马拉利西他可降低青少年Alagille综合征患者的血清胆汁酸并改善胆汁淤积性瘙痒。

IF 6 2区医学 Q1 GASTROENTEROLOGY & HEPATOLOGY

Liver International Pub Date : 2025-01-17 DOI:10.1111/liv.16201

Gideon Hirschfield, Shannon M. Vandriel, Douglas B. Mogul, Marshall Baek, Pamela Vig, Binita M. Kamath

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引用次数: 0

摘要

背景：Alagille综合征（ALGS）是一种多系统胆汁淤积症。马拉利西他被批准用于治疗ALGS患者的胆汁淤积性瘙痒，但成人数据有限。方法：在先前发表的三个马拉利西坦ALGS临床试验中，如果参与者在≥16岁时接受了≥2剂量的马拉利西坦，则纳入受试者。结果：11人开始治疗。结论：青少年期接受马力西他治疗的受试者瘙痒和sBA得到改善，并持续到青年期。

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Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome

Background

Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults.

Methods

Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical trials.

Results

Eleven initiated treatment < 16 years old (median age, 13.0) with a median follow-up of 4.1 years. Three participants began maralixibat at ≥ 16 years old, with a median follow-up of 3.8 years. Participants starting maralixibat at age < 16 had minimal-to-no itch (change from baseline [CFB]: −1.8; p = 0.002) persisting throughout the study. Serum bile acids (sBA) decreased (CFB: 29 μmol/L; p = 0.03), persisting throughout the study. Participants starting maralixibat ≥ 16 years old had pruritus improvements (CFB: −2.8, −0.6, −1.0). One had a large decrease in sBA (CFB: −112 μmol/L), and two had small increases (CFB: 8, 11 μmol/L). Maralixibat was well tolerated.

Conclusion

Participants receiving maralixibat in adolescence demonstrated improvements in pruritus and sBA, which persisted through young adulthood.

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来源期刊

Liver International 医学-胃肠肝病学

CiteScore

13.90

自引率

4.50%

发文量

348

审稿时长

2 months

期刊介绍： Liver International promotes all aspects of the science of hepatology from basic research to applied clinical studies. Providing an international forum for the publication of high-quality original research in hepatology, it is an essential resource for everyone working on normal and abnormal structure and function in the liver and its constituent cells, including clinicians and basic scientists involved in the multi-disciplinary field of hepatology. The journal welcomes articles from all fields of hepatology, which may be published as original articles, brief definitive reports, reviews, mini-reviews, images in hepatology and letters to the Editor.