A randomised evaluation of low‐dose Ara‐C plus pegylated recombinant arginase BCT‐100 versus low dose Ara‐C in older unfit patients with acute myeloid leukaemia: Results from the LI‐1 trial

• Published in: British journal of haematology 2023-03, Vol.200 (5), p.573-578
• Main Authors: Mussai, Francis, De Santo, Carmela, Cheng, Paul, Thomas, Ian F., Ariti, Cono, Upton, Laura, Scarpa, Ugo, Stavrou, Victoria, Sydenham, Mia, Burnett, Alan K., Knapper, Steven K., Mehta, Priyanka, McMullin, Mary F., Copland, Mhairi, Russell, Nigel H., Dennis, Mike
• Format: Article
• Language: English
• Subjects: Acute myeloid leukemia; Aged; AML; Antineoplastic Combined Chemotherapy Protocols - adverse effects; Arginase; BCT‐100; Cytarabine; Cytogenetics; Hematology; Humans; Leukemia, Myeloid, Acute; Middle Aged; Polyethylene Glycols - therapeutic use; Remission; Survival
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.18560

Summary The survival of acute myeloid leukaemia (AML) patients aged over 60 has been suboptimal historically, whether they are treated using hypomethylating agents, low‐dose cytarabine (LDAC) or venetoclax‐based regimens. Progress is being made, however, for subgroups with favourable molecular or cytogenetic findings. Arginine metabolism plays a key role in AML pathophysiology. We report the only randomised study of LDAC with recombinant arginase BCT‐100 versus LDAC alone in older AML patients unsuitable for intensive therapy. Eighty‐three patients were randomised to the study. An overall response rate was seen in 19.5% (all complete remission [CR]) and 15% (7.5% each in CR and CR without evidence of adequate count recovery [CRi]) of patients in the LDAC+BCT‐100 and LDAC arms respectively (odds ratio 0.73, confidence interval 0.23–2.33; p = 0.592). No significant difference in overall or median survival between treatment arms was seen. The addition of BCT‐100 to LDAC was well tolerated.