OC40 Impact of maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome
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Bibliographic record
Abstract
<h3></h3> Data in ALGS has primarily focused on pediatric patients, however adults with ALGS who survive with their native liver may require treatment for cholestasis and pruritus. We report on the efficacy and safety of MRX, an IBAT inhibitor, in young adults aged ≥16 years with ALGS transitioning to adult care. Participants received ≥1 dose of MRX ≥16 years of age within the MRX ALGS clinical development program. Pruritus [ItchRO(Obs)] and serum bile acids (sBA) were assessed at Baseline, before and after 16 years, and at study end. 14 individuals were included; 11 began treatment at <16 years of age and 3 patients began MRX ≥16 years. Baseline mean (SE) pruritus score was 2.5 (0.21), and significantly decreased to 0.8 prior to 16 years (delta = -1.7; p = 0.002); pruritus response was durable with no significant change before and after age 16 years (delta = -0.2; p = 0.2), or to end of therapy (delta = 0.2; p = 3) in individuals that started MRX <16 years old. Baseline mean sBA was 130 µmol/L, significantly decreased to 52 µmol/L (delta = -79; p = 0.03) prior to 16 years; no significant change before and after age 16 years (delta = -7; p = 0.3), or to end of therapy (delta = -3; p = 0.4) was observed. Three individuals that started MRX ≥16 years had improvements in pruritus from Baseline (delta = -2.8, -0.6, and -1.0). One patient had a large decrease in sBA (delta = -112 µmol/L) and two had small increases in sBA (delta = 8 and 11 µmol/L). MRX was generally well tolerated with the same safety profile previously reported. MRX was effective, durable, and well tolerated in ALGS patients ≥16 years, providing critical data for patients who transition to adulthood while on therapy.
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Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it