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Biogeneric native and polyethylene glycol-conjugated E. coli asparaginases for treating children with acute lymphoblastic leukaemia

Datta, N.; Jana, B.; Goswami, S.; Roy, A.; Samaddar, S.; Kundu, S.; Gogoi, M. P.; Dasgupta, B.; Dutta, T.; Das, N.; Saha, V.; Sidhu, J.; Krishnan, S.

2025-10-30 oncology
10.1101/2025.10.28.25337165 medRxiv
Show abstract

Escherichia coli asparaginases (EcASNase) available for the treatment of children with acute lymphoblastic leukaemia (ALL) are largely of suboptimal quality in low-middle income countries (LMIC) and contribute to inferior outcomes. The pharmacokinetics, activity, and immunogenicity of a native (EcASNase) and a PEGylated ASNase (PEG-EcASNase) were analysed. Biogeneric EcASNase 10,000 IU/m2 was administered intramuscularly every 72 hours (Cohort 1, 76 patients) or every 48 hours (Cohort 2, 69 patients). Cohort 3 (176 patients) received a PEG-EcASNase biogeneric 1000 IU/m2 intramuscularly every 14 days. In Cohort 1, 69% of trough induction samples were suboptimal (<100 IU/L). Cohort 2 achieved a median trough activity >600 IU/L in induction, with no suboptimal activity. In Cohort 3 median trough activity in induction was 467 IU/L with suboptimal activity in 7-16%. Patients receiving one induction PEG-EcASNase dose had eightfold higher odds of suboptimal activity post-induction. Hypersensitivity was higher with EcASNase (12% vs. 5%) and pancreatitis more often with PEG-EcASNAse (5% vs. 1%). Anti-drug antibodies were strongly associated with silent inactivation and hypersensitivity (p=<0.0001). Pharmacological monitoring enabled optimization of EcASNase dosing, detection of silent PEG-EcASNase inactivation and identification of induction dosing intensity as a determinant of sustained activity for generic ASNase products marketed in LMICs.

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