Tolerability and efficacy of L-asparaginase therapy in pediatric patients with acute lymphoblastic leukemia
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Updated: Jun 10, 2026
In Ovo Xenografting of Patient-Derived Acute Lymphoblastic Leukemia (ALL) Cells (PDX-ALL)
Published on: August 1, 2025
Elizabeth A Raetz1, Wanda L Salzer
1Division of Pediatric Hematology/Oncology, Department of Pediatrics, New York University School of Medicine, New York, NY 10016, USA. elizabeth.raetz@nyumc.org
Insights
L-asparaginase (L-ASNase) is crucial for childhood leukemia chemotherapy. This review details its common toxicities, like hypersensitivity and pancreatitis, and compares different formulations.
Area of Science:
- Oncology
- Pharmacology
Background:
- L-asparaginase (L-ASNase) is a cornerstone of pediatric acute lymphoblastic leukemia chemotherapy.
- Two FDA-approved L-ASNase formulations exist, derived from Escherichia coli, alongside an Erwinia chrysanthemi-derived option.
Purpose of the Study:
- To review the spectrum of common toxicities associated with L-asparaginase therapy.
- To compare the efficacy of different L-asparaginase formulations.
Main Methods:
- Review of existing literature on L-asparaginase formulations and their clinical use.
- Analysis of reported toxicities and efficacy data.
Main Results:
- L-ASNase is generally well-tolerated with minimal myelosuppression.
- Significant toxicities occur in up to 30% of patients, with hypersensitivity being the most common.
- Other toxicities include pancreatitis, thrombosis, CNS complications, and liver dysfunction, linked to reduced protein synthesis.
Conclusions:
- L-asparaginase remains vital in treating childhood leukemia.
- Understanding and managing L-ASNase toxicities are crucial for patient care.
- Pegylation has improved L-ASNase by reducing immunogenicity and increasing half-life.
Abstract:
L-asparaginase (L-ASNase) has been an essential component of multiagent chemotherapy for acute lymphoblastic leukemia in childhood for over 3 decades. There are currently 2 Food and Drug Administration (FDA)-approved formulations of L-ASNase derived from Escherichia coli and 1 non-FDA approved formulation derived from Erwinia chrysanthemi. Modifications in L-ASNase have included pegylation, which decreases drug immunogenicity and increases the half-life, allowing less frequent administration. Although L-ASNase is well-tolerated in most patients and causes little myelosuppression, significant toxicities occur in up to 30% of patients. Hypersensitivity is the most common toxicity of L-ASNase therapy and limits the further use of the drug. Other significant toxicities relate to a reduction in protein synthesis and include pancreatitis, thrombosis, central nervous system complications, and liver dysfunction. The spectrum of common toxicities and the efficacy of different formulations of L-ASNase are presented in this review.
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