Abstract：
Alagille syndrome (ALGS) is a rare, debilitating inheritable disease that is associated with refractory pruritus due to chronic cholestasis. The following systemic review and meta-analysis presents the latest evidence for ileal bile acid transport (IBAT) blockers in AGLS patients in order to improve their efficacy. This study adhered to PRISMA 2020 Statement guidelines. A systematic search of PubMed/MEDLINE, Web of Science, Scopus, and the Cochrane library was conducted from inception until 23 October 2022. A combination of the following keywords was used: Alagille syndrome, therapeutics, treatment, therapy. Meta-analytical outcomes included effect directions of end-line changes in serum bile acids (sBAs), Itch Scale scores (ItchRO), Multidimensional Fatigue Scale scores, pediatric quality of life (QL), alanine aminotransferase (ALT), and total bilirubin. A total of 94 patients across four trials were enrolled and received maralixibat, odevixibat, or a placebo. There was a significant reduction in ItchRO scores by 1.8 points, as well as in sBAs by 75.8 μmol/L. Both the Multidimensional Fatigue Scale and Pediatric QL scale were also improved by 11.4 and 8.3 points, respectively. However, ALT levels were raised by 40 U/L. The efficacy of IBAT inhibitors across current trials was noted. Future trials may focus on the optimization of dosing regimens, considering gastrointestinal side effects and drug-induced ALT elevation in AGLS patients.
摘要：
Alagille综合征(ALGS)是一种罕见的,与慢性胆汁淤积引起的难治性瘙痒相关的衰弱遗传性疾病。以下系统回顾和荟萃分析提供了AGLS患者回肠胆汁酸转运（IBAT）阻滞剂的最新证据，以提高其疗效。这项研究遵循了PRISMA2020声明指南。对PubMed/MEDLINE的系统搜索，WebofScience,Scopus,Cochrane图书馆从成立到2022年10月23日进行。使用了以下关键词的组合：Alagille综合征，治疗学,治疗,治疗。荟萃分析结果包括血清胆汁酸（sBA）终线变化的影响方向，瘙痒量表评分（ItchRO），多维疲劳量表评分，儿科生活质量（QL），丙氨酸氨基转移酶（ALT），和总胆红素.在四项试验中，共有94名患者被纳入并接受了maralixibat，奥德维希巴特,或者安慰剂.ItchRO分数显著下降1.8分,以及在sBA中的75.8μmol/L。多维疲劳量表和小儿QL量表也分别提高了11.4和8.3分,分别。然而,ALT水平提高40U/L注意到目前试验中IBAT抑制剂的功效。未来的试验可能集中在给药方案的优化上，考虑到AGLS患者的胃肠道副作用和药物引起的ALT升高。
