患有难治性克罗恩病和 ATM 基因突变的儿童服用 Upadacitinib 后病情迅速缓解：病例报告

IF 1.6 Q3 MEDICINE, RESEARCH & EXPERIMENTAL

Current Therapeutic Research-clinical and Experimental Pub Date : 2024-01-01 DOI:10.1016/j.curtheres.2024.100756

Yan Liu MD, XiaoMei Song MD, LingYa Xiang MD, Wei Tan MD, Min Zou MD, Hong Guo MD

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引用次数: 0

摘要

由于严重的并发症和较高的生物治疗需求，儿科克罗恩病（PCD）的治疗面临挑战。从抗肿瘤坏死因子药物过渡到标签外疗法增加了复杂性。虽然奥达帕替尼在成人炎症性肠病和小儿特应性皮炎中显示出疗效和耐受性，但其在 PCD 中的应用数据却很有限。本病例报告描述了一名对英夫利西单抗、乌斯特库单抗、阿达木单抗、沙利度胺和泼尼松难治的CD患儿使用达帕替尼成功缓解病情的情况。值得注意的是，该患者携带有共济失调毛细血管扩张症突变（ATM）基因突变。这些发现为PCD的管理提供了宝贵的证据，并凸显了达达西尼在这一人群中的潜在益处。

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Rapid Remission With Upadacitinib in a Child With Refractory Crohn's Disease and ATM Mutation: A Case Report

Managing pediatric Crohn's disease (PCD) presents challenges due to severe complications and higher biologic therapy needs. Transitioning from anti–tumor necrosis factor agents to off-label therapies adds complexity. Although upadacitinib has demonstrated efficacy and tolerability in adult inflammatory bowel disease and pediatric atopic dermatitis, there are limited data for its application in PCD. This case report delineates successful remission with upadacitinib in a child with CD refractory to infliximab, ustekinumab, adalimumab, thalidomide, and prednisone. Notably, the patient carried an ataxia telangiectasia mutated (ATM) gene mutation. These findings provide valuable evidence for PCD management and highlight the potential benefits of upadacitinib in this population.

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来源期刊

Current Therapeutic Research-clinical and Experimental 医学-药学

CiteScore

3.50

自引率

0.00%

发文量

审稿时长

3 months

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