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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.
- 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