Hepatoblastoma, a common liver cancer in young children, often requires aggressive treatment strategies. Historically, a combination chemotherapy regimen of cisplatin and doxorubicin has been a standard approach for standard-risk hepatoblastoma. However, a pivotal study investigated whether cisplatin alone could be as effective, potentially reducing unnecessary exposure to doxorubicin and its associated side effects. This article delves into a comparative analysis of cisplatin monotherapy versus cisplatin combined with doxorubicin for treating standard-risk hepatoblastoma.
Study Design and Methodology
A randomized controlled trial was conducted to directly compare the effectiveness of cisplatin alone against cisplatin plus doxorubicin in children under 16 years old with standard-risk hepatoblastoma. Standard-risk was defined as tumors involving three or fewer liver sectors and an alpha-fetoprotein level greater than 100 ng per milliliter. After an initial cycle of cisplatin, patients were randomly assigned to receive either cisplatin alone or cisplatin plus doxorubicin for three preoperative and two postoperative cycles. The primary goal of the study was to determine if cisplatin monotherapy was non-inferior to the combination therapy in achieving complete tumor resection.
Efficacy in Achieving Complete Resection and Survival Rates
The results of the study demonstrated that cisplatin monotherapy was indeed non-inferior to the cisplatin-doxorubicin combination. In the intention-to-treat analysis, the complete resection rate was remarkably high in both groups: 95% for cisplatin alone and 93% for the combination therapy. A per-protocol analysis further reinforced these findings, with complete resection rates of 99% and 95% respectively. Furthermore, there was no significant difference in survival outcomes. The three-year event-free survival rates were 83% in the cisplatin group and 85% in the cisplatin-doxorubicin group. Similarly, overall survival rates were 95% and 93% for cisplatin alone and combination therapy, respectively, after a median follow-up of 46 months.
Safety and Tolerability: Adverse Event Comparison
While both treatment approaches showed similar efficacy, a significant difference emerged in terms of safety. The study revealed that combination therapy with cisplatin and doxorubicin was associated with a substantially higher incidence of acute grade 3 or 4 adverse events (74.4%) compared to cisplatin monotherapy (20.6%). This highlights a key advantage of cisplatin alone in reducing the burden of treatment-related toxicities in young patients.
Conclusion: The Role of Cisplatin in Hepatoblastoma Treatment
This comparative study provides strong evidence that cisplatin monotherapy achieves comparable rates of complete resection and survival to cisplatin plus doxorubicin in children with standard-risk hepatoblastoma. Crucially, cisplatin alone is associated with significantly fewer severe adverse events. These findings suggest that doxorubicin can be safely omitted from the standard treatment regimen for this patient population, potentially minimizing treatment-related toxicity without compromising treatment effectiveness. This has important implications for reducing the short-term and long-term side effects experienced by children undergoing treatment for standard-risk hepatoblastoma.
