APPROVED AND EMERGING OPTIONS FOR ONCOLOGY TREATMENT-RELATED TOXICITIES: A TARGETED LITERATURE REVIEW OF THE LANDSCAPE AND REMAINING UNMET NEEDS

Author(s)

Setareh A. Williams, PhD1, Richard J. Weiss, MD2, Charles D. Williams, BS1, Ted W. Everson, PhD3;
1Star Biopharma Consulting, LLC., Health Economics and Outcomes Research, Malvern, PA, USA, 2Star Biopharma Consulting, LLC., Medical Affairs, Malvern, PA, USA, 3Fore Biotherapeutics, Medical Affairs, Philadelphia, PA, USA
OBJECTIVES: To characterize approved drugs for toxicity mitigation, describe real-world utilization patterns and outcomes, and highlight residual unmet needs and emerging approaches.
METHODS: A targeted PubMed review (2010-2025) used structured queries to identify evidence on (1) treatment-related toxicity epidemiology and (2) management, utilization, and outcomes across oncology settings. Agents were cataloged by intended use: (a) rescue therapies for acute overdose or early severe toxicity (e.g., glucarpidase, uridine triacetate, leucovorin rescue); (b) prophylactic organ-protective agents (e.g., mesna, amifostine, dexrazoxane, sodium thiosulfate, palifermin, trilaciclib); (c) countermeasures for immune-mediated toxicities (e.g., tocilizumab for cytokine release syndrome); and (d) radiation normal-tissue protectants in development (e.g., superoxide dismutase mimetics, avasopasem [GC-4419]).
RESULTS: Across drug classes, agents demonstrated substantial clinical benefit in narrow, time-sensitive indications (e.g., rescue for high-dose methotrexate or fluoropyrimidine toxicity; cardioprotection with dexrazoxane; otoprotection with sodium thiosulfate in pediatric cisplatin-treated patients; and reduced mucositis with palifermin in select HSCT regimens). Uptake is variable, influenced by provider recognition, monitoring, and timing requirements; operational logistics and site readiness; drug stocking and handling needs; labeling restrictions; and cost and payer access. Agents embedded in protocol care pathways (e.g., leucovorin rescue, mesna use, and CAR-T cytokine release syndrome management algorithms) show more consistent utilization. Gaps remain for common or chronic toxicities (e.g., chemotherapy-induced peripheral neuropathy, non-anthracycline cardiotoxicity, chronic immune-related adverse events, broad chemoradiation-associated mucositis, and adult ototoxicity) and for patient-centered outcomes addressing persistent low-grade symptoms.
CONCLUSIONS: Timely rescue administration can improve outcomes for patients experiencing severe treatment-related toxicities. Utilization of toxicity-mitigating therapies is constrained by implementation barriers, contributing to potentially avoidable morbidity and treatment disruption. Standardized protocols, earlier risk stratification, and access-enabling operational models may improve uptake. Unmet need persists for mechanism-based interventions targeting chronic toxicities and next-generation radiation protectants, supporting clinical development and positioning toxicity management as an integral component of oncology care.

Conference/Value in Health Info

2026-05, ISPOR 2026, Philadelphia, PA, USA

Value in Health, Volume 29, Issue S6

Code

CO30

Topic

Clinical Outcomes

Topic Subcategory

Relating Intermediate to Long-term Outcomes

Disease

SDC: Oncology

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