Table 1. Examples of clinical trials with PD-1 and PD-L1 blockade.

Clinical trials with less than 20 cases or published after 1 October 2015 were not included in the table. CRC, colorectal cancer; HSCT, hematopoietic stem cell transplantation.

Target and drug informationClinical response rate in different cancer typesPhaseCasesReferences
Target: PD-1
Name: Nivolumab, Opdivo, BMS-936558,
MDX-1106, ONO-4538
Isotype: Humanized IgG4a
Source: Bristol-Myers Squibb,
Ono Pharmaceuticals
12.8% in treatment-refractory metastatic melanoma,
castrate-resistant prostate cancer, RCC, NSCLC, or CRC
139(50)
28% in advanced melanoma, 18% in NSCLC, 27% in RCC1296(51)
40% in melanoma treated with nivolumab + ipilimumab,
20% in nivolumab followed by ipilimumab
186(58)
87% in relapsed or refractory Hodgkin’s lymphoma123(56)
14.5% in refractory NSCLC2117(70)
31.7% in advanced melanoma progressed after
anti–CTLA-4
3405(65)
40% in previously untreated melanoma without
BRAF mutation
3418(64)
17% in previously treated advanced NSCLC2129(69)
29% in previously treated advanced RCC134(71)
20% in advanced squamous-cell NSCLC3272(68)
57.6% (nivolumab + ipilimumab) versus 19% (ipilimumab)
versus 43.7% (nivolumab) in untreated stage III or
IV melanoma
3945(63)
Target: PD-1
Name: Pembrolizumab, Keytruda,
MK-3475, lambrolizumab
Isotype: Humanized IgG4κ
Source: Merck
38% in melanoma1135(57)
26% in ipilimumab-refractory advanced melanoma1173(61)
63% versus 0% in stage IV NSCLC patients with high
and low nonsynonymous mutation burden
129(67)
19.4% in advanced NSCLC1495(66)
40% and 0% in mismatch repair–deficient/proficient CRC241(53)
33% (pembrolizumab) and 11.9% (ipilimumab) in
advanced melanoma
3834(62)
Target: PD-1
Name: Pidilizumab or CT-011
Isotype: Humanized IgG1
Source: CureTech Ltd.
51% in diffuse large B cell lymphoma (after HSCT)266(54)
66% in relapsed follicular lymphoma232(55)
Target: PD-L1
Name: MPDL3280A, RG7446
Isotype: Fc-modified human IgG1b
Source: Genentech/Roche
21% overall response rate in advanced incurable cancer NSCLC,
SCLC, melanoma, RCC, CRC, gastric cancer, head and neck
squamous cell carcinoma, breast cancer, ovarian, pancreatic
cancer, uterine cancer, sarcoma, pancreaticoduodenal cancer
1277(49)
52% in metastatic bladder cancer168(47)
Target: PD-L1
Name: BMS-936559, MDX-1105
Isotype: Fully human IgG4a
Source: Bristol-Myers Squibb
17.3% in melanoma, 11.7% in RCC, 10.2% in NSCLC, 5.9%
in ovarian cancer
1207(48)