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Area of Science:

  • Ophthalmology
  • Pediatric Oncology
  • Chemotherapy

Background:

  • Retinoblastoma (RB) is the most common primary intraocular malignancy in children.
  • Intra-arterial chemotherapy (IAC) is a vital eye-salvage treatment for RB.
  • Comparing single-agent versus multi-agent IAC regimens is crucial for optimizing treatment outcomes.

Purpose of the Study:

  • To compare ocular and systemic outcomes of single-agent (melphalan) versus triple-agent (melphalan, topotecan, carboplatin) IAC for retinoblastoma eye salvage.
  • To evaluate tumor response, recurrence rates, ocular survival, and complication profiles of different IAC regimens.

Main Methods:

  • Retrospective clinical cohort study of children under 18 with RB undergoing IAC between 2016 and 2024.
  • Analysis included clinical data, IAC details, additional treatments, complications, and follow-up (minimum 6 months).
  • Outcomes compared included tumor response, recurrence-free ocular survival, ocular survival, and systemic/ocular complications.

Main Results:

  • Triple-agent IAC was associated with significantly better tumor response (91% vs. 62%, P = .030).
  • Two-year ocular survival was higher in the triple-agent group (82.6% vs. 53.8%, P = .059), though recurrence-free survival was similar (P = .700).
  • Systemic complications were more frequent with triple-agent IAC (74% vs. 38%, P = .036), but ocular complications were not significantly different (52% vs. 31%, P = .215).

Conclusions:

  • Triple-agent IAC demonstrates superior tumor response and a trend towards improved ocular survival in retinoblastoma.
  • While triple-agent IAC is linked to a higher rate of systemic complications, these are generally mild and transient.
  • The findings support the consideration of triple-agent IAC for enhanced RB eye salvage, balancing efficacy with potential side effects.