placebo + Nab-Paclitaxel | 451 | 56 (26–86) | 1/450 | |||||
| IMpower11094,95 | Lung cancer | Open label | 1 | Atezolizumab | 277 | 64 (30–81) | 196/81 | 30.0 |
| Chemotherapy | 277 | 65 (30–87) | 193/84 | |||||
| IMpower13096 | Lung cancer | Open label | 1 | Atezolizumab + chemotherapy | 451 | 64 (18–86) | 266/185 | 18.5 |
| chemotherapy | 228 | 65 (38–85) | 134/94 | 19.2 | ||||
| IMpower13197 | Lung cancer | Open label | 1 | Atezolizumab + carboplatin + nab-paclitaxel | 343 | 65 (23–83) | 280/63 | 26.8 |
| Carboplatin + nab-paclitaxel | 340 | 65 (38–86) | 277/63 | 24.8 | ||||
| IMpower13298 | Lung cancer | Open label | 1 | Atezolizumab + chemotherapy | 292 | 64 (31–85) | 192/100 | 28.4 |
| Chemotherapy | 286 | 63 (33–83) | 192/94 | |||||
| IMpower13399,100 | Lung cancer | Double blind | 1 | Atezolizumab + chemotherapy | 201 | 64 (28–90) | 129/72 | 23.1 |
| Chemotherapy | 202 | 64 (26–87) | 132/70 | 22.6 | ||||
| IMpower150101, 102, 103, 104 | Lung cancer | Open label | 1 | Atezolizumab + chemotherapy | 402 | 63 (32–85) | 241/161 | 38.8 |
| Atezolizumab + bevacizumab + chemotherapy | 400 | 63 (31–89) | 240/160 | 39.8 | ||||
| Bevacizumab + chemotherapy | 400 | 63 (31–90) | 239/161 | 40.0 | ||||
| IMvigor211105,106 | Urothelial cancer | Open label | 2+ | Atezolizumab | 467 | 67 (33–88) | 110/357 | 33.0 |
| Chemotherapy | 464 | 67 (31–84) | 103/361 | |||||
| IPSOS107 | Lung cancer | Open label | 1 | Atezolizumab | 302 | 75 (69–81) | 108/43 | 41.0 |
| Chemotherapy | 151 | 75 (68–80) | 108/43 | |||||
| JAVELIN Bladder 100108 | Urothelial cancer | Open label | 1 | Avelumab | 350 | 68 (37–90) | 266/84 | >19.0 |
| Placebo | 350 | 69 (32–89) | 275/75 | |||||
| JAVELIN Gastric 100109 | GC/GEJC | Open label | 1 | Avelumab | 249 | 62 | 164/85 | 24.1 |
| Chemotherapy | 250 | 61 | 167/83 | 24.0 | ||||
| JAVELIN Lung 200110,111 | Lung cancer | Open label | 2 | Avelumab | 396 | 64 (58–69) | 269/127 | 18.9 |
| Docetaxel | 396 | 63 (57–69) | 273/123 | 17.8 | ||||
| JAVELIN Ovarian 200112 | Ovarian cancer | Open label | 2 | Avelumab + chemotherapy | 188 | 60 (53–67) | 0/188 | 18.4 |
| Avelumab | 188 | 61 (53–70) | 0/188 | 18.2 | ||||
| Chemotherapy | 190 | 60 (53–69) | 0/190 | 17.4 | ||||
| JAVELIN Renal 101113,114 | Renal cancer | Open label | 1 | Avelumab + axitinib | 270 | 62 (29–83) | 203/67 | 19.3 |
| Sunitinib | 290 | 61 (27–88) | 224/66 | 19.2 | ||||
| KESTR EL115 | Head and Neck cancer | Open label | 1 | Durvalumab | 204 | 62 (26–89) | 175/29 | NR |
| Durvalumab + tremelimumab | 413 | 61 (25–87) | 340/73 | |||||
| Chemotherapy | 206 | 61 (22–84) | 174/32 | |||||
| KEYLYNK-010116 | Prostate cancer | Open label | 2+ | Pembrolizumab + Olaparib | 529 | 71 (40–89) | 529/0 | 15.7 |
| Chemotherapy | 264 | 69 (49–84) | 264/0 | |||||
| KEYNOTE-010117, 118, 119 | Lung cancer | Open label | 3+ | Pembrolizumab | 690 | 63 (56–69) | 425/265 | 67.4 |
| Docetaxel | 343 | 62 (56–69) | 209/134 | |||||
| KEYNOTE-033120 | Lung cancer | Open label | 2+ | Pembrolizumab | 213 | 61 (28–83) | 157/56 | 22.3 |
| Chemotherapy | 212 | 61 (34–81) | 164/48 | |||||
| KEYNOTE-042121 | Lung cancer | Open label | 1 | Pembrolizumab | 637 | 63 (57–69) | 450/187 | 12.8 |
| Chemotherapy | 637 | 63 (57–69) | 452/185 | |||||
| KEYNOTE-045122,123 | Urothelial cancer | Open label | 2 | Pembrolizumab | 270 | 67 (29–88) | 200/70 | 27.7 |
| Chemotherapy | 272 | 65 (26–84) | 202/70 | |||||
| KEYNOTE-048124,125 | Head and neck cancer | Open label | 1 | Pembrolizumab + chemotherapy | 281 | 61 (55–68) | 224/57 | 13.0 |
| Cetuximab + chemotherapy | 278 | 61 (55–68) | 242/93 | 10.7 | ||||
| KEYNOTE-062126 | GC/GEJC | Partial-Blind | 1 | Pembrolizumab | 256 | 61 (20–83) | 180/76 | 29.4 |
| Pembrolizumab + chemotherapy | 257 | 62 (22–83) | 195/62 | |||||
| Chemotherapy | 250 | 63 (23–87) | 179/71 | |||||
| KEYNOTE-063127 | GC/GEJC | Open label | 2 | Pembrolizumab | 47 | 61 (32–75) | 32/15 | 24.0 |
| Paclitaxel | 47 | 61 (37–91) | 37/10 | |||||
| KEYNOTE-119128 | Breast cancer | Open label | 2+ | Pembrolizumab | 312 | 50 (43–59) | 0/312 | 31.4 |
| Chemotherapy | 310 | 53 (44–61) | 2/308 | 31.5 | ||||
| KEYNOTE-189129, 130, 131 | Lung cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 410 | 65 (34–84) | 256/156 | 31.0 |
| Chemotherapy | 206 | 64 (34–84) | 109/97 | |||||
| KEYNOTE-240132 | HCC | Double blind | 2 | Pembrolizumab | 278 | 67 (18–91) | 226/52 | 13.8 |
| Placebo | 135 | 65 (23–89) | 112/23 | 10.6 | ||||
| KEYNOTE-355133,134 | Breast cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 220 | 52 (44–62) | 0/220 | 44.1 |
| Chemotherapy | 103 | 55 (43–63) | 0/103 | |||||
| KEYNOTE-361135 | Urothelial cancer | Open label | 1 | Pembrolizumab + chemotherapy | 351 | 69 (62–75) | 272/79 | 31.7 |
| Pembrolizumab | 307 | 68 (61–74) | 228/79 | |||||
| Chemotherapy | 352 | 69 (61–75) | 262/90 | |||||
| KEYNOTE-407136,137 | Lung cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 278 | 65 (29–87) | 220/58 | 14.3 |
| Chemotherapy | 281 | 65 (36–88) | 235/46 | |||||
| KEYNOTE-426138,139 | Renal cancer | Open label | 1 | Pembrolizumab + axitinib | 432 | 62 (30–89) | 308/124 | 30.6 |
| Sunitinib | 429 | 61 (26–90) | 320/109 | |||||
| KEYNOTE-590140 | EC | Double blind | 1 | Pembrolizumab + chemotherapy | 373 | 64 (28–94) | 306/67 | 22.6 |
| Chemotherapy | 376 | 62 (27–89) | 319/57 | |||||
| KEYNOTE-604141 | Lung cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 228 | 64 (24–81) | 152/76 | 21.6 |
| Chemotherapy | 225 | 65 (37–83) | 142/83 | |||||
| KEYNOTE-775142 | Endometrial cancer | Open label | 2+ | Pembrolizumab + lenvatinib | 411 | 64 (30–82) | 0/411 | 12.2 |
| Chemotherapy | 416 | 65 (35–86) | 0/416 | 10.7 | ||||
| KEYNOTE-826143 | Cervical cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 308 | 51 (25–82) | 0/308 | 22.0 |
| Chemotherapy | 309 | 50 (22–79) | 0/309 | |||||
| KEYNOTE-966144 | Biliary tract cancer | Double blind | 1 | Pembrolizumab + chemotherapy | 533 | 64 (57–71) | 280/253 | 25.6 |
| Chemotherapy | 536 | 63 (55–70) | 272/264 | |||||
| MYSTIC145 | Lung cancer | Open label | 1 | Durvalumab | 163 | 64 (32–84) | 113/50 | 30.2 |
| Durvalumab + tremelimumab | 163 | 65 (34–87) | 118/45 | |||||
| Chemotherapy | 162 | 65 (35–85) | 106/56 | |||||
| NINJA146 | Ovarian cancer | Open label | 2+ | Nivolumab | 157 | 58 (29–84) | 0/157 | <48.0 |
| Chemotherapy | 159 | 60 (34–80) | 0/159 | |||||
| OAK147, 148, 149, 150, 151 | Lung cancer | Open label | 2+ | Atezolizumab | 425 | 63 (33–82) | 261/164 | 47.7 |
| Docetaxel | 425 | 64 (34–85) | 259/166 | |||||
| ORIENT-15152 | EC | Double blind | 1 | Sintilimab + chemotherapy | 327 | 63 (57–67) | 279/48 | 16.0 |
| Placebo + chemotherapy | 332 | 63 (56–67) | 288/44 | 16.9 | ||||
| PACIFIC153, 154, 155, 156 | Lung cancer | Double blind | 3+ | Durvalumab | 476 | 64 (31–84) | 334/142 | 34.2 |
| Placebo | 237 | 64 (23–90) | 166/71 | |||||
| ORIENT-3157 | Lung cancer | Open label | 2 | Sintilimab | 145 | 61 (38–74) | 136/9 | 23.6 |
| Chemotherapy | 135 | 60 (34–75) | 122/13 | |||||
| RATIONALE 303158 | Lung cancer | Open label | 2+ | Tislelizumab | 535 | 61 (28–88) | 416/119 | 16.0 |
| Chemotherapy | 270 | 61 (32–81) | 206/64 | 10.7 | ||||
| RATIONALE 306159 | EC | Double blind | 1 | Tislelizumab + chemotherapy | 326 | 64 (59–68) | 282/44 | 16.3 |
| Chemotherapy | 323 | 65 (58–70) | 281/42 | 9.8 |
CRC, colorectal cancer; EC, Oesophageal cancer; GC, gastric cancer; GEJC, gastroesophageal junction cancer; HCC, Hepatocellular cancer.