Garcia Amy, Hsu Evelyn, Lin Henry C
From the Department of Pediatrics, Essentia Health, Duluth, MN.
Department of Pediatrics, University of Washington School of Medicine, Seattle, WA.
JPGN Rep. 2023 Jul 17;4(3):e335. doi: 10.1097/PG9.0000000000000335. eCollection 2023 Aug.
Intractable pruritus is one of the most prominent and debilitating features of Alagille syndrome. Maralixibat is the first US Food and Drug Administration-approved drug for the treatment of cholestatic pruritus in children with Alagille syndrome aged 3 months and older. Clinical trials of maralixibat have reported follow-up to 4 years and reported a ≥1-pt reduction using the Itch-Reported Outcome (Observer) (ItchRO[Obs]) instrument (0-4 scale), as this decrease was previously defined as a clinically meaningful improvement in pruritus; participants in clinical trials were expected to be maintained on stable doses of antipruritic agents. We report on a patient with 3 notable features: (1) complete resolution of her pruritus; (2) durability of this response for over 7 years; and (3) ability to discontinue all other antipruritic medications.
顽固性瘙痒是阿拉吉耶综合征最突出且使人衰弱的特征之一。玛拉利昔巴特是美国食品药品监督管理局批准的首个用于治疗3个月及以上阿拉吉耶综合征儿童胆汁淤积性瘙痒的药物。玛拉利昔巴特的临床试验报告了长达4年的随访情况，并使用瘙痒报告结果（观察者）（ItchRO[Obs]）工具（0 - 4级）报告了瘙痒评分降低≥1分，因为此前将这种降低定义为瘙痒临床上有意义的改善；临床试验中的参与者预计要维持稳定剂量的止痒药。我们报告了一名具有3个显著特征的患者：（1）瘙痒完全缓解；（2）这种反应持续了7年以上；（3）能够停用所有其他止痒药物。
