Liver function changes in pediatric FMF: exploring disease dynamics and therapy impacts.

Familial Mediterranean fever (FMF) is the most common autoinflammatory disease, with colchicine being the standard treatment. Despite its effectiveness, colchicine has a narrow therapeutic index and potential adverse effects, including gastrointestinal discomfort, neutropenia, and hepatotoxicity. This study aimed to assess the impact of colchicine on liver function tests (LFTs), investigate potential causes of LFT elevations, and monitor the course of liver enzyme abnormalities in children with FMF. Children diagnosed with FMF were included if they had at least one LFT elevation ≥ 2 weeks after starting colchicine and were followed every 3 months. Data collected included demographics, clinical features, MEFV variants, disease severity (ISSF), colchicine dose, laboratory markers, and comorbidities. LFT elevations were categorized as mild, moderate, or severe. Fifty-four patients were included. Most had mild enzyme elevation (94.4%). LFT abnormalities appeared after a median of 46.5 months of colchicine. In 74%, enzymes normalized within a month. Recurrent/persistent LFT elevation occurred in 26 patients; hepatosteatosis and obesity were more common in this group. Forty-three patients were administered colchicine as monotherapy, while four patients were treated with a combination of colchicine and IL-1 inhibitors. Two patients with persistently high LFTs with colchicine during follow-up were switched to anti-IL-1 monotherapy. Five patients without attacks and with normal acute phase reactants were closely monitored without medication. Conclusion: Colchicine-associated LFT elevations were typically mild, transient, and manageable, and it appears to be safe for the liver in pediatric FMF, though ongoing monitoring and further long-term studies are warranted. What is Known: • Liver enzyme elevations are uncommon and typically mild in colchicine-treated pediatric FMF patients. • Elevations of LFT may reflect factors other than colchicine, like infections, obesity, or FMF flares. • A structured algorithm based on study findings may assist in managing LFT elevations.