Randomized phase 2 study to assess the role of single-agent nivolumab to maintain remission in acute myeloid leukemia

• Published in: Blood advances Vol. 9; no. 9; pp. 2144 - 2152
• Main Authors: Pyzer, Athalia R., Dillon, Laura W., Sharon, Elad, Karrison, Theodore G., Zha, Yuanyuan, Fulton, Noreen, Gui, Gege, Andrew, Georgia, Streicher, Howard, Sweet, Kendra, Yaghmour, George, Liu, Jane Jijun, Jonas, Brian A., Schimmer, Aaron D., Grant, Steven, Zeidan, Amer M., Hildebrandt, Gerhard C., Lowrey, Christopher H., Mattison, Ryan J., Palmisiano, Neil, Salhotra, Amandeep, Tzachanis, Dimitrios, Baer, Maria R., Lin, Tara L., Patel, Prapti, Chen, Helen, Stadler, Walter M., Odenike, Olatoyosi, Larson, Richard A., Gajewski, Thomas F., Hourigan, Christopher S., Stock, Wendy, Liu, Hongtao
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 13.05.2025; The American Society of Hematology; Elsevier
• Subjects: Adult; Aged; Antineoplastic Agents, Immunological - adverse effects; Antineoplastic Agents, Immunological - therapeutic use; Clinical Trials and Observations; Female; Humans; Leukemia, Myeloid, Acute - drug therapy; Leukemia, Myeloid, Acute - mortality; Male; Middle Aged; Nivolumab - administration & dosage; Nivolumab - adverse effects; Nivolumab - therapeutic use; Remission Induction; Treatment Outcome; Young Adult
• ISSN: 2473-9529; 2473-9537
• DOI: 10.1182/bloodadvances.2024015176

•Nivo maintenance after AML chemotherapy did not improve the PFS and OS in this randomized phase 2 study.•There were increased AEs and SAEs with Nivo maintenance, but these AEs and SAEs were expected and manageable. [Display omitted] We conducted a multicenter, open-label, randomized phase 2 study to assess the efficacy of nivolumab (Nivo) as maintenance therapy for patients with acute myeloid leukemia (AML) in first complete remission (CR) or CR with incomplete hematologic recovery who were not candidates for stem cell transplant. Patients were stratified and randomized to observation (Obs) or Nivo (3 mg/kg IV every 2 weeks for 46 doses). The primary end point was progression-free survival (PFS) defined as time to disease relapse or death due to any reason. Secondary end points included overall survival (OS), and evaluation of adverse events (AEs) after Nivo administration. Eighty patients were enrolled with median duration of follow-up of 24 months (33 months among survivors). PFS was 13.2 months in the Nivo arm and 10.9 months in the Obs arm. Overall PFS curves were not statistically significantly different. The median OS was 53.9 months in the Nivo arm and 30.9 months in the Obs arm. There were more AEs of any type (regardless of attribution) on the Nivo arm; 27 patients (71%) on the Nivo arm had a grade ≥3 AE compared with 5 patients (12%) on the Obs arm (P < .001). Nivo maintenance after AML chemotherapy failed to improve the PFS and OS in this randomized phase 2 study. There were increased AEs and serious AEs (SAEs) with Nivo, but these AEs and SAEs were expected and manageable. This trial was registered at www.ClinicalTrials.gov as #NCT02275533.