Marked improvement in pruritus after maralixibat in children with severe cholestasis

In children with severe cholestasis secondary to Alagille syndrome, treatment with maralixibat was associated with improvement in pruritus and quality of life (QoL), according to a new analysis.

The study analyzed data from 47 children with severe cholestasis secondary to Alagille Syndrome enrolled in 2 similar randomized placebo-controlled trials with subsequent extension phases investigating maralixibat in this population.

The primary outcomes were measures of pruritus (ItchRO[Obs]) and clinician scratch scale (CSS), both increasing in severity from 0 to 4 and QoL (Parent PedsQL and Multidimensional Fatigue Scale module [MFS] scaled 0-100 with increased QoL) at week 48 of the extension phase relative to the baseline of the placebo-controlled trials (week 13).

Statistically and clinically significant least square mean (95% CI) improvements in pruritus and QoL were observed at week 48:

-ItchRO[Obs]: -1.59 [-1.81, -1.36]
-CSS: -1.36 [-1.67, -1.05]
-PedsQL: +10.17 [4.48, 15.86]
-MFS:  +13.97 [7.85, 20.08]).

The study authors cautioned that due to the complex natural history of severe cholestasis in Alagille syndrome these findings are difficult to interpret.

Reference
Shneider BL, Spino CA, Kamath BM, et al; for ChiLDReN and UK IMAGO/IMAGINE Investigators. Impact of long-term administration of maralixibat on children with cholestasis secondary to Alagille syndrome. Hepatol Commun. 2022;doi: 10.1002/hep4.1992. Epub ahead of print. PMID: 35672955.

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