Effectiveness of cisplatin monotherapy in standard-risk hepatoblastoma

Abstract

Risk-adapted therapy is the standard of care for hepatoblastoma (HB). The aim of this study was to analyze the effectiveness of cisplatin monotherapy in patients with standard-risk HB. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation. For the period 02.2012–12.2019 (95 months) 60 patients with standard-risk HB aged 0–8 years were treated within the framework of the cooperation of Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation and B.V. Petrovsky Russian Scientific Center of Surgery. The SIOPEL criteria were used for stratification into risk groups. Throughout the study period, standard-risk patients received therapy per SIOPEL-3 SR protocol, including cisplatin monotherapy. Survival was assessed by the Kaplan–Meier method. For the purposes of this study, overall survival (OS), event-free survival (EFS), where any modification of chemotherapy regimen towards its escalation were considered as an additional event, and progression-free survival (PFS) were calculated. The survival analysis was carried out on 15.01.2021; 54/60 (90%) patients were treated with cisplatin monotherapy and included in the final analysis. Median age at diagnosis was 11.3 (range 0.0–87.7) months. Male:female ratio – 0.86:1. Distribution by PRETEXT stages: I – 14 (25.9%), II – 30 (55.6%), III – 10 (18.5%) patients. The median alphafetoprotein level at the time of diagnosis was 162 979 (range 129–2 000 000) ng/ml. Modification of therapy without confirmed relapse/progression was required in 3/54 patients. Median follow-up was 47.1 (range 2–99) months. Among 54 patients 52 (96.3%) are alive, 2 (3.7%) patients died (1/2 – complications of surgical treatment). Relapses/progressions were noted in 4/54 (7.4%) patients, one of whom died due to disease progression. The three-year OS was 98.1% (95% confidence interval (CI) 94.6–100), EFS – 85.1% (95% CI 75.5–94.6), PFS – 90.5% (95% CI 82.5–98.4). Our data are consistent with the original studies of the SIOPEL group and convincingly confirm the effectiveness of cisplatin monotherapy in patients with HB of the standard risk group in the Russian Federation. Currently this regimen is incorporated into the national clinical guidelines of HB therapy and considered as a standard of care.