BICR = blinded independent central review; CI = confidence interval; HCC = hepatocellular carcinoma; ORR = objective response rate; RECIST v1.1 = Response
Evaluation Criteria In Solid Tumors v1.1.
Study design: KEYNOTE-224 was a single-arm, multicenter trial in patients with HCC who had disease progression on or after sorafenib or were intolerant to sorafenib; had measurable disease; and Child-Pugh class A liver impairment. Patients with active autoimmune disease, greater than 1 etiology of hepatitis, a medical condition that required immunosuppression, or clinical evidence of ascites by physical exam were ineligible for the trial. Patients received KEYTRUDA 200 mg intravenously Q3W until unacceptable toxicity, investigator-assessed confirmed disease progression (based on repeat scan at least 4 weeks from the initial scan showing progression), or completion of 24 months of KEYTRUDA. Assessment of tumor status was performed every 9 weeks. The major efficacy outcome measures were ORR and duration of response according to RECIST v1.1, modified to follow a maximum of 10 target lesions and a maximum of 5 target lesions per organ, as assessed by BICR.
Q3W = every 3 weeks.
bAll of the HBV cases and 3 of the HCV cases were inactive.
cAll patients received prior sorafenib, of whom 20% were unable to tolerate sorafenib. No patient received more than 1 prior systemic therapy (sorafenib).
HBV = hepatitis B virus; HCV = hepatitis C virus; ECOG PS = Eastern Cooperative Oncology Group performance status;
AFP = alpha-fetoprotein.