Vincristine/irinotecan/temsirolimus upfront window treatment of high-risk hepatoblastoma: A report from the Children’s Oncology Group AHEP0731 Study Committee

Thompson PA, Malogolowkin MH, Furman WL, Piao J, Krailo MD, Chung N, Brock L, Towbin AJ, McCarville EB, Finegold MJ, Ranganathan S, Dunn SP, Langham MR, McGahren ED, Tiao GM, Weldon CB, O’Neill AF, Rodriguez-Galindo C, Meyers RL, Katzenstein HM. Vincristine/irinotecan/temsirolimus upfront window treatment of high-risk hepatoblastoma: A report from the Children’s Oncology Group AHEP0731 Study Committee. Pediatr Blood Cancer. 2023 Jul;70(7):e30365. doi: 10.1002/pbc.30365. Epub 2023 Apr 19. PubMed PMID: 37073741; PubMed Central PMCID: PMC10205685.

Survival for children with metastatic hepatoblastoma (HB) remains sub-optimal. We report the response rate and outcome of two courses of vincristine/irinotecan/temsirolimus (VIT) in children with high-risk (HR)/metastatic HB. Patients with newly diagnosed HB received HR window chemotherapy if they had metastatic disease or a serum alpha fetoprotein (AFP) level < 100 ng/ml. Patients received vincristine (days 1 and 8), irinotecan (days 1-5), and temsirolimus (days 1 and 8). Cycles were repeated every 21 days. Responders had either a 30% decrease using RECIST criteria OR a 90% (> 1 log10 decline) AFP decline after two cycles. Responders received 2 additional cycles of VIT intermixed with 6 cycles of cisplatin/doxorubicin/5-fluorouracil/vincristine (C5VD). Non-responders received 6 cycles of C5VD alone.