用达帕替尼对药物难治性小儿溃疡性结肠炎进行抢救性治疗

JPGN Reports Pub Date : 2024-04-01 DOI:10.1002/jpr3.12067
Maria Miller, Ashish S. Patel, Brad Pasternak
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引用次数: 0

摘要

小儿炎症性肠病(IBD)晚期治疗的获批方案有限。尽管 Janus 激酶 (JAK) 抑制剂已获准用于成人 IBD,但其对儿科人群的益处尚未明确。我们介绍了一位 13 岁的女性溃疡性结肠炎(UC)患者,她对多种疗法和泼尼松疗程均无效,最终对 JAK 抑制剂产生了反应。最初的治疗包括5-氨基水杨酸盐和硫唑嘌呤。由于症状持续存在,治疗方案改为阿达木单抗。重复结肠镜检查发现了胰腺炎,因此她转而接受了维多珠单抗治疗。使用维多珠单抗后,她两次因症状无法控制而住院,随后的结肠镜检查显示她仍有胰腺炎。因此,在调整为每月给药一次后,她转用了乌司替尼,但症状没有得到缓解。家属拒绝接受结肠切除术,选择用尽所有药物疗法。开始服用乌达替尼后,她的症状在一周内得到缓解,目前仍处于无类固醇缓解期。该病例说明了JAK抑制剂在结肠切除术前对广泛难治性小儿UC患者可能发挥的作用。
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Rescue therapy with upadacitinib in medically refractory pediatric ulcerative colitis
Approved options for advanced therapy in pediatric inflammatory bowel disease (IBD) are limited. Although Janus kinase (JAK) inhibitors are approved in adult IBD, their benefit in pediatric populations is not yet delineated. We present a 13‐year‐old female patient with ulcerative colitis (UC) refractory to numerous therapies and courses of prednisone that ultimately responded to a JAK inhibitor. Initial treatment consisted of 5‐aminosalicylate and azathioprine. This was changed to adalimumab due to persistent symptoms. Repeat colonoscopy revealed pancolitis, thus she was transitioned to vedolizumab. She was hospitalized twice for uncontrolled symptoms on vedolizumab and subsequent scope showed continued pancolitis. As a result, she transitioned to ustekinumab without symptomatic relief after adjusting to monthly dosing. The family declined colectomy, opting to exhaust all medical therapies. Upadacitinib was started and her symptoms resolved within 1 week, and she remains in steroid‐free remission. This case illustrates the possible role of JAK inhibitors in extensively refractory pediatric UC patients before colectomy.
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