Highlights
– Condition-specific weight-for-age and height-for-age charts for children with Alagille syndrome (ALGS) were generated from 1204 children using generalized additive models for location, scale and shape (GAMLSS), and differ substantially from US CDC charts.
– Estimated median adult heights on ALGS-specific charts are reduced compared with CDC curves (boys: ~171.5 cm vs 176 cm; girls: ~156.5 cm vs 163 cm).
– The ASSERT phase 3 randomized trial found that odevixibat (120 μg/kg/day) significantly improved caregiver-reported pruritus and reduced serum bile acids over 24 weeks versus placebo, with manageable safety profile.
Background and clinical need
Specialty calculators
For clinical reference; use professional judgment.
