Single-center phase 2 study of PD-1 inhibitor combined with DNA hypomethylation agent + CAG regimen in patients with relapsed/refractory acute myeloid leukemia

• Published in: Cancer Immunology, Immunotherapy Immunotherapy, 2023-08, Vol.72 (8), p.2769-2782
• Main Authors: Gao, Xiao-Ning, Su, Yong-Feng, Li, Meng-yue, Jing, Yu, Wang, Jun, Xu, Lei, Zhang, Lin-Lin, Wang, An, Wang, Yi-Zhi, Zheng, Xuan, Li, Yan-Fen, Liu, Dai-Hong
• Format: Article
• Language: English
• Subjects: 5-aza-2'-deoxycytidine; Acute myeloid leukemia; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Bronchopulmonary infection; Cancer Research; CD4 antigen; Cytarabine - therapeutic use; Decitabine; Humans; Immune Checkpoint Inhibitors - therapeutic use; Immunology; Leukemia; Leukemia, Myeloid, Acute - drug therapy; Lymphocytes T; Medicine; Medicine & Public Health; Oncology; Patients; PD-1 protein; PD-L1 protein; Remission; Remission (Medicine); Statistical analysis; Treatment Outcome; Trinucleotide repeats
• ISSN: 0340-7004; 1432-0851
• DOI: 10.1007/s00262-023-03454-y

Anti-PD-1 monotherapy had limited clinical efficacy in relapsed/refractory (r/r) AML patients with higher PD-1 and PD-L1 expression. Hence, we investigated the efficacy and safety of PD-1 inhibitor with DNA hypomethylating agent (HMA) + CAG regimen in patients who had failed prior AML therapy. In this phase 2, single-arm study, r/r AML patients received azacitidine or decitabine plus CAG regimen with tislelizumab. Primary endpoints were efficacy (objective response rate [ORR]) and safety. Secondary endpoints included overall survival (OS), event-free survival (EFS) and duration of response (DOR). Statistical analyses were performed using Stata 14.0 and SPSS 20.0 software where P