IRCCS Azienda Ospedaliero-Universitaria Di Bologna, Istituto Di Ematologia "Seràgnoli, Bologna, Italy.
Dipartimento Di Medicina Specialistica, Diagnostica E Sperimentale, Università Di Bologna, Bologna, Italy.
Ann Hematol. 2022 May;101(5):963-978. doi: 10.1007/s00277-022-04786-y. Epub 2022 Feb 24.
Chronic primary immune thrombocytopenia (ITP) can today benefit from multiple therapeutic approaches with proven clinical efficacy, including rituximab, thrombopoietin receptor agonists (TPO-RA), and splenectomy. However, some ITP patients are unresponsive to multiple lines of therapy with prolonged and severe thrombocytopenia. The diagnosis of refractory ITP is mainly performed by exclusion of other disorders and is based on the clinician's expertise. However, it significantly increases the risk of drug-related toxicity and of bleedings, including life-threatening events. The management of refractory ITP remains a major clinical challenge. Here, we provide an overview of the currently available treatment options, and we discuss the emerging rationale of new therapeutic approaches and their strategic combination. Particularly, combination strategies may target multiple pathogenetic mechanisms and trigger additive or synergistic effects. A series of best practices arising both from published studies and from real-life clinical experience is also included, aiming to optimize the management of refractory ITP.
慢性原发性免疫性血小板减少症 (ITP) 如今可受益于多种经临床证实有效的治疗方法，包括利妥昔单抗、血小板生成素受体激动剂 (TPO-RA) 和脾切除术。然而，一些 ITP 患者对多种治疗方案无反应，表现为持续且严重的血小板减少。难治性 ITP 的诊断主要通过排除其他疾病来进行，基于临床医生的专业知识。然而，这显著增加了药物相关毒性和出血（包括危及生命的事件）的风险。难治性 ITP 的管理仍然是一个主要的临床挑战。在这里，我们提供了目前可用的治疗选择的概述，并讨论了新的治疗方法的出现及其战略组合的基本原理。特别是，联合策略可能针对多种发病机制并触发相加或协同作用。还包括一系列源自已发表研究和真实临床经验的最佳实践，旨在优化难治性 ITP 的管理。
