AASLD 2019 — Pediatric Alagille syndrome: long-term maralixibat reduces itch, xanthoma


  • Brandon May
  • Conference Reports
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Takeaway

  • Long-term treatment with maralixibat is associated with durable control of serum bile acid (sBA), pruritus, and xanthomas in pediatric patients with Alagille syndrome.

Why this matters

  • Standard therapies currently used to manage pruritus, among other symptoms associated with Alagille syndrome, have varying efficacy profiles, and no prospective trials have assessed their benefit in this condition.
  • The FDA has granted Breakthrough Therapy Designation for maralixibat in patients ≥1 year of age with Alagille syndrome.

Study design

  • Findings were from an analysis of the long-term extension phase of the ICONIC trial, comprising children who reached 48 weeks of treatment with maralixibat (n=23).
  • Participants received up to 400 μg/kg twice-daily maralixibat if sBA was greater than the upper limit of normal and/or if pruritus persisted.
  • The median duration of therapy was 44.5 (range, 42.1-51.7) months in 15 patients who entered the long-term extension.
  • Funding: None reported.

Key results

  • The mean change from baseline to last data point in sBA was –158.5 μmol/L (95% CI, −260.1 to –56.9; P=.0048).
  • There were also significant changes in the pruritus score (–2.3; 95% CI, –2.8 to –1.8; P<.0001 xanthoma score ci to p=".0453)," and scratch>

Limitations

  • Small sample size and lack of a comparator group.