Abstract
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.
Highlights
Retinoblastoma is the most common ocular malignancy in children, and the incidence is about 11 new cases per million individuals under 5 years old in Europe and the US [1, 2].75% of these patients will present with unilateral disease, with a median age peak of 2 to 3 years [1, 3]
intra-arterial chemotherapy (IAC), a local administration method, importantly avoided several adverse reactions caused by systemic chemotherapy such as ototoxicity and neurotoxicity [5]
Melphalan-based intra-arterial chemotherapy has been extensively applied for the treatment of retinoblastoma patients [7]
Summary
75% of these patients will present with unilateral disease, with a median age peak of 2 to 3 years [1, 3]. Enucleation, systemic chemotherapy, radiotherapy, and local therapies are considered standard treatment methods. In the past decade, intra-arterial chemotherapy (IAC) was used for improving tumor control and increasing globe salvage rates as a primary or secondary treatment [4]. IAC, a local administration method, importantly avoided several adverse reactions caused by systemic chemotherapy such as ototoxicity and neurotoxicity [5]. Before the application of IAC, nearly 80% of advanced patients would eventually be forced to choose enucleation [6]. Melphalan-based intra-arterial chemotherapy has been extensively applied for the treatment of retinoblastoma patients [7]. Though an increasing number of centers worldwide have adopted IAC, the optimal role for IAC is still undetermined
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