Presentation (CTI)

OBJECTIVES: Within the MARALL protocol for pediatric acute lymphoblastic leukemia (ALL), asparaginase is administered through repeated intramuscular injections, resulting in a considerable number of hospital visits for patients. Pegaspargase, owing to its prolonged half-life, enables a reduction in the number of injections. This study aims to comprehensively assess the pharmacoeconomic impact of transitioning from asparaginase to pegaspargase, incorporating real-world consumption data from 2022 and 2025.
METHODS: A cost-consequence analysis was conducted across the full duration of the MARALL protocol (induction, consolidation, interphase, intensification). Real-world consumption data were retrieved from pharmacy records for 2022 (asparaginase) and 2025 (pegaspargase). For each ALL-risk group (standard and high), the number of protocol-required injections, drug acquisition costs, and number of hospital visits were compared. Indirect costs, including school absenteeism, were estimated. Tolerability was assessed based on adverse event profiles.
RESULTS: Regarding logistical burden, the transition to pegaspargase reduced hospital visits and school absenteeism by 84% per patient (from 19 to 3 visits for standard-risk ALL, and from 21 to 3 for high-risk). Despite a unit acquisition cost 8.6-fold higher (300€ vs 35€/vial), the estimated per-patient drug cost remains 665€ for asparaginase versus 900€ for pegaspargase per protocol course. This modest difference is offset by substantial indirect cost savings per patient, particularly among families residing in remote areas. In terms of tolerability, pegaspargase demonstrated a more favorable immunogenicity profile, resulting in fewer treatment interruptions and hospitalizations attributable to adverse events.
CONCLUSIONS: Although the annual acquisition cost of pegaspargase is higher, this agent presents an overall favorable pharmacoeconomic profile when the full burden of care is considered: an 84% reduction in per-patient hospital visits and a significant improvement in tolerability. Its adoption within the MARALL protocol represents a high-value therapeutic choice, offering particular benefit to families from geographically remote areas and contributing meaningfully to the quality of life of young patients.