Table 1.

Summary of pembrolizumab efficacy and safety in advanced malignancies other than melanoma and NSCLC

Study (clinical trials.gov identifier)	N	Study design	Pembrolizumab dose/schedule	Efficacy (RECIST v1.1, central review)	Safety
HNSCC
KEYNOTE-012	60	Phase I, international, open-label, nonrandomized cohort of PD-L1-positive advanced HNSCC	10 mg/kg Q2W	• ORR: 20 %	• Grade 3-4 DRAEs: 17 %
(NCT01848834) [23]
				• Median duration of response: NR (range 8+ to 41+ weeks)	• DR discontinuations: not reported
				• Median PFS: 9.3 weeks (95 % CI: 8.0–20.1)	• DR deaths: none
				• Median OS: 12.6 months
				• 6-month OS rate: 65 %
Gastric cancer
KEYNOTE-012	39	Phase I, international, open-label, nonrandomized cohort of PD-L1-positive advanced gastric cancer	10 mg/kg Q2W	• ORR: 22 %	• Grade 3-4 DRAEs: 10 %
(NCT01848834) [24]
				• Median duration of response: 24 weeks (range 8+ to 33+ weeks)	• DR discontinuations: none
				• Median PFS: 1.9 months (95 % CI: 1.8–3.5)	• DR deaths: n = 1
				• Median OS: NR
				• 6-month OS rate: 69 %
Urothelial cancer
KEYNOTE-012	33	Phase I, international, open-label, nonrandomized cohort of PD-L1-positive advanced urothelial cancer	10 mg/kg Q2W	• ORR: 25 %	• Grade 3-4 DRAEs: 15 %
(NCT01848834) [25]
				• Median duration of response: NR (range 16 to 50+ weeks)	• DR discontinuations: 3 %
				• Median PFS: 2 months (95 % CI: 1.7–4.0)	• DR deaths: none
				• Median OS: 9.3 months
				• 12-month OS rate: 55 %
Triple-negative breast cancer
KEYNOTE-012	32	Phase I, international, open-label, nonrandomized cohort of PD-L1-positive advanced triple-negative breast cancer	10 mg/kg Q2W	• ORR: 19 %	• Grade 3-4 DRAEs: 16 %
(NCT01848834) [26]
				• Median duration of response: NR (range 15 to 40+ weeks)	• DR discontinuations: 3 %
				• Median PFS: 1.9 months (95 % CI: 1.7–5.4)	• DR deaths: n = 1
				• OS: not reported
Hodgkin lymphoma
KEYNOTE-013	29	Phase I, international, open-label, nonrandomized cohort of PD-L1-positive Hodgkin lymphoma	10 mg/kg Q2W	• ORR: 66 %	• Grade 3-4 DRAEs: 10 %
(NCT01953692) [27]
				• Median duration of response: NR (range 1+ to 185+ days)	• DR discontinuations: not reported
				• PFS: not reported	• DR deaths: n = 0
				• OS: not reported

Clinical data reported to date

Abbreviations: AE adverse event; CI confidence interval; DR drug-related; DRAE drug-related AE; HNSCC head and neck squamous cell carcinoma; NR not reached; NSCLC non-small cell lung cancer; ORR overall response rate; OS overall survival; PD-L1 programmed death receptor ligand 1; PFS progression-free survival; Q2W once every 2 weeks; Q3W once every 3 weeks