结缔组织疾病的个性化医疗:历史和未来的观点

Masataka Kuwana
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引用次数: 0

摘要

结缔组织病(CTD),以前被称为胶原蛋白病,是一组以风湿病症状、自身免疫和细胞外基质病理组织学变性为特征的疾病。更好的分类和疾病亚群,以及免疫抑制剂和分子靶向药物的引入,大大改善了患者的生活质量和预后。目前,在许多CTDs中提倡采用从治疗到目标的战略,同时强调早期诊断和干预。然而,由于患者的临床表现和治疗反应差异很大,仍然需要确定个体患者的适当管理。基于综合患者资料的个性化医疗方法可以通过减少治疗失败次数、提高药物安全性、减少与疾病及其治疗相关的并发症和器官损害来改善患者护理和长期结果。鉴别需要早期积极治疗的患者已经付出了巨大的努力,但目前对预测个体药物治疗反应的知识是有限的。基础科学和临床科学之间更好的协调是为CTD个体患者提供量身定制治疗方法的关键。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Personalized medicine for connective tissue disease: Historical and future perspectives

Connective tissue disease (CTD), previously termed collagen disease, is a group of disorders, characterized by rheumatic symptoms, autoimmunity, and degeneration of the pathohistology of the extracellular matrix. Better classification and disease subsetting, as well as introduction of immunosuppressants and molecular-targeting drugs, have much improved patients' quality of life and prognosis. The treat-to-target strategy in combination with emphasis on early diagnosis and intervention is now advocated for many CTDs. However, there remains a need to determine the appropriate management for individual patients, as clinical presentation and treatment response are highly variable among patients. A personalized medicine approach based on comprehensive patient profiles can improve patient care and long-term outcomes by decreasing the number of treatment failures, improving drug safety, and reducing the complications and organ damage associated with the disease and its treatment. Tremendous efforts have been made to identify patients who require early aggressive treatment, but the current knowledge on predicting the treatment response to individual drugs is limited. Better coordination between basic and clinical science is the key to providing a basis for tailored approaches for individual patients with CTD.

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