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
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.
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