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

Mussai, F. et al. (2023) 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. British Journal of Haematology, 200(5), pp. 573-578. (doi: 10.1111/bjh.18560) (PMID:36413792)

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.

• Item Type: Articles
• Keywords: Arginase, AML, BCT‐100, cytarabine.
• Status: Published
• Refereed: Yes
• Glasgow Author(s) Enlighten ID: Burnett, Professor Alan and Copland, Professor Mhairi
• Authors: Mussai, F., De Santo, C., Cheng, P., Thomas, I. F., Ariti, C., Upton, L., Scarpa, U., Stavrou, V., Sydenham, M., Burnett, A. K., Knapper, S. K., Mehta, P., McMullin, M. F., Copland, M., Russell, N. H., and Dennis, M.
• College/School: College of Medical Veterinary and Life Sciences > School of Cancer Sciences
• Journal Name: British Journal of Haematology
• Publisher: Wiley
• ISSN: 0007-1048
• ISSN (Online): 1365-2141
• Published Online: 22 November 2022
• Copyright Holders: Copyright © 2022 The Authors
• First Published: First published in British Journal of Haematology 200(5): 573-578
• Publisher Policy: Reproduced under a Creative Commons License