Phase 2 study of axicabtagene ciloleucel in Japanese patients with relapsed or refractory large B-cell lymphoma

Koji Kato, Shinichi Makita, Hideki Goto, Junya Kanda, Nobuharu Fujii, Kazuyuki Shimada, Koichi Akashi, Koji Izutsu, Takanori Teshima, Natsuko Fukuda, Tokuhito Sumitani, Hiroyuki Sumi, Shinji Shimizu, Yasuyuki Kakurai, Kenji Yoshikawa, Kensei Tobinai, Noriko Usui, Kiyohiko Hatake

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Axicabtagene ciloleucel (axi-cel) is an autologous chimeric antigen receptor T-cell based anti-CD19 therapy. The ZUMA-1 study, multicenter, single-arm, registrational Phase 1/2 study of axi-cel demonstrated high objective response rate in patients with relapsed/refractory large B-cell lymphoma. Here, we present the results of the bridging study to evaluate the efficacy and safety of axi-cel in Japanese patients (JapicCTI-183914). Methods: This study was the phase 2, multicenter, open-label, single-arm trial. Following leukapheresis, axi-cel manufacturing and lymphodepleting chemotherapy, patients received a single infusion of axi-cel (2.0 × 106 cells/kg). Bridging therapy between leukapheresis and conditioning chemotherapy was not allowed. The primary endpoint was objective response rate. Results: Among 17 enrolled patients, 16 received axi-cel infusion. In the 15 efficacy evaluable patients, objective response rate was 86.7% (95% confidence interval: 59.5–98.3%); complete response/partial response were observed in 4 (26.7%)/9 (60.0%) patients, respectively. No dose-limiting toxicities were observed. Grade ≥ 3 treatment-emergent adverse events occurred in 16 (100%) patients—most commonly neutropenia (81.3%), lymphopenia (81.3%) and thrombocytopenia (62.5%). Cytokine release syndrome occurred in 13 (81.3%) patients (12 cases of grade 1 or 2 and 1 case of grade 4). No neurologic events occurred. Two patients died due to disease progression, but no treatment-related death was observed by the data-cutoff date (October 23, 2019). Conclusion: The efficacy and safety of axi-cel was confirmed in Japanese patients with relapsed/refractory large B-cell lymphoma who have otherwise limited treatment options. Trial registration: JapicCTI-183914.

Original languageEnglish
JournalInternational Journal of Clinical Oncology
DOIs
Publication statusAccepted/In press - 2021
Externally publishedYes

Keywords

  • Axicabtagene ciloleucel
  • CD19-specific chimeric antigen receptor
  • Japan
  • KTE-C19
  • Non-Hodgkin lymphoma

ASJC Scopus subject areas

  • Surgery
  • Hematology
  • Oncology

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