Spotlight on Targeted Chemotherapy in Retinoblastoma: Safety, Efficacy, and Patient Outcomes

Abstract

As the most common primary intraocular malignancy of childhood, retinoblastoma (RB) has had a complex journey in its management, following a course from enucleation as the first life-saving treatment to numerous globe-salvaging therapies during the last century. Currently, this potentially lethal disease has achieved high survival rates owing to multidisciplinary management and the introduction of neoadjuvant and multimodal chemotherapy. Therefore, the goal of treatment is shifting toward conserving the globe and vision as much as possible. Up until recently, many advanced cases of RB were enucleated primarily; however, targeted chemotherapy via the ophthalmic artery and management of intraocular seeding by local administration of chemotherapeutic agents have revolutionized the globe-conserving therapies. The added benefit of avoiding systemic complications of cytotoxic drugs resulted in these methods gaining popularity, and they are becoming a main part of care in many referral centers. Initially, there were some safety concerns regarding these approaches; however, increasing experience has shown that these modalities are relatively safe procedures and many complications can be averted by changing the choice of the drug and using some prophylactic measures. It is hoped that, in the near future, with advances in early diagnosis and patient-targeted molecular therapies, as well as gene-editing techniques, the patient's vision can be saved even in advanced RB.

Keywords: local therapy; retinoblastoma; treatment.

Figure 1

Unilateral group B retinoblastoma in the left eye of a 1-year-old baby girl (A), which regressed completely after three sessions of intra-arterial chemotherapy (B). The patient developed transient eyelid edema during the course of the disease (C).

Figure 2

Extensive chorioretinal atrophy following intra-arterial chemotherapy.

Figure 3

Sequela of periocular skin necrosis following intra-arterial chemotherapy. (Written informed consent was obtained from the parents for the publication of the image).

Figure 4

Unilateral group D retinoblastoma in the left eye of a 1.5-year-old baby boy with significant vitreous seeding (A), which regressed completely after three sessions of intra-arterial chemotherapy and four intravitreal injections of melphalan (B).