The Safety and Effectiveness of Melphalan-Based Intra-Arterial Chemotherapy for Retinoblastoma: An Updated Single-Arm Systematic Review and Meta-Analysis

Melphalan-based intra-arterial chemotherapy was considered an innovative treatment for retinoblastoma patients because high rates of globe salvage could be obtained. Now it has been widely applied for primary or secondary treatment of retinoblastoma. This meta-analysis summarizes the most up-to-date evidence regarding the safety and effectiveness of melphalan-based intra-arterial chemotherapy in the treatment of retinoblastoma. The authors searched PubMed, EMBASE, and the Web of Science electronic databases for studies investigating the safety and effectiveness of melphalan-based intra-arterial chemotherapy in the treatment of retinoblastoma. Studies reporting outcomes and complications of melphalan-based intra-arterial chemotherapy for the treatment of retinoblastoma patients would be included. A total of 33 observational studies that involved 1900 patients and 2336 eyes were included. The overall globe salvage rate was 79.6% (773/971 eyes, 0.74 [95% CI: 0.66, 0.80]) for patients treated with IAC as primary therapy in 28 studies. The overall globe salvage rate was 66.4% (923/1391 eyes, 0.68 [95% CI: 0.60, 0.76]) for patients treated with IAC as secondary therapy in 25 studies. The most common ocular complications were retinopathy (32%) and palpebral edema (29.7%). The most common systemic complications were nausea/vomiting (20.9%). The overall metastasis rate was 1.1% (21/1793 patients, 0.038 [95% CI: 0.020, 0.038]). Twenty-nine studies that involved 1783 patients reported the mortality and the overall mortality was 1.5% (26/1783 patients, 0.029 [95% CI: 0.020, 0.048]). Our meta-analysis showed that melphalan-based IAC treatment was an option for retinoblastoma patients with acceptable efficacy according to retrospective studies. Further high-quality randomized control trials are necessary to provide more accurate and reliable results.