"Safety Assessment of Aqueous Humor Liquid Biopsy in Retinoblastoma: A Multicenter Study of 1,203 Procedures".

Abstract

Purpose: To evaluate the safety profile of aqueous humor (AH) liquid biopsy in pediatric patients with retinoblastoma (RB) and RB-simulating lesions through a multicenter analysis of paracentesis procedures.

Design: Retrospective multicenter study.

Subjects: A total of 1,203 paracentesis procedures were performed on 484 eyes of 425 pediatric patients, including 352 patients with RB and 73 with RB-simulating lesions.

Methods: Medical records were reviewed retrospectively to identify complications from anterior chamber paracentesis performed to obtain AH for liquid biopsy. Procedures were conducted during routine examination under anesthesia (EUA). AH samples were extracted using a 32-gauge needle and underwent cell-free DNA analysis. Complications were classified as mild, moderate, or severe based on clinical impact and required interventions.

Main outcome measures: Incidence and severity of complications related to the paracentesis procedure.

Results: Among 1,203 procedures, 1 mild complication was identified, representing an overall complication rate of 0.08%. No moderate or severe complications, including vision, eye loss, extraocular tumor spread, or death were observed at a median follow-up of 16 months. The procedure demonstrated an excellent safety profile across multiple centers, with no permanent adverse outcomes.

Conclusion: AH liquid biopsy via anterior chamber paracentesis is a safe and well-tolerated procedure in pediatric patients with RB when performed by trained ocular surgeons under general anesthesia. This large-scale analysis supports the procedure's use as a minimally invasive diagnostic tool with minimal risk, providing valuable molecular insights for RB management. These findings offer reassurance to clinicians and parents regarding the safety of AH liquid biopsy for RB.