# Retreatment with Cisplatin May Provide a Survival Advantage for Children with Relapsed/Refractory Hepatoblastoma: An Institutional Experience — GCMD Library
Katherine M Somers, Rachel Bernstein Tabbouche, Alexander Bondoc, Alexander J Towbin, Sarangarajan Ranganathan, Greg Tiao, James I Geller
Background: Hepatoblastoma (HB) is the most common liver malignancy in children. There is no standard of care for management of relapsed/refractory HB (rrHB) and reports in the literature are limited.
Objective: To describe presenting features, biology, treatment strategies, and outcomes for pediatric patients with relapsed/refractory hepatoblastoma.
Methods: An IRB-approved retrospective institutional review of patients with rrHB who presented for consultation and/or care from 2000-2019. Clinical, radiographic, and histologic data were collected from all patients.
Results: Thirty subjects were identified with a median age of 19.5 months (range 3-169 months) at initial diagnosis and 32.5 months (range 12-194 months) at time of first relapse. 63% of subjects were male, 70% Caucasian, and 13% were born premature. Three subjects had a known cancer predisposition syndrome. Eight patients had refractory disease while 22 patients had relapsed disease. Average time from initial diagnosis to relapse or progression was 12.5 months. Average alpha-fetoprotein (AFP) at initial diagnosis was 601,203 ng/mL (range 121-2,287,251 ng/mL). Average AFP at relapse was 12,261 ng/mL (range 2.8-201,000 ng/mL). For patients with tumor sequencing (n = 17), the most common mutations were in CTNNB1 (13) and NRF2 (4). First relapse sites were lungs (n = 12), liver (n = 11) and both (n = 6). More than one relapse/progression occurred in 47% of subjects; 6 had ≥3 relapses. Pathology in patients with multiply relapsed disease was less differentiated including descriptions of small cell undifferentiated (n = 3), pleomorphic (n = 1), transitional liver cell tumor (n = 2) and HB with carcinoma features (n = 1). All subjects underwent surgical resection of site of relapsed disease with 7 subjects requiring liver transplantation. Overall survival was 50%. Survival was associated with use of cisplatin at relapse (78.6% with vs. 25% without, p = 0.012). The most common late effect was ototoxicity with at least mild sensorineural hearing loss found in 80% of subjects; 54% required hearing aids.
Conclusions: Retreatment with cisplatin at the time of relapse may provide an advantage for some patients with hepatoblastoma. Multiply relapsed disease was not uncommon and not associated with a worse prognosis. Careful attention should be paid to cumulative therapy-induced toxicity while concurrently aiming to improve cure.
Type: video · 1 min · posted 2026-01-19
Canonical: https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394
## Chapters
- [0:00](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=0) Cisplatin Retreatment in Relapsed Hepatoblastoma
## Statements
- "There is no established standard salvage therapy regimen for relapsed hepatoblastoma" — Sophia Schermerhorn (guideline) [0:12](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=12)
- "The Cincinnati Children's retrospective review of 30 patients represents one of the largest published cohorts evaluating patients with refractory or recurrent hepatoblastoma" — Sophia Schermerhorn (epidemiological) [0:17](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=17)
- "The overall survival for the cohort was about 50%" — Sophia Schermerhorn (clinical) [0:25](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=25)
- "Children who received cisplatin as part of their salvage therapy had survival of about 80% compared to 25% for those who did not receive cisplatin" — Sophia Schermerhorn (clinical) [0:28](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=28)
- "The improved survival outcomes with cisplatin may be due to persistent platinum sensitivity or better tumor biology rather than proving causality" — Sophia Schermerhorn (opinion) [0:36](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=36)
- "Most patients underwent additional surgery as part of their salvage therapy, either liver resection, liver transplant, or pulmonary metastasectomy" — Sophia Schermerhorn (clinical) [0:49](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=49)
- "Salvage therapy for relapsed hepatoblastoma is multimodal" — Sophia Schermerhorn (clinical) [0:57](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=57)
- "Cisplatin retreatment should be balanced with the risk of cumulative toxicity" — Sophia Schermerhorn (opinion) [0:59](https://library.globalcastmd.com/watch/retreatment-with-cisplatin-may-provide-a-survival-advantage-for-children-with-relapsed-refractory-hepatoblastoma-an-institutional-experience-11394?t=59)
## Transcript
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.
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Not medical advice · citation policy: https://library.globalcastmd.com/ai