Ten-year experience with intracameral chemotherapy for aqueous seeding in retinoblastoma: long-term efficacy, safety and toxicity

Br J Ophthalmol. 2023 Dec 18;108(1):124-130. doi: 10.1136/bjo-2022-322492.

Abstract

Aims: To report long-term results of intracameral chemotherapy (ICC) for aqueous seeding (AS) in retinoblastoma.

Methods: Retrospective study including 20 patients with primary (n=4) or secondary non-iatrogenic (n=16) AS treated with ICC according to a previously described technique between 2011 and 2020 with at least 1-year follow-up.

Results: AS control was initially achieved in all cases with a mean 5 injections of melphalan (n=13) or topotecan (n=7). Three eyes had an isolated AS relapse at a mean interval of 8 months after the first ICC course, which regressed with a second course of intracameral melphalan. Concomitant interciliary process seed implantation was treated with additional brachytherapy if sectorial (n=3) or proton therapy if annular (n=1). Other therapies including systemic, intra-arterial chemotherapy and/or focal treatments were given in 15 eyes to treat concomitant tumour sites. Eye preservation was achieved in 85% of the eyes (n=17/20) at a mean event-free follow-up of 45 months for aqueous disease, and 40 months for any other intraocular tumour activity. Three cases were enucleated due to refractory non-aqueous disease. All patients are alive without metastasis (mean follow-up of 48 months after first ICC). ICC-related intraocular toxicity included iris atrophy (n=5), cataract (n=4), posterior synechiae (n=2) and iris heterochromia (n=1). No patient suffered irreversible vision loss. Useful to normal vision was found in 82% of the cases (n=14/17).

Conclusion: ICC appears to be safe and efficient for AS without irreversible vision-threatening adverse effects. More data are needed to determine any superiority in efficiency/toxicity of topotecan versus melphalan.

Keywords: Child health (paediatrics); Neoplasia; Retina; Treatment other.

MeSH terms

  • Antineoplastic Agents, Alkylating / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Humans
  • Infant
  • Melphalan
  • Neoplasm Recurrence, Local / pathology
  • Neoplasm Seeding
  • Retinal Neoplasms* / drug therapy
  • Retinal Neoplasms* / pathology
  • Retinoblastoma* / drug therapy
  • Retinoblastoma* / pathology
  • Retrospective Studies
  • Topotecan / adverse effects
  • Vitreous Body / pathology

Substances

  • Melphalan
  • Topotecan
  • Antineoplastic Agents, Alkylating