Randomized trial of 3 versus 6 cycles of adjuvant chemotherapy for high-risk retinoblastoma

Adjuvant therapy is an important and effective treatment for retinoblastoma. A randomized noninferiority trial at two academic eye centers in China compared 3 versus 6 cycles of adjuvant CEV chemotherapy (carboplatin, etoposide, and vincristine) for enucleated unilateral retinoblastoma with high-risk pathologic features (massive choroidal infiltration, retrolaminar optic nerve invasion, or scleral infiltration. The study included 187 patients (median age 25.0 months; IQR 20.0-37.0; 3-cycle group n=94; 6-cycle group n=93). Five-years disease-free survival rate was 90.4% in the 3-cycle group and 89.2% in the 6-cycle group. The 6-cycle group experienced a higher frequency of adverse events, greater reduction in quality of life scores, and increased costs compared with the 3-cycle group. The authors conclude that among patients with unilateral pathologic high-risk retinoblastoma, 3 cycles of CEV chemotherapy resulted in a 5-year DFS rate that was noninferior to 6 cycles of CEV chemotherapy (visual abstract).