Are Three Cycles of Chemotherapy as Effective as Six for Retinoblastoma?

TOPLINE:

Compared with six cycles, three cycles of chemotherapy for high-risk retinoblastoma showed noninferior 5-year disease-free survival (DFS). The three-cycle regimen also resulted in fewer adverse events and lower costs.

METHODOLOGY:

• The introduction of chemotherapy has increased survival rates for patients with retinoblastoma, but the optimal number of postoperative adjuvant cycles remains unclear due to scant randomized clinical trial data for high-risk patients.
• In the new trial, participants at two premier eye centers in China were randomly assigned to receive either three (n = 94) or six (n = 93) cycles of carboplatin, etoposide, and vincristine (CEV) chemotherapy after enucleation.
• The primary endpoint was 5-year DFS, and the secondary endpoints were overall survival, safety, economic burden, and quality of life.
• Patients were followed up every 3 months for the first 2 years and then every 6 months thereafter, with a median follow-up of 79 months.
• Adverse events were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (version 5.0).

TAKEAWAY:

• The 5-year DFS rates were 90.4% and 89.2% for the three- and six-cycle groups, respectively, meeting the noninferiority criterion (P = .003).
• The six-cycle group experienced a higher frequency of adverse events, including neutropenia, anemia, and nausea, than the three-cycle group.
• The quality-of-life scores were higher in the three-cycle group, particularly in physical, emotional, and social functioning parameters.
• The total, direct, and indirect costs were significantly lower in the three-cycle group than in the six-cycle group.

IN PRACTICE:

“A three-cycle CEV regimen demonstrated noninferiority, compared with a six-cycle approach, and was and proved to be an efficacious adjuvant chemotherapy regimen for individuals diagnosed with pathologically high-risk retinoblastoma,” the authors of the study wrote.

In an accompanying editorial, Ning Li, MD, and colleagues wrote that the findings “could lead to changes in clinical practice, reducing treatment burden and costs without compromising patient outcomes.”

SOURCE:

This study was led by Huijing Ye, MD, PhD, State Key Laboratory of Ophthalmology, Zhongshan Ophthalmic Center, Sun Yat-sen University in Guangzhou, China. Both the study and editorial were published online in JAMA.

LIMITATIONS:

The open-label design of the study might introduce bias, although an independent, blinded committee evaluated the clinical outcomes. The 12% noninferiority margin was notably substantial, considering the rarity of retinoblastoma and the wide range of survival rates. The criteria for adjuvant therapy, especially regarding choroidal invasion, were debatable and required further follow-up to clarify the prognosis related to various pathologic features.

DISCLOSURES:

This study was supported by the Sun Yat-Sen University Clinical Research 5010 Program and the Shanghai Committee of Science and Technology. No relevant conflict of interest was disclosed by the authors of the paper or the editorial.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.