Alagille Syndrome
Conference Roundup

Catch-up height, weight seen after maralixibat treatment in patients with ALGS

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Patients with Alagille syndrome (ALGS) treated with the ileal bile acid transporter inhibitor (IBATi), maralixibat, experienced catch-up height and weight, with the greatest catch-up in height observed in those with the greatest height disadvantage at baseline, according to a presentation at ESPGHAN 2022.

Data from 34 participants from 3 clinical trials of maralixibat for treatment of cholestatic pruritus in ALGS that had height and weight data at baseline and week 204 follow-up were included.

Patients had a baseline mean (SD) age of 6.7 (3.8) years, height Z-score of -1.66 (1.17), and weight Z-score of -1.46 (0.95). At week 204, the mean height Z-score increased to -1.29 (1.03).

There was an increase from -3.1 (0.71) at baseline to -2.38 (0.82) at week 204 amongst patients within the lowest baseline quartile height Z-scores. There was a significant correlation between the change in height and baseline height. In addition, there was a significant correlation between change in weight and baseline weight, with greater catch-up weight gain seen in patients with lower baseline weight Z-scores.

“Increased catch-up height was also seen in patients that achieved lower serum bile acid with maralixibat, suggesting an improvement in bile acid homeostasis may be a factor,” the authors concluded.

Kamath BM, et al. Maralixibat improves growth in patients with Alagille syndrome: a 4-year analysis. Presented at ESPGHAN 2022.