利用丹麦真实世界数据对克罗恩病进行药物再利用。

IF 2.3 Q2 MEDICINE, GENERAL & INTERNAL Pragmatic and Observational Research Pub Date : 2024-02-21 eCollection Date: 2024-01-01 DOI:10.2147/POR.S444569
Saeed Shakibfar, Kristine H Allin, Tine Jess, Maria Antonietta Barbieri, Vera Battini, Eva Simoncic, Julien Kirchgesner, Trond Ulven, Maurizio Sessa
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引用次数: 0

摘要

目的:利用电子医疗记录(EHR)进行药物再利用是一种很有前景的替代方法,它将现有药物再利用于新的治疗适应症,尤其是对缺乏有效疗法的患者。肠纤维化是克罗恩病(CD)的一种严重并发症,它带来了巨大的挑战,在没有药物治疗的情况下增加了发病率和死亡率。本文的重点是通过全人群真实世界数据和人工智能(AI)方法,确定与克罗恩病患者纤维化风险升高或降低相关的药物:对丹麦电子病历中 1996 年至 2019 年诊断为 CD 的 65 岁及以上患者进行了长达 24 年的随访。主要结果是是否需要进行特定的外科手术,即直肠结肠切除术加回肠造口术和回盲部切除术,以此作为肠纤维化的替代指标。研究通过机器学习驱动的生存分析,探讨了与增加或降低研究结果风险相关的药物:在9179名CD患者中,1029人(11.2%)接受了手术治疗,主要为男性(58.5%),平均年龄为76岁。相比之下,有 10 种药物与这些病症的手术风险降低有关:这项研究的重点是利用丹麦电子病历和先进的统计方法,重新确定现有药物的用途,以防止 CD 患者接受与肠纤维化相关的手术。研究结果为这种疾病的潜在治疗方法提供了宝贵的见解,满足了尚未满足的关键医疗需求。还需要进一步的研究和临床试验来验证这些再利用药物在预防 CD 患者肠纤维化相关手术方面的有效性。
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Drug Repurposing in Crohn's Disease Using Danish Real-World Data.

Aim: Drug repurposing, utilizing electronic healthcare records (EHRs), offers a promising alternative by repurposing existing drugs for new therapeutic indications, especially for patients lacking effective therapies. Intestinal fibrosis, a severe complication of Crohn's disease (CD), poses significant challenges, increasing morbidity and mortality without available pharmacological treatments. This article focuses on identifying medications associated with an elevated or reduced risk of fibrosis in CD patients through a population-wide real-world data and artificial intelligence (AI) approach.

Methods: Patients aged 65 or older with a diagnosis of CD from 1996 to 2019 in the Danish EHRs were followed for up to 24 years. The primary outcome was the need of specific surgical procedures, namely proctocolectomy with ileostomy and ileocecal resection as proxies of intestinal fibrosis. The study explored drugs linked to an increased or reduced risk of the study outcome through machine-learning driven survival analysis.

Results: Among the 9179 CD patients, 1029 (11.2%) underwent surgery, primarily men (58.5%), with a mean age of 76 years, 10 drugs were linked to an elevated risk of surgery for proctocolectomy with ileostomy and ileocecal resection. In contrast, 10 drugs were associated with a reduced risk of undergoing surgery for these conditions.

Conclusion: This study focuses on repurposing existing drugs to prevent surgery related to intestinal fibrosis in CD patients, using Danish EHRs and advanced statistical methods. The findings offer valuable insights into potential treatments for this condition, addressing a critical unmet medical need. Further research and clinical trials are warranted to validate the effectiveness of these repurposed drugs in preventing surgery related to intestinal fibrosis in CD patients.

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Pragmatic and Observational Research
Pragmatic and Observational Research MEDICINE, GENERAL & INTERNAL-
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期刊介绍: Pragmatic and Observational Research is an international, peer-reviewed, open-access journal that publishes data from studies designed to closely reflect medical interventions in real-world clinical practice, providing insights beyond classical randomized controlled trials (RCTs). While RCTs maximize internal validity for cause-and-effect relationships, they often represent only specific patient groups. This journal aims to complement such studies by providing data that better mirrors real-world patients and the usage of medicines, thus informing guidelines and enhancing the applicability of research findings across diverse patient populations encountered in everyday clinical practice.
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