Efficacy of intravitreal injections of melphalan in the treatment of retinoblastoma vitreous seeding

Adv Clin Exp Med. 2024 Feb;33(2):119-125. doi: 10.17219/acem/166180.

Abstract

Background: The introduction of intravitreal injections of melphalan (IVIM) has significantly improved the efficacy of retinoblastoma treatment and the prognosis for eye preservation.

Objectives: To evaluate the results of using IVIM to treat retinoblastoma vitreous seeding.

Material and methods: This was a clinical, retrospective, single-center study. Twenty-six children (27 eyes) who met all of the following inclusion criteria qualified for the study: 1) active vitreous seeding at the time of retinoblastoma diagnosis; 2) IVIM performed between 1 January 2017 and 30 September 2020; and 3) a minimum follow-up period of 12 months since the last IVIM. Doses of 20-40 μg melphalan per injection were used.

Results: The eye observation period from the last IVIM to the last ophthalmic examination averaged 32.41 months (median 30.00; range 13.00-56.00). Success (no active tumors in the vitreous body) was achieved in 24 eyes (88.9%), and a doubtful result (recurrence in the retina with a difficult-to-determine etiology) in 2 eyes (7.4%). In 1 eye (3.7%), despite treatment, active tumors were still present in the vitreous body. Out of all 27 eyes, 4 eyeballs were removed, but the direct cause of enucleation was not vitreous seeding. There were no complications in the form of intraocular inflammation, extraocular retinoblastoma or distant metastases. There was 1 case of anterior uveitis and 1 case of cataract.

Conclusions: The IVIM is a highly effective and safe form of treatment for retinoblastoma vitreous seeding.

Keywords: injection; intravitreal; melphalan; retinoblastoma; therapy.

MeSH terms

  • Antineoplastic Agents, Alkylating / therapeutic use
  • Child
  • Humans
  • Infant
  • Intravitreal Injections
  • Melphalan / therapeutic use
  • Neoplasm Seeding
  • Retinal Neoplasms* / drug therapy
  • Retinal Neoplasms* / pathology
  • Retinoblastoma* / drug therapy
  • Retinoblastoma* / pathology
  • Retrospective Studies

Substances

  • Melphalan
  • Antineoplastic Agents, Alkylating