Neoadjuvant cemiplimab for resectable hepatocellular carcinoma: A single-arm, open-label, phase 2 trial
The Lancet: Gastroenterology & Hepatology — Marron TU, Fiel MI, Hamon P, et al. | February 11, 2022
In this largest clinical trial of a neoadjuvant anti-PD-1 monotherapy reported to date in hepatocellular carcinoma, the noted pathological responses to cemiplimab (an anti-PD-1) favor the design of larger trials to determine the optimal treatment duration and definitively establish the clinical advantage of preoperative PD-1 blockade in cases of hepatocellular carcinoma.
In this single-arm, open-label, phase 2 trial, 21 patients with resectable hepatocellular carcinoma (stage Ib, II, and IIIb) were enrolled; all patients were treated with neoadjuvant cemiplimab, and 20 patients had successful resection (4 [20%] of which had significant tumor necrosis).
A partial response was achieved in three (15%) of 20 patients, and all other patients maintained stable disease.
During the neoadjuvant treatment period, a treatment-emergent adverse event of any grade occurred in 20 (95%) patients.
Elevated aspartate aminotransferase (in four patients), increased blood creatine phosphokinase (in three), constipation (in three), and fatigue (in three) were documented as the most common adverse events of any grade.
Grade 3 adverse events, including raised blood creatine phosphokinase (in two patients) and hypoalbuminemia (in one), occurred in seven patients.
Grade 4 or 5 events did not occur, and pneumonitis developed in one patient resulting in a delay in surgery by 2 weeks.