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678 Updated safety and efficacy of toripalimab combined with cetuximab in platinum-refractory recurrent or metastatic head and neck squamous cell carcinoma (R/M-HNSCC): a phase Ib/II clinical trial
  1. Ye Guo1,
  2. Zhendong Li2,
  3. Desheng Hu3,
  4. Song Qu4,
  5. Youhua Zhu3,
  6. Meiyu Fang5,
  7. Wantao Chen6,
  8. Chuanzheng Sun7,
  9. Hao Jiang8,
  10. Jingfeng Zong9,
  11. Jinguan Lin10,
  12. Siyang Wang11,
  13. Wei Wang10,
  14. Chuan Jin12,
  15. Guochun Cao13,
  16. Minghua Ge14,
  17. Xiaoming Huang15,
  18. Xudong Wang16,
  19. Zhiming Li17,
  20. Mo Wang18,
  21. Xianming Luo18,
  22. Shuanghui Wei18 and
  23. Yuteng Shen18
  1. 1Shanghai East Hospital, Shanghai, China
  2. 2Liaoning Cancer Hospital and Institute, Shenyang, China
  3. 3Hubei Cancer Hospital, Wuhan, China
  4. 4Guangxi Medical University Cancer Hospital, Nanning, China
  5. 5Zhejiang Cancer Hospital, Hangzhou, China
  6. 6Shanghai Ninth People’s Hospital, Shanghai Jiao Tong Univetsity School of Medicine, Shanghai, China
  7. 7Yunnan Cancer Hospital, Kunming, China
  8. 8The First Affiliated Hospital of Bengbu Medical College, Bengbu, China
  9. 9Fujian Cancer Hospital, Fuzhou, China
  10. 10Hunan Cancer Hospital, Changsha, China
  11. 11The Fifth Affiliated Hospital Sun Yat-sen University, Zhuhai, China
  12. 12Affiliated Cancer Hospital and Institute of Guangzhou Medical University, Guangzhou, China
  13. 13Jiangsu Cancer Hospital, Nanjing, China
  14. 14Zhejiang Provincial People’s Hospital, Hangzhou, China
  15. 15Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, China
  16. 16Tianjin Medical University Cancer Institute and Hospital, Tianjin, China
  17. 17Sun Yat-sen University Cancer Center, Guangzhou, China
  18. 18Shanghai Junshi Biosciences, Shanghai, China
  • Journal for ImmunoTherapy of Cancer (JITC) preprint. The copyright holder for this preprint are the authors/funders, who have granted JITC permission to display the preprint. All rights reserved. No reuse allowed without permission.


Background PD-1 inhibitors and EGFR inhibitors are effective and may provide potential synergy in R/M HNSCC. An open-label, multicenter phase Ib/II study of toripalimab (a humanized IgG4K monoclonal antibody specific for PD-1) with cetuximab was conducted in platinum-refractory or PD-L1 positive previously untreated R/M-HNSCC (NCT04856631). Here we report the results of Cohort A (platinum-refractory).

Methods Eligible patients with R/M HNSCC progressed upon 1st-line platinum-containing treatment or developed R/M disease within 6 months of platinum-containing neo-adjuvant/adjuvant or chemo-radiation therapy who had no prior immunotherapy or EGFR inhibitors therapy were enrolled. Toripalimab was administered at 240mg intravenously (IV) Q3W and cetuximab was given as a loading dose of 400mg/m2 IV followed by 250mg/m2 QW. The primary endpoint was objective response rate (ORR) by an independent review committee (IRC) per RECIST v1.1. Secondary endpoints included ORR, disease control rates (DCR), duration of response (DOR), progression-free survival (PFS) by the investigators and IRC, overall survival (OS), and safety.

Results By the data cutoff date of Apr. 14, 2023, a total of 45 patients including 35 (77.8%) males were enrolled in Cohort A, with the median follow-up duration of 10.0 months. The median age of the patients was 59 (range 32–74) years. Eighteen (40.0%) patients had distant metastases and 31 (68.9%) were PD-L1 CPS ≥1. As assessed by the IRC, the confirmed ORR was 60% (95% CI 44.3%, 74.3%) with 1 CR and 26 PR, and the median DOR was 17.9 (95% CI 7.8, NA) months, the median PFS was 9.9 (95% CI 4.2, NA) months, 12 months PFS rate was 40.7%. Similar results were seen per investigators’ assessment. The median OS was 15.4 (95% CI 8.5, 17.7) months, 12 months OS rate was 54.4%. Patients with positive PD-L1 expression (CPS≥1) may benefit more than negative patients (ORR: 64.5% vs 40%, median PFS: 10.4 m vs 4.0 m, median OS: 15.4 m vs 11.7 m). Forty-two (93.3%) patients experienced treatment-related adverse events (TRAEs). Eleven (24.4%) patients experienced immune-related adverse events (irAEs). Ten (22.2%) patients occurred Grade≥3 TRAEs and no Grade≥3 irAEs occurred. No fatal AEs related to the study treatment was reported.

Conclusions Toripalimab combined with cetuximab were well tolerated and showed promising clinical efficacy in patients with R/M HNSCC.

Trial Registration NCT04856631

Ethics Approval The study obtained ethics approval from Shanghai East Hospital Drug clinical Trial Ethics Committee [[2020]&x4E34;&x5BA1;&x7B2C; (072) &x53F7;], and participants gave informed consent before taking part.

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