Treatment Adaptation to Surgical Timing in Pediatric Hepatoblastoma: A 19‐Year Single‐Center Experience With Resection and Liver Transplantation

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ABSTRACT Background The management of hepatoblastoma (HB) relies on chemotherapy combined with surgery, either liver resection (LR) or liver transplantation (LT), with reported overall survival (OS) of 80%, cardiac toxicity of 6%, nephrotoxicity of 21%, ototoxicity of 32%–46%. In clinical practice, deviations from protocol‐defined chemotherapy are often required to achieve resectability or bridge to transplantation. We aimed to evaluate the impact of treatment adaptation to surgical timing on OS and toxicity in a selected group of patients referred to a transplantation center. Methods We retrospectively analyzed patients with HB treated according to SIOPEL protocols at our center between 2004 and 2022. We collected clinical data, focusing on deviations from protocol‐specified therapy, timing of medical and surgical treatment, OS, treatment‐related toxicities (left ventricular ejection fraction, LVEF< 50%, estimated glomerular filtration rate, eGFR< 90 mL/mL/m 2 , increased hearing threshold of 20–40 dB at 4 kHz). Results Forty‐two patients, median age at diagnosis 1.9 years (IQR 0.7–3.5), 16/42 (38.1%) with metastatic disease, 24/42 (57.1%) with vascular involvement were recorded. Additional chemotherapy beyond protocol indications, due to persistent pulmonary metastases, delayed organ availability, or disease progression was administered to 17/20 (85%) who underwent first‐line LT and 10/22 (45%) who underwent LR ( p = 0.007). Death occurred in 3/20 (15%) versus 7/22 (31.8%) ( p = 0.2) respectively. After median follow‐up of 4.5 years (IQR 1.2–6.2) OS is 32/42 (71.4%); cardiotoxicity developed in 1/42 (2.4%), nephrotoxicity in 8/42 (19%), ototoxicity in 9/42 (21.4%). Conclusions A large proportion of patients with HB require adaptation of protocol‐specified therapy, especially LT candidates. This favors successful management and is not associated with increased toxicity compared to previous reports.