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 Jay MussaiCarmela De SantoPaul ChengIan F ThomasCono AritiLaura UptonUgo ScarpaVictoria StavrouMia SydenhamAlan K BurnettSteven K KnapperPriyanka MehtaMary Frances Frances McMullinMhairi CoplandNigel H RussellMike DennisPublished in: British journal of haematology (2022)
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.
Keyphrases
- low dose
- end stage renal disease
- acute myeloid leukemia
- newly diagnosed
- clinical trial
- prognostic factors
- study protocol
- randomized controlled trial
- nitric oxide
- open label
- systemic lupus erythematosus
- stem cells
- patient reported outcomes
- bone marrow
- community dwelling
- nitric oxide synthase
- acute respiratory distress syndrome
- extracorporeal membrane oxygenation