Complete remission of refractory Hepatoblastoma post liver transplantation in a child with sorafenib monotherapy – A new hope?

Abstract

Background: Hepatoblastoma (HB), in children has a good survival chance in standard risk cases, while the prognosis for recurrent HB is guarded. Complete remission of hepatoblastoma with Sorafenib monotherapy has never been documented previously. Methods: 5 year old female child, born prematurely with birth weight of 2030grams was diagnosed with embryonal epithelial hepatoblastoma of PRETEXT stage 3, was treated with cisplatin and doxorubicin. Inoperability after four cycles prompted therapy with irinotecan followed by liver transplantation 1 year after the initial diagnosis. She had an initial recurrence in pericardium and diaphragm and 9 months later further metastatic lesions in lung, adrenal and mesenteric lymph nodes. Chemotherapy with carboplatin, vincristine and irinotecan were not effective. Metastatic disease following liver transplantation and exhaustion of all feasible chemotherapeutic and surgical options, and based on the fact that the tumor was behaving like a transitional liver cell tumor (older age at diagnosis and recurrence after conventional chemotherapy) single agent chemotherapy with Sorafenib was initiated. Results: PET-CT done at 14 months later showed complete resolution of lung nodules and with no areas of abnormal uptake confirming complete remission. There were no adverse events noted during 8 months of continuation therapy with good graft function. Conclusion: This is the first report of complete remission in paediatric hepatoblastoma on sorafenib monotherapy.