INSELSPITAL, Department of Haematology and Central Haematology Laboratory, Bern University Hospital, Bern, Switzerland.
Division of Oncology, Hematology and Transfusion Medicine, Kantonsspital Aarau, Aarau, Switzerland.
PLoS One. 2022 Apr 21;17(4):e0267342. doi: 10.1371/journal.pone.0267342. eCollection 2022.
Thrombopoietin receptor agonists (TPO-RAs) are approved for immune thrombocytopenia (ITP), but their impact on health-related quality of life (HRQoL) remains poorly investigated in clinical practice. This observational study aimed to gain insight into real-world patient-reported experiences of the burden of ITP and TPO-RAs.
An online questionnaire of closed questions was used to collect views of patients with primary ITP from Switzerland, Austria, and Belgium, between September 2018 and April 2020.
Of 46 patients who completed the questionnaire (total cohort), 41% were receiving TPO-RAs. A numerically higher proportion of patients reported being free from symptoms at the time of the questionnaire (54%) than at diagnosis (24%), irrespective of treatment type. Bleeding, the most frequently reported symptom at diagnosis (59%), was reduced at the time of the questionnaire (7%). Conversely, fatigue was reported by approximately 40% of patients at both diagnosis and the time of the questionnaire. Having a normal life and their disease under control was reported by 83% and 76%, respectively, but 41% were worried/anxious about their condition. Nearly 50% reported that ITP impaired their engagement in hobbies/sport or energy levels and 63% reported no impact on employment. When stratified by TPO-RA use, bleeding was better controlled in those receiving TPO-RAs than not (0% vs 11%). A numerically lower proportion receiving TPO-RAs than not reported worry/anxiety about their condition (16% vs 59%) and shifting from full-time to part-time employment (11% vs 22%). Similar proportions were satisfied with their therapy whether they were receiving TPO-RAs or not (89% vs 85%).
Many factors affect HRQoL in patients with ITP. Of patients receiving TPO-RAs, none experienced bleeding at the time of the questionnaire; they also showed a more positive perspective for some outcomes than those not using TPO-RAs. However, fatigue was not reduced by any treatment.
血小板生成素受体激动剂（TPO-RA）已被批准用于治疗免疫性血小板减少症（ITP），但其在临床实践中对健康相关生活质量（HRQoL）的影响仍研究甚少。本观察性研究旨在深入了解 ITP 患者的真实体验及其 TPO-RA 治疗负担。
本研究于 2018 年 9 月至 2020 年 4 月期间，采用封闭式在线问卷，收集来自瑞士、奥地利和比利时的原发性 ITP 患者的观点。
在完成问卷的 46 名患者（总队列）中，41%正在接受 TPO-RA 治疗。无论治疗类型如何，报告在问卷时无任何症状（54%）的患者比例均高于诊断时（24%）。在诊断时最常报告的症状（59%）出血，在问卷时有所减少（7%）。相反，疲劳在诊断时（约 40%）和问卷时（约 40%）均有报告。分别有 83%和 76%的患者报告生活正常且疾病得到控制，但 41%的患者对其病情感到担忧/焦虑。近 50%的患者报告 ITP 影响了他们的业余爱好/运动或精力水平，63%的患者报告对其就业没有影响。根据 TPO-RA 的使用情况分层，接受 TPO-RA 治疗的患者出血控制情况优于未接受 TPO-RA 治疗的患者（0% vs 11%）。接受 TPO-RA 治疗的患者报告对其病情感到担忧/焦虑的比例（16% vs 59%）和从全职工作转为兼职工作的比例（11% vs 22%）均低于未接受 TPO-RA 治疗的患者。无论是否接受 TPO-RA 治疗，患者对其治疗的满意度相似（89% vs 85%）。
许多因素会影响 ITP 患者的 HRQoL。接受 TPO-RA 治疗的患者在问卷时均无出血；与未接受 TPO-RA 治疗的患者相比，他们对某些结果的看法更为积极。然而，任何治疗都不能减轻疲劳。
