Agarwal Rajiv, Burton James, Gallieni Maurizio, Kalantar-Zadeh Kamyar, Mayer Gert, Pollock Carol, Szepietowski Jacek C
Richard L. Roudebush VA Medical Center and Indiana University, Indianapolis, IN, USA.
Department of Cardiovascular Sciences, University Hospitals of Leicester NHS Trust, Leicester, UK.
Clin Kidney J. 2022 Aug 24;16(1):30-40. doi: 10.1093/ckj/sfac187. eCollection 2023 Jan.
Since the breakthrough of kidney replacement therapy, increases in life expectancy for patients with end-stage kidney disease have been limited. However, patients have become increasingly vocal that, although mortality and life expectancy matter to them, the quality of their life, and particularly the relief of symptoms associated with their treatment, are in many cases more important. The majority of dialysis-associated symptoms and adverse effects do not currently have any approved treatments in this patient population, with the few treatments that are available used off-label, frequently without proven efficacy, yet still potentially adding further adverse effects to patients' current symptom burden. This article will illustrate how understanding the pathophysiology of a single, particularly burdensome symptom of dialysis (chronic kidney disease-associated pruritus) resulted in the design, development and regulatory approval of a treatment for that symptom. The pathway described here can be applied to other symptoms associated with dialysis, meaning that if we cannot add years to patients' lives, we can at least add life to their remaining years.
自肾脏替代疗法取得突破以来,终末期肾病患者的预期寿命增长有限。然而,患者越来越直言不讳地表示,尽管死亡率和预期寿命对他们很重要,但他们的生活质量,尤其是与治疗相关症状的缓解,在很多情况下更为重要。目前,大多数与透析相关的症状和不良反应在该患者群体中尚无任何获批的治疗方法,少数可用的治疗方法属于超适应症使用,往往没有经过证实的疗效,但仍可能给患者当前的症状负担增加更多不良反应。本文将说明,对透析中一种特别令人困扰的单一症状(慢性肾病相关性瘙痒)的病理生理学理解如何促成了针对该症状的治疗方法的设计、开发和监管批准。这里描述的途径可应用于与透析相关的其他症状,这意味着如果我们无法延长患者的生命年限,至少可以提高他们余年的生活质量。