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Children with non-acetaminophen acute liver failure did not benefit from NAC

Intravenous N-acetylcysteine did not improve survival rates and increased the likelihood of liver transplant within 1 year of treatment among children with non-acetaminophen acute liver failure in a recent study.

In a double blind trial, researchers randomly assigned 184 patients with non-acetaminophen pediatric acute liver failure (non-APAP PALF) aged 17 years or younger to receive either 150 mg/kg N-acetylcysteine (NAC) (n=92) or placebo (n=92) daily for up to 7 days. Evaluated factors included survival rate at 1 year, liver transplant-free survival, incidence of transplant, the length of hospital and ICU stays, maximum hepatic encephalopathy (HE) score and incidence of organ system failure.

After 1 year, 82% of placebo patients and 73% of treated patients survived (P=.19). The survival rate among patient groups with HE grades of 0 to 1 was similar (65% of treated patients vs. 68% receiving placebo; P=.37).

The transplant-free survival rate was lower among patients aged 2 years and younger who received NAC (35% of patients compared with 53% receiving placebo, P=.03), with a particularly large difference among patients with an HE grade of 0 to 1 (25% of treated patients vs. 60% receiving placebo, P=.0493). Post-transplant survival rates did not differ significantly between treated patients and those receiving placebo. The cumulative probability of survival at 9 months was 88% in the treated group and 84% in the placebo group (P=.59).

No significant differences were observed between the placebo and treated groups regarding organ system failures, HE grades or the length of hospital or ICU stays. Incidence rates of adverse events were similar between groups (21 events among 19 treated patients and 17 events among 16 receiving placebo), with infection reported most commonly (11 treated patients and eight in the placebo group). Five treated patients experienced serious events, including respiratory distress, hypoglycemia, bradycardiac episode and Staphylococcus aureus bacteremia.

“NAC did not improve 1-year survival in children with non-APAP PALF,” the researchers concluded. “This study does not support the broad use of NAC in non-APAP PALF, and it emphasizes the importance of conducting prospective pediatric drug trials, regardless of results in adults.”