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目前对接受CFTR调节剂治疗的囊性纤维化患者进行长期评估、监测和管理的策略。

Current strategies for the long-term assessment, monitoring, and management of cystic fibrosis patients treated with CFTR modulator therapy.

作者信息

Elborn J Stuart, Davies Jane, Mall Marcus A, Flume Patrick A, Plant Barry

机构信息

Queen's University of Belfast, Belfast, Northern Ireland, United Kingdom.

Imperial College London, Royal Brompton and Harefield NHS Foundation Trust, London, United Kingdom.

出版信息

J Cyst Fibros. 2017 Jan;16(1):163-164. doi: 10.1016/j.jcf.2016.11.009. Epub 2016 Dec 13.

DOI:10.1016/j.jcf.2016.11.009
PMID:27986495
Abstract

The content for this activity is based on the satellite symposium, "Current Strategies for the Long-term Assessment, Monitoring, and Management for Cystic Fibrosis Patients Treated with CFTR Modulator Therapy" that was presented at the 39th European Cystic Fibrosis Society Conference on June 10, 2016 (Online access: http://courses.elseviercme.com/ecfs2016e/619e). The emergence of novel targeted agents, that directly correct CFTR loss function alleles, has created new treatment opportunities for patients with cystic fibrosis with advanced disease. Knowledge of the role of these agents in the clinical setting is quickly evolving and will require physicians to stay acquainted with the latest data as well as evidence-based treatment guidelines in order to achieve optimized cystic fibrosis patient care. Ideally, after diagnosis, a personalized approach would be adapted and tailored to the patient through genome-informed medicine. However, due to the relative recentness of genomic-based therapeutics, physicians may have a limited knowledge base regarding these new treatment options and how to best incorporate these agents into patient management plans. Although cystic fibrosis is still largely regarded as a pediatric disease, the median survival for patients is 35years of age. Consequently, pediatric-to-adult cystic fibrosis care programs would allow suitable preparation time for this transition and develop a standardized group of self-care and management skills.

摘要

本次活动的内容基于卫星专题研讨会“CFTR调节剂治疗囊性纤维化患者的长期评估、监测和管理的当前策略”,该研讨会于2016年6月10日在第39届欧洲囊性纤维化协会会议上发表(在线访问:http://courses.elseviercme.com/ecfs2016e/619e)。新型靶向药物的出现,即直接纠正CFTR功能缺失等位基因,为患有晚期疾病的囊性纤维化患者创造了新的治疗机会。了解这些药物在临床环境中的作用正在迅速发展,这将要求医生熟悉最新数据以及循证治疗指南,以实现对囊性纤维化患者的优化护理。理想情况下,在诊断后,应通过基因组信息医学为患者采用个性化方法并进行量身定制。然而,由于基于基因组的治疗方法相对较新,医生可能对这些新的治疗选择以及如何最好地将这些药物纳入患者管理计划的知识基础有限。尽管囊性纤维化在很大程度上仍被视为一种儿科疾病,但患者的中位生存期为35岁。因此,儿科到成人的囊性纤维化护理计划将为这种转变留出适当的准备时间,并培养一组标准化的自我护理和管理技能。

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Current strategies for the long-term assessment, monitoring, and management of cystic fibrosis patients treated with CFTR modulator therapy.目前对接受CFTR调节剂治疗的囊性纤维化患者进行长期评估、监测和管理的策略。
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