Retreatment with Cisplatin May Provide a Survival Advantage for Children with Relapsed/Refractory Hepatoblastoma: An Institutional Experience
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- - Cisplatin retreatment at relapse significantly improved survival in pediatric hepatoblastoma (78.6% vs 25% without, p=0.012). - Multiple relapses occurred in 47% of patients but were not associated with worse prognosis compared to single relapse. - Ototoxicity affected 80% of survivors, with 54% requiring hearing aids—highlighting need for audiology monitoring. - Multiply relapsed disease showed less differentiated histology including small cell undifferentiated and carcinoma features. - Surgical resection remained critical for relapsed disease management, with 7 patients requiring liver transplantation.
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When hepatoblastoma relapses, what treatment options do you have? Hi, I'm Doctor Sophia Skermorn from Cincinnati Children, and this study examines outcomes of patients with relapse or refractory hepatoblastoma who are retreated with cisplatin therapy. In relapse hepatoblastoma, there's no established standard salvage therapy regimen. This Cincinnati Children's retrospective review of 30 patients represents one of the largest published cohorts evaluating patients. With refractory or recurrent disease. The overall survival for the cohort was about 50%. Children who received cisplatin as part of their salvage therapy had a much better survival than those who did not, about 80% compared to 25%. It's important to note that this association does improve causality, and these improved survival outcomes may be due to persistent platinum sensitivity or better tumor biology. Another key feature. of this cohort was aggressive surgical management. Most patients underwent additional surgery as part of their salvage therapy, either liver resection, liver transplant, or pulmonary metacystectomy. This highlights that salvage therapy is multimodal. The key takeaway is that cisplatin retreatment may be considered in carefully selected patients, especially if it's paired with surgical management, while balancing potential benefits with the risk of cumulative toxicity.