Adjuvant Chemotherapy for High-Risk Retinoblastoma
Trial question
Is 3 cycles of adjuvant chemotherapy noninferior to 6 cycles of adjuvant chemotherapy in patients with unilateral high-risk retinoblastoma?
Study design
Multi-center
Open label
RCT
Population
Characteristics of study participants
44.0% female
56.0% male
N = 187
187 patients (83 female, 104 male).
Inclusion criteria: patients with enucleated unilateral retinoblastoma with high-risk pathological features.
Key exclusion criteria: prior treatment for retinoblastoma; clinically evident extraocular infiltration; evidence of distant metastasis.
Interventions
N=94 3-cycle adjuvant chemotherapy (receipt of 3 cycles of carboplatin, etoposide, and vincristine chemotherapy).
N=93 6-cycle adjuvant chemotherapy (receipt of 6 cycles of carboplatin, etoposide, and vincristine chemotherapy).
Primary outcome
Disease-free survival at 5 years
90.4%
89.2%
90.4 %
67.8 %
45.2 %
22.6 %
0.0 %
3-cycle adjuvant
chemotherapy
6-cycle adjuvant
chemotherapy
Difference not exceeding
non-inferiority
margin ✓
Difference not exceeding non-inferiority margin in disease-free survival at 5 years (90.4% vs. 89.2%; AD 1.2%, 95% CI -7.5 to 9.8).
Secondary outcomes
No significant difference in overall survival at 5 years (91.5% vs. 89.3%; HR 1.28, 95% CI 0.51 to 3.23).
No significant difference in decline in physical functioning, Pediatric QoL Inventory 4.0 score at 6 months (-2.2 points vs. -8 points; AD 5.8 points, 95% CI -3.43 to 15.03).
No significant difference in decline in emotional functioning, Pediatric QoL Inventory 4.0 score at 6 months (-0.1 points vs. -4.4 points; AD 4.3 points, 95% CI -64.36 to 72.96).
Safety outcomes
Significant difference in grade 1 or 2 adverse events (78.5% vs. 95.7%).
Conclusion
In patients with enucleated unilateral retinoblastoma with high-risk pathological features, 3-cycle adjuvant chemotherapy was noninferior to 6-cycle adjuvant chemotherapy with respect to a disease-free survival at 5 years.
Reference
Huijing Ye, Kang Xue, Ping Zhang et al. Three vs 6 Cycles of Chemotherapy for High-Risk Retinoblastoma: A Randomized Clinical Trial. JAMA. 2024 Nov 19;332(19):1634-1641.
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