Department of Haematology, Hammersmith Hospital, Imperial College London, London, UK.
Platelet Disorder Support Association, Cleveland, Ohio, USA.
Am J Hematol. 2021 Feb 1;96(2):188-198. doi: 10.1002/ajh.26045. Epub 2020 Dec 19.
Immune thrombocytopenia (ITP) is now well-known to reduce patients' health-related quality of life. However, data describing which signs and symptoms patients and physicians perceive as having the greatest impact are limited, as is understanding the full effects of ITP treatments. I-WISh (ITP World Impact Survey) was an exploratory, cross-sectional survey designed to establish the multifaceted impact of ITP, and its treatments, on patients' lives. It focused on perceptions of 1507 patients and 472 physicians from 13 countries regarding diagnostic pathway, frequency and severity of signs and symptoms, and treatment use. Twenty-two percent of patients experienced delayed diagnosis (caused by several factors), 73% of whom felt anxious as a result. Patients rated fatigue among the most frequent, severe symptom associated with ITP at diagnosis (58% most frequent; 73% most severe), although physicians assigned it lower priority (30%). Fatigue was one of the few symptoms persisting at survey completion (50% and 65%, respectively) and was the top symptom patients wanted resolved (46%). Participating physicians were experienced at treating ITP, thereby recognizing the need to limit corticosteroid use to newly-diagnosed or first-relapse patients and espoused increased use of thrombopoietin receptor agonists and anti-CD20 after relapse in patients with persistent/chronic disease. Patient and physicians were largely aligned on diagnosis, symptoms, and treatment use. I-WISh demonstrated that patients and physicians largely align on overall ITP symptom burden, with certain differences, for example, fatigue. Understanding the emotional and clinical toll of ITP on the patient will facilitate shared decision-management, setting and establishment of treatment goals and disease stage-appropriate treatment selection.
免疫性血小板减少症(ITP)现已广为人知,可降低患者的健康相关生活质量。然而,描述患者和医生认为对生活影响最大的症状和体征的数据有限,对 ITP 治疗的全部影响的了解也有限。I-WISh(ITP 全球影响调查)是一项探索性的、横断面调查,旨在确定 ITP 及其治疗对患者生活的多方面影响。它侧重于来自 13 个国家的 1507 名患者和 472 名医生对诊断途径、症状的频率和严重程度以及治疗方法的看法。22%的患者经历了延迟诊断(由多种因素引起),其中 73%的患者因此感到焦虑。患者将疲劳评为与 ITP 相关的最常见和最严重的症状之一(58%为最常见症状;73%为最严重症状),尽管医生将其优先级较低(30%)。疲劳是调查完成时持续存在的少数症状之一(分别为 50%和 65%),也是患者最希望解决的首要症状(46%)。参与的医生在治疗 ITP 方面经验丰富,因此认识到需要将皮质类固醇的使用限制在新诊断或首次复发的患者,并主张在持续性/慢性疾病患者复发后增加使用血小板生成素受体激动剂和抗 CD20。患者和医生在诊断、症状和治疗方法上基本一致。I-WISh 表明,患者和医生在整体 ITP 症状负担方面基本一致,但存在一些差异,例如疲劳。了解 ITP 对患者的情绪和临床影响将有助于共同决策管理、设定和确立治疗目标以及选择适合疾病阶段的治疗方法。