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Table 1 Summary of pembrolizumab efficacy and safety in advanced malignancies other than melanoma and NSCLC

From: Pembrolizumab

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
  1. Clinical data reported to date
  2. 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