Institute of Hematology "L. E A. Seràgnoli," Department of Experimental, Diagnostic and Specialty Medicine, University of Bologna, Italy.
Am J Hematol. 2016 Jun;91(4):E267-72. doi: 10.1002/ajh.24310.
In the last years, rituximab (RTX) and agonists of the thrombopoietin receptor (TPO-R) eltrombopag and romiplostim have provided new treatment options in persistent and chronic immune thrombocytopenia (ITP). Here, we analyzed the changes in therapeutic choices over time and their impact on clinical outcomes in a cohort of 557 ITP outpatients followed at the "L. and A. Seràgnoli" Institute of Hematology, Bologna, Italy, from 1980 to 2015. Overall 397 patients (71%) required front-line corticosteroids, mainly prednisone. Over the decades, splenectomy was delayed from second to third-line, but was steadily used in around 15-25% of patients refractory or relapsing after first-line treatment. Consensually, RTX and TPO-R agonists emerged as second and third-line therapy of choice, respectively. Splenectomy was associated with the best response rates and the lower incidences of relapse, while the relapse rate after RTX was comparable to that observed with corticosteroids and other immunosuppressive agents. The introduction of TPO-R agonists gave an alternative to the administration of immunosuppressive drugs and probably contributed to moderate the incidence of infectious complications that remained stable over the decades, despite an increasing use of RTX from the 2000s onwards. Overall responses were similar over time, with over 97% achieving a response in all time-periods. However, the cumulative risk of bleeding significantly decreased [14.3% (1980-89) vs. 7% (1990-99) vs. 5.6% (2000-09) vs. 0.2% (2010-15)] (P < 0.001), mainly thanks to the optimization of front-line corticosteroids therapy and to the wider availability of second and third-line therapies.
在过去的几年中，利妥昔单抗（RTX）和血小板生成素受体（TPO-R）激动剂艾曲波帕和罗米司亭为持续性和慢性免疫性血小板减少症（ITP）提供了新的治疗选择。在这里，我们分析了意大利博洛尼亚“L. 和 A. Seràgnoli”血液学研究所从 1980 年到 2015 年随访的 557 例 ITP 门诊患者随时间推移治疗选择的变化及其对临床结局的影响。总体而言，397 例患者（71%）需要一线皮质类固醇，主要是泼尼松。几十年来，脾切除术从二线推迟到三线，但在一线治疗后抵抗或复发的约 15-25%的患者中稳定使用。一致地，RTX 和 TPO-R 激动剂分别作为二线和三线治疗选择。脾切除术与最佳反应率和较低的复发率相关，而 RTX 后的复发率与皮质类固醇和其他免疫抑制剂相似。TPO-R 激动剂的引入为免疫抑制药物的治疗提供了替代方案，并且可能有助于减轻感染并发症的发生率，尽管从 21 世纪初开始 RTX 的使用不断增加，但几十年来感染并发症的发生率仍保持稳定。总体反应随时间相似，所有时间段的反应率均超过 97%。然而，出血的累积风险显著降低[14.3%（1980-89 年）比 7%（1990-99 年）比 5.6%（2000-09 年）比 0.2%（2010-15 年）]（P < 0.001），主要得益于一线皮质类固醇治疗的优化和二线和三线治疗的更广泛应用。
