Have splenectomy rate and main outcomes of ITP changed after the introduction of new treatments? A monocentric study in the outpatient setting during 35 years
Have splenectomy rate and main outcomes of ITP changed after the introduction of new treatments? A monocentric study in the outpatient setting during 35 years
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DOI：
10.1002/ajh.24310
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发表时间：
2016-04-01
影响因子：
12.8
通讯作者：
Vianelli, Nicola
中科院分区：
文献类型：
--
作者：
Palandri, Francesca;Polverelli, Nicola;Vianelli, Nicola
在过去的几年里，利妥昔单抗(RTX)和血小板生成素受体(TPO-R)激动剂eltrombopogg和romiplostin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1980年至2015年在意大利博洛尼亚L.和A.Seragnoli血液学研究所跟踪的557名ITP门诊患者的治疗选择随时间的变化及其对临床结果的影响。总体而言，397名患者(71%)需要一线皮质类固醇，主要是强的松。几十年来，脾切除术从二线延迟到三线，但在一线治疗后出现难治性或复发的患者中，约有15%-25%的患者稳定使用。一致同意，RTX和TPO-R激动剂分别成为二线和三线治疗的选择。脾切除与最好的应答率和较低的复发率有关，而RTX后的复发率与皮质类固醇和其他免疫抑制剂的复发率相当。TPO-R激动剂的引入为免疫抑制药物的应用提供了另一种选择，并可能有助于缓和感染并发症的发生率，尽管从本世纪头十年开始RTX的使用越来越多，但感染并发症的发生率在几十年内保持稳定。随着时间的推移，总体反应是相似的，超过97%的人在所有时间段都得到了反应。然而，累积出血风险显著降低[14.3%(1980-89)比7%(1990-99)比5.6%(2000-09)比0.2%(2010-15)](P
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. Seragnoli 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