Alagille Syndrome
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Study finds predictors of survival, event-free survival in patients with ALGS treated with maralixibat

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Reduced pruritus symptoms, lowered bilirubin levels, and minimized serum bile acid levels (sBA) at week 48 after initiating treatment with maralixibat are associated with a lower risk of adverse events in patients with Alagille syndrome (ALGS), according to a study.

The results may potentially aid in identifying markers of disease progression and improving outcomes for maralixibat -treated patients with ALGS, the authors said.

The study analyzed data from 3 clinical trials of maralixibat involving 76 patients with a follow-up period of up to 6 years and a mean duration of maralixibat treatment of 4.7 years. The researchers assessed 46 potential predictors, including age, pruritus severity measured on the ItchRO[Obs] scale, biochemistries, platelet counts, and sBA.

Overall, 16 patients had events, including liver transplant (n = 10), decompensation (n = 3),  death (n = 2), and surgical biliary diversion (n = 1).

The study found that a clinically significant improvement of >1 point on the ItchRO(Obs) scale from baseline to week 48 was associated with a higher 6-year EFS (88% vs 57%; P = 0.005). Similarly, achieving a week 48 bilirubin level below 6.5 mg/dL (90% vs 43%; P < 0.0001) and a week 48 sBA level below 200 µmol/L (85% vs 49%; P = 0.001) were also predictive of improved 6-year EFS. These parameters were also found to be predictive of 6-year TFS.

Sokol RJ, Gonzales EM, Kamath BM, et al. Predictors of 6-year event-free survival in alagille syndrome patients treated with maralixibat, an ileal bile acid transporter inhibitor. Hepatology. 2023;doi: 10.1097/HEP.0000000000000502. Epub ahead of print. PMID: 37278241.