Therapeutic plasma exchange in Amatoxin-associated pediatric acute liver failure-Results from the Amanita-PEX study

Pediatric acute liver failure (PALF) is a rare but life-threatening condition. Amanita poisoning can lead to PALF with rapid clinical deterioration. Therapeutic plasma exchange (TPE) has been proposed as a potential supportive intervention and is established in adult acute liver failure. Evidence regarding TPE in Amanita toxin-induced PALF (AT-PALF) remains scarce, and clinical practice varies across liver transplant centers.

The Amanita-PEX study is a retrospective, international, multicenter cohort study including pediatric patients with AT-PALF treated between 2013 and 2024. The combined primary endpoint was 28-day transplant-free survival.

A total of 30 patients from 10 centers were included; 17 received at least one session of TPE, and 13 were treated with standard-of-care (SOC) alone. Baseline characteristics were comparable between groups. The overall 28-day transplant-free survival rate was 53.3% (16/30), with a 28-day mortality of 10% (3/30). All patients received N-acetylcysteine, and 93% were treated with silibinin. Native liver survival did not differ significantly between the TPE and SOC groups (47% vs. 62%, p = 0.63). In contrast, transplant-free survival was primarily associated with established markers of liver failure severity and organ dysfunction, including MELD and LIU scores, the degree of hepatic encephalopathy, acute kidney injury, and the need for vasopressors or mechanical ventilation. After adjusting for these prognostic markers, TPE remained unassociated with improved outcomes.

In this real-world cohort of children with AT-PALF, adjunctive TPE showed no clear improvement in transplant-free survival. Outcomes were predominantly determined by the severity of liver injury and multi-organ dysfunction.

ClinicalTrials.gov identifier: NCT06187220, https://clinicaltrials.gov/study/.