Abstract
BackgroundTo evaluate the safety and efficacy of intra-arterial chemotherapy (IAC) for the primary or secondary treatment of infants diagnosed with advanced retinoblastoma before 3 months of age.MethodsThis single-center retrospective study included 39 infants (42 eyes) aged ≤3 months who were diagnosed with unilateral or bilateral advanced intraocular retinoblastoma (group D and E eyes) and received IAC as primary or secondary treatment between June 2012 and February 2017. Based on each patient’s therapeutic history and response to chemotherapeutic drugs, melphalan, topotecan, and/or carboplatin were used for IAC. The main outcomes included the technical success rate for IAC, survival rates, and adverse events.ResultsIn total, 29 and 13 eyes received IAC as primary and secondary treatments, respectively. Catheterization was successful in 136 of 137 procedures. All eyes in the secondary IAC group had previously received intravenous chemotherapy. The mean number of IAC sessions for each eye was 3 (range, 2–6). The 2-year ocular survival rates were 80.7% (95% confidence interval [CI], 58.9–91.7) in the primary IAC group and 91.7% (95% CI, 53.9–98.8) in the secondary IAC group. During the follow-up period, 1 patient with unilateral disease (group E) developed extraocular disease and died. The 2-year recurrence-free survival rates in the primary and secondary IAC groups were 71.9% (95% CI, 49.4–85.7) and 75.0% (95% CI, 40.8–91.2), respectively. During each catheterization procedure, the main complications included eyelid erythema (2.4%), fundus hemorrhage (11.9%), myelosuppression (7.7%), transient vomiting and hair loss (2.6%), and transient pancytopenia (2.6%). Prolonged complications included phthisis bulbi (19.0%), vision loss (19.0%), poor vision (9.5%), and cataract (2.4%). There was no case of stroke, neurological impairment, secondary malignant tumor, or metastasis.ConclusionsOur findings suggest that IAC, whether primary or secondary, is effective and fairly safe for the management of advanced retinoblastoma in infants aged < 3 months. However, adverse events related to intra-arterial injection and the visual outcomes cannot be neglected and require further investigation.
Highlights
To evaluate the safety and efficacy of intra-arterial chemotherapy (IAC) for the primary or secondary treatment of infants diagnosed with advanced retinoblastoma before 3 months of age
Phthisis bulbi and cataract were observed in 8 eyes and 1 eye, respectively. In this retrospective single-center study, we found that IAC, whether primary or secondary, is an effective treatment for advanced intraocular retinoblastoma in infants < 3 months of age, with a technical success rate of 99.3% and globe salvage rate of > 80%
Complications, such as phthisis bulbi, vision loss, poor vision, and cataract, occurred in some patients, serious adverse events related to intra-arterial injection were not observed, which suggests that IAC is fairly safe for such small infants
Summary
To evaluate the safety and efficacy of intra-arterial chemotherapy (IAC) for the primary or secondary treatment of infants diagnosed with advanced retinoblastoma before 3 months of age. Retinoblastoma is the most common primary intraocular malignancy in children, affecting approximately 1 in 15, 000–20,000 live births worldwide each year [1]. The average age at diagnosis of intraocular retinoblastoma is 18 months: 12 months for bilateral disease and 24 months for unilateral disease. With improvements in both parent and physician awareness levels, many children are being diagnosed before 3 months of age [2, 3]
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