Intra-Arterial Chemotherapy effective and safe treatment for children with intraocular retinoblastoma

Currently, the procedure of IAC is done under general anesthesia by an interventional neuroradiologist experienced with the anatomic dimensions of pediatric vasculature. A microcatheter is positioned at the ostium of the ophthalmic artery through a femoral artery access. A selective contrast angiogram is performed to confirm the catheter placement and to assure a proper flow of the ophthalmic artery to the eye, and then the chemotherapy drugs (melphalan, topotecan and/ or carboplatin) are injected manually by repeated small boli. The aim of study by Castela et al was to report their initial 5-years’ experience with IAC for the treatment of intraocular retinoblastoma.

Retrospective analysis was done of consecutive cases of retinoblastoma selected to initiate IAC between 2015 and 2020, at the Portuguese National Reference Center. All included patients underwent complete ophthalmological evaluation under anesthesia with fundus photography. Diagnosis and classification of retinoblastoma was made according to the International Classification of Intraocular Retinoblastoma (ICRB). The patients were further divided into two groups: Group I for primary IAC and Group II for secondary IAC. Tumor recurrence or relapses, systemic metastasis and deaths were documented. Main efficacy outcome included ocular salvage and recurrence-free survival rates estimated using the Kaplan–Meier method.

Twenty-eight eyes (19 eyes included in Group I and 9 eyes included in Group II) were eligible and a total of 130 IAC procedures were performed, with a median number of sessions of 4 (range 1–8) for each treated eye, during a median follow-up of 21 months (range 4–64). Of the included eyes, 22 (78.6%) were preserved. An overall survival of 100% was achieved. Considering the preserved eyes, the overall median decimal visual acuity achieved at the last visit was 0.15 (range 0.02–0.8). Three patients had permanent adverse events related to IAC (cataract, vitreous hemorrhage and choroidal ischemia). Considering the survival analysis of recurrence, the mean survival without recurrence was 84.2% for Group I and 66.7% for Group II, and the mean survival without enucleation was 78.6% (no events in Group II).

The study’s results reflect the initial experience with IAC in the one Portuguese reference center for retinoblastoma treatment. During these 5 years, authors gained experience in the treatment of different staging-groups and with vast treatment modalities, including tandem therapy, bridge therapy and adjuvant local treatments such as transpupillary thermotherapy, cryotherapy and intravitreal chemotherapy, achieving a global globe preservation of 78.6%. Patients that would have previously been treated abroad were successfully managed in country, lowering the cost and burden of the treatment for the children and their parents. Considering the preserved eyes, they also achieved a favorable functional outcome with a median decimal visual acuity of 0.15, which corresponds to a preliminary value that can possibly be improved in the future with improved collaboration of older children allied to amblyopia management of vision-deprived eyes. Authors did not experience severe systemic complications associated with IAC and believe this treatment presents a good safety profile. This study is therefore important to demonstrate that IAC is an effective treatment that can be successfully applied in centers with a moderate number of patients when a multidisciplinary team is available.

“IAC has been shown to be an effective and safe treatment for children with intraocular retinoblastoma in our center. Our gained experience and favorable results with this treatment modality will allow us to broaden the use of IAC to more advanced stages of retinoblastoma and also in the treatment of recurrent and relapsing tumors. In the future, more globes will be preserved in our country, possibly with a good visual function.”

Source: Castela et al; Clinical Ophthalmology 2023:17https://doi.org/10.2147/OPTH.S398488