肠胆盐转运抑制剂治疗Alagille综合征瘙痒症的安慰剂对照随机试验。
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Shneider BL, Spino C, Kamath BM, Magee JC, Bass LM, Setchell KD, Miethke A, Molleston JP, Mack CL, sres RH, Murray KF, Loomes KM, Rosenthal P, Karpen SJ, Leung DH, Guthery SL, Thomas D, Sherker AH, Sokol RJ
肠胆盐转运抑制剂治疗Alagille综合征瘙痒症的安慰剂对照随机试验。
国际肝病杂志。2018年9月24日;2(10):1184-1198。doi: 10.1002 / hep4.1244。2018年10月
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30288474 (PubMed视图］
- 摘要
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医学上难治性的、严重的、胆汁淤积引起的Alagille综合征瘙痒可通过手术中断肠肝循环得到改善。这项多中心试验(NCT02057692)验证了肠胆汁酸转运抑制剂maralixibat可以类似地减少Alagille综合征患者的瘙痒的假设。37名患有Alagille综合征的儿童被随机分配到双盲给药组，分别为安慰剂、70微克/公斤/天、140微克/公斤或280微克/公斤/天，持续13周。瘙痒由护理人员(瘙痒报告结果仪[ItchRO])和临床医生报告(范围0-4[严重])评估。测定肝脏化学物质和血清胆汁酸。主要结果是与安慰剂相比，ItchRO从基线到第13周的变化。在主要疗效终点的先验首次分析中，接受140或280微克/千克/天与安慰剂的参与者之间的平均校正差异为-0.47(95%可信区间[CI]， -1.14, 0.20;P = 0.16)。70毫克/公斤/天和140毫克/公斤/天的剂量观察到有统计学意义的下降(平均调整后差异，-0.89;95% ci， -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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