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