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Three vs 6 Cycles of Chemotherapy for High-Risk Retinoblastoma: A Randomized Clinical Trial.

Three vs 6 Cycles of Chemotherapy for High-Risk Retinoblastoma: A Randomized Clinical Trial.

Authors :
Ye, Huijing
Xue, Kang
Zhang, Ping
Chen, Rongxin
Zhai, Xiaowen
Ling, Li
Xiao, Wei
Tang, Lijuan
Wang, Hongsheng
Mao, Yuxiang
Ai, Siming
Bi, Yingwen
Liu, Qing
Zou, Yusha
Qian, Jiang
Yang, Huasheng
Source :
JAMA: Journal of the American Medical Association; 11/19/2024, Vol. 332 Issue 19, p1634-1641, 8p
Publication Year :
2024

Abstract

Key Points: Question: Is the long-term efficacy of adjuvant 3-cycle carboplatin, etoposide, and vincristine (CEV) regimen considered noninferior to the standard 6-cycle CEV regimen in patients with pathologically high-risk retinoblastoma? Findings: In this noninferiority randomized clinical trial of 187 patients with a median follow-up of 79.0 months, 5-year disease-free survival for patients receiving 3-cycle and 6-cycle CEV was 90.4% and 89.2%, respectively. The difference met the noninferiority margin criterion of 12%. Meaning: A 3-cycle CEV regimen demonstrated noninferiority compared with a 6-cycle approach and was and proved to be an efficacious adjuvant chemotherapy regimen for individuals diagnosed with pathologically high-risk retinoblastoma. Importance: Adjuvant therapy is an important and effective treatment for retinoblastoma. However, there is a lack of head-to-head clinical trials comparing 3 vs 6 cycles of CEV chemotherapy (carboplatin, etoposide, and vincristine) for enucleated unilateral retinoblastoma with high-risk pathological features. Objective: To assess whether 3 cycles of CEV chemotherapy is noninferior to 6 cycles for enucleated unilateral retinoblastoma with high-risk pathological features. Design, Setting, and Participants: This double-center, randomized, open-label, noninferiority trial was conducted at 2 premier eye centers in China and included 187 patients who had undergone enucleation for unilateral retinoblastoma with high-risk pathological features (massive choroidal infiltration, retrolaminar optic nerve invasion, or scleral infiltration) between August 2013 and March 2024. The final date of follow-up was March 21, 2024. Interventions: Patients were randomly assigned to receive either 3 (n = 94) or 6 (n = 93) cycles of CEV chemotherapy regimen after enucleation. Main Outcomes and Measures: The primary end point was disease-free survival, with a noninferiority margin of 12%. Secondary end points encompassed overall survival, safety, economic burden, and the quality of life of children. Results: All 187 patients (median [IQR] age, 25.0 [20.0-37.0] months; 83 [44.4%] female) completed the trial. Median (IQR) follow-up was 79.0 (65.5-102.5) months. Five-year disease-free survival was 90.4% for the 3-cycle group vs 89.2% for the 6-cycle group (difference, 1.2% [95% CI, −7.5% to 9.8%]), which met the noninferiority criterion (P =.003 for noninferiority). 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. Conclusions and Relevance: Among patients with unilateral pathologic high-risk retinoblastoma, 3 cycles of CEV chemotherapy resulted in 5-year disease-free survival that was noninferior to 6 cycles of CEV chemotherapy. Trial Registration: ClinicalTrials.gov Identifier: NCT01906814 This randomized clinical trial examines whether 3 cycles of carboplatin, etoposide, and vincristine chemotherapy is noninferior to 6 cycles for enucleated unilateral retinoblastoma with high-risk pathological features. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00987484
Volume :
332
Issue :
19
Database :
Complementary Index
Journal :
JAMA: Journal of the American Medical Association
Publication Type :
Academic Journal
Accession number :
181106895
Full Text :
https://doi.org/10.1001/jama.2024.19981