Department of Haematology, Hospital Universitario de Burgos, Burgos, Spain.
Department of Haematology/Oncology, University Children's Hospital Basel, Basel, Switzerland.
Br J Haematol. 2023 Oct;203(1):101-111. doi: 10.1111/bjh.19082.
The only way to prevent immune thrombocytopenia (ITP) from becoming refractory would be to restore tolerance to platelets at an early phase of the disease. Numerous immune alterations probably accumulate in chronic ITP; thus, the chances of cure decrease significantly with time. Currently, sustained remission off treatment (SROT) is a clinical definition describing patients who can discontinue their ITP treatment without risk and maintain a state of remission. Different treatment strategies are presently being evaluated with the goal of attaining SROT, mostly combining drugs targeting the innate and/or the adaptive immune system, the inflammation state, so as increasing the platelet load. In this sense, thrombopoietin receptor agonists (TPO-RAs) have shown promising results if used as upfront treatment. TPO-RAs seem to exhibit immunomodulation and immune tolerance properties, increasing not only the platelet antigen mass but also increasing the transforming growth factor-β concentration, and stimulating regulatory T and B lymphocytes. However, more immunological studies are needed to establish accurate molecular alterations in ITP that are potentially reversed with treatments.
预防免疫性血小板减少症 (ITP) 变为难治性的唯一方法是在疾病的早期阶段恢复对血小板的耐受性。在慢性 ITP 中,可能会积聚大量的免疫改变;因此,随着时间的推移,治愈的机会显著降低。目前,停药持续缓解(SROT)是一种临床定义,描述了可以在没有风险的情况下停止 ITP 治疗并保持缓解状态的患者。目前正在评估不同的治疗策略,以实现 SROT,主要是将靶向固有和/或适应性免疫系统、炎症状态的药物联合起来,从而增加血小板负荷。从这个意义上说,如果作为一线治疗,促血小板生成素受体激动剂 (TPO-RA) 已显示出有希望的结果。TPO-RA 似乎具有免疫调节和免疫耐受特性,不仅增加血小板抗原质量,还增加转化生长因子-β浓度,并刺激调节性 T 和 B 淋巴细胞。然而,需要更多的免疫学研究来确定 ITP 中潜在可逆转治疗的准确分子改变。