CHLA Study Finds Aqueous Humor Liquid Biopsy is Safe for Children With Retinoblastoma
Liquid biopsy was pioneered at Children’s Hospital Los Angeles as an effective test for retinoblastoma, the most common eye cancer in babies and toddlers. Traditional biopsy is invasive and risks spreading cancer outside the eye. The CHLA researchers found that using aqueous humor—the clear fluid of the eye—could safely diagnose retinoblastoma and in 2023, launched the CAP/CLIA-validated LBSeq4Kids liquid biopsy clinical test. But the question remained, how safe is paracentesis—the procedure that extracts the aqueous humor—for analysis?
A group led by Jesse Berry, MD, Head of the Vision Center and Director of Ocular Oncology and the Retinoblastoma Program at CHLA, conducted a retrospective multicenter study assessing the safety of paracentesis to extract aqueous humor for a liquid biopsy. Their large-scale analysis, published in the journal Ophthalmology, supports paracentesis as safe for children with retinoblastoma when performed by trained ocular surgeons under general anesthesia.
Proving safety
“When we started this research, safety was the most often-cited concern for everyone,” says Dr. Berry. “But this multicenter study cohort showed that the risk of mild issues is less than one in one thousand, and the risk of more significant issues is zero. This procedure demonstrated an excellent safety profile across multiple centers and by multiple surgeons, with no permanent adverse outcomes for any child.”
The team retrospectively examined medical records from 1,203 paracentesis procedures performed on 484 eyes of 425 pediatric patients, including 352 patients with retinoblastoma and 73 with retinoblastoma-simulating lesions, to identify any complications from anterior chamber paracentesis performed under anesthesia during routine examinations. The aqueous humor samples were extracted using a 32-gauge needle and analyzed with cell-free DNA analysis.
Only one mild self-resolving complication was identified, representing an overall complication rate of 0.08%. Over a median follow-up of 16 months, no moderate or severe complications, including vision, eye loss, extraocular tumor spread, or death were observed.
“These findings offer reassurance to clinicians and parents that aqueous humor extraction for liquid biopsy for retinoblastoma is safe, and well-tolerated by patients,” says Dr. Berry. “This evidence will help clinicians in using this minimally invasive diagnostic procedure.”
