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

Francis Mussai, Carmela De Santo, Paul Cheng, Ian F Thomas, Cono Ariti, Laura Upton, Ugo Scarpa, Victoria Stavrou, Mia Sydenham, Alan K Burnett, Steven K Knapper, Priyanka Mehta, Mary F McMullin, Mhairi Copland, Nigel H Russell, Mike Dennis

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Abstract

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.

Original languageEnglish
Pages (from-to)573-578
Number of pages6
JournalBritish Journal of Haematology
Volume200
Issue number5
Early online date22 Nov 2022
DOIs
Publication statusPublished - Mar 2023

Bibliographical note

© 2022 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.

Keywords

  • Hematology

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