Hepatoblastoma, a common liver cancer in children, often requires intensive chemotherapy. Historically, a combination of cisplatin and doxorubicin has been a standard treatment for standard-risk hepatoblastoma, defined as tumors involving three or fewer liver sections and elevated alpha-fetoprotein levels. However, a recent study rigorously compared cisplatin monotherapy to cisplatin combined with doxorubicin to determine if the addition of doxorubicin provides a significant advantage. This analysis delves into the findings of this crucial research to compare cisplatin with doxorubicin and assess the strength and efficacy of each approach.
Study Methods: Comparing Treatment Regimens
The clinical trial enrolled children under 16 years old diagnosed with standard-risk hepatoblastoma. All participants initially received one cycle of cisplatin. Subsequently, they were randomly assigned to either continue with cisplatin alone or receive cisplatin plus doxorubicin. Treatments were administered in preoperative and postoperative cycles. The primary endpoint of the study was the rate of complete tumor resection, a critical factor in successful cancer treatment. The study was designed to demonstrate if cisplatin alone was non-inferior to the combination therapy, with a pre-defined margin of less than a 10% difference in complete resection rates.
Key Findings: Efficacy and Adverse Events
The results of the study, involving 255 patients, indicated that cisplatin monotherapy was remarkably effective. In the intention-to-treat analysis, the complete resection rate was 95% in the cisplatin-alone group and 93% in the cisplatin-doxorubicin group. This small difference of 1.4% (95% CI, -4.1 to 7.0) firmly established the non-inferiority of cisplatin alone. When considering the per-protocol analysis, the resection rates were even higher, reaching 99% for cisplatin alone and 95% for the combination therapy. Furthermore, the three-year event-free survival and overall survival rates were statistically similar in both groups, demonstrating comparable long-term outcomes. Specifically, event-free survival was 83% in the cisplatin group and 85% in the cisplatin-doxorubicin group, while overall survival was 95% and 93% respectively. Importantly, the study highlighted a significant difference in acute grade 3 or 4 adverse events. The combination therapy arm experienced a much higher incidence of these severe adverse events (74.4%) compared to the cisplatin monotherapy arm (20.6%).
Conclusion: Reassessing Treatment Strength
This study conclusively demonstrates that for children with standard-risk hepatoblastoma, cisplatin monotherapy achieves comparable rates of complete resection and survival to cisplatin plus doxorubicin. Given the significantly lower incidence of severe adverse events associated with cisplatin alone, the findings strongly suggest that doxorubicin can be safely omitted from the standard treatment regimen for this patient group. This evidence allows for a reassessment of the necessity of combination chemotherapy in standard-risk hepatoblastoma, indicating that cisplatin, when used alone, represents a strong and effective treatment option, minimizing unnecessary toxicity without compromising treatment outcomes.
Source: Adapted from a research article published in a peer-reviewed medical journal. ClinicalTrials.gov number, NCT00003912.
