安慰剂对照的随机试验肠道胆汁盐运输抑制剂的瘙痒Alagille综合症。

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人物包括提单,Spino C, Kamath BM,麦基JC,低音LM, Setchell KD, Miethke, Molleston JP,麦克CL, Squires RH,默里KF,织机公里,罗森塔尔P, Karpen SJ,梁DH, Guthery SL,托马斯•D Sherker啊,索科尔RJ

安慰剂对照的随机试验肠道胆汁盐运输抑制剂的瘙痒Alagille综合症。

乙醇Commun。2018年9月24日;2 (10):1184 - 1198。doi: 10.1002 / hep4.1244。eCollection 2018年10月。

PubMed ID

30288474 (在PubMed

]

文摘

医学耐火材料,严重,cholestasis-induced瘙痒在Alagille综合症可能提高了手术肝肠循环的中断。这个多中心试验(NCT02057692)测试的假设肠道胆汁酸运输抑制剂maralixibat同样会减少瘙痒Alagille综合症。37 Alagille综合症患儿被随机双盲安慰剂管理局,70,140,或280的maralixibat microg /公斤/天13周。瘙痒是由照顾者评估(itch-reported结果仪器[ItchRO])和临床医生报告(严重)4 - 0(范围)。肝脏化学反应和血清胆汁酸测定。主要结果是改变从基线到13周ItchRO相对于安慰剂。首先在先验分析的主要功效端点,意味着参与者调整差别接收140年或280年microg /公斤/天和安慰剂是-0.47(95%可信区间[CI], -1.14, 0.20;P = 0.16)。显著降低观察与剂量的70年和140年microg /公斤/天(平均调整差异,-0.89;95%可信区间,-1.70,-0.08; P = 0.032; and mean adjusted difference, -0.91; 95% CI, -1.62, -0.19; P = 0.014) but not 280 microg/kg/day (mean adjusted difference, -0.04; 95% CI, -0.94, 0.86; P = 0.44) or all doses combined (mean adjusted difference, -0.61; 95% CI, -1.24, 0.20; P = 0.055). A 1-point reduction in pruritus was more common in maralixibat-treated versus placebo-treated participants (caregiver ItchRO, 65% versus 25%; P = 0.06; clinician score, 76% versus 25%; P = 0.01). There were no significant changes in liver chemistries or bile acids relative to placebo. Adverse and serious adverse events were similar between maralixibat and placebo. Conclusion: Although the prespecified primary analyses of ItchRO were not all statistically significant, the data suggest that maralixibat is safe and may reduce pruritus in Alagille syndrome.

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药物

Maralixibat