■血小板输注难治性(PTR)是血液学患者的一种危及生命且难以治疗的疾病。血小板生成素受体激动剂如avatrombopag促进血小板产生并调节免疫不耐受。然而,其在PTR中的应用尚未得到广泛研究。
我们的目的是比较难治性PTR患者的最佳可用疗法(BAT)和avatrombopag(Ava)治疗之间的血小板反应(PR)以及出血事件和死亡率。
■南方医院共纳入71例难治性PTR患者。静脉免疫球蛋白,类固醇,对BATs组30例患者进行人白细胞抗原匹配的血小板输注。Ava组包括41例患者。
■回顾性收集难治性PTR患者的数据。主要终点是PR(定义为连续7天没有血小板输注支持的情况下,血小板计数增加到50×109/L)。次要终点包括血小板输注独立率,累积血小板输注单位,世界卫生组织出血等级,不良事件,总生存期(OS),和无出血事件生存率(EFS)。
■在Ava组和BATs组中,有75.6%和13.3%的难治性PTR患者在3个月内达到PR。从第7天开始,Ava组的中位血小板计数显着升高。Ava组血小板输注独立率高于BATs组。Ava组的累积血小板输注单位中位数低于BATs组。与BATs组相比,Ava组的OS和无出血事件EFS率在3个月内有所改善。Cox比例风险回归分析显示,Ava治疗是OS和EFS的保护因素。由于不耐受,未观察到原发性疾病进展或avatrombopag终止。
■我们的研究表明,avatrombopag是难治性PTR患者的有效和安全的治疗选择。
Avatrombopag在血小板输注难治性PTR中是血液系统疾病患者的一个具有挑战性的临床问题,其增加了早期死亡和住院费用。血小板生成素受体激动剂在治疗血小板减少症方面显示出鼓舞人心的作用。然而,很少有研究关注这些药物在PTR患者中的应用。在这项研究中,我们调查了71例PTR患者,其中30例患者接受了最好的治疗,41例患者接受avatrombopag治疗。我们发现avatrombopag增加血小板反应率,减少血小板输注依赖和严重出血事件的发生,以及改善PTR患者的总体生存率和无事件生存率。Avatrombopag也表现出良好的耐受性和安全性。我们首次报道avatrombopag是一种有效和安全的PTR治疗方法,这也可能有助于扩大TPO-RA的临床应用。
UNASSIGNED: Platelet transfusion refractoriness (PTR) is a life-threatening and intractable condition in hematological patients. Thrombopoietin receptor agonists such as
avatrombopag promote platelet production and modulate immune intolerance. However, its application in PTR has not been extensively studied.
UNASSIGNED: We aimed to compare the platelet response (PR) as well as bleeding events and mortality rate between the best available therapies (BATs) and avatrombopag (Ava) treatments in refractory PTR patients.
UNASSIGNED: A total of 71 refractory PTR patients were enrolled at Nanfang Hospital. Intravenous immunoglobulin, steroids, and human leucocyte antigen-matched platelet transfusions were administered to 30 patients in the BATs group. The Ava group included 41 patients.
UNASSIGNED: Data of refractory PTR patients were retrospectively collected. The primary endpoint was PR (defined as an increase of platelet count to ⩾50 × 109/L without platelet transfusion support for 7 consecutive days). Secondary endpoints included platelet-transfusion independence rate, cumulative platelet transfusion units, World Health Organization bleeding grades, adverse events, overall survival (OS), and bleeding event-free survival (EFS).
UNASSIGNED: There were 75.6% and 13.3% refractory PTR patients who reached PR within 3 months in Ava and BATs groups. The median platelet counts were significantly higher in Ava group from day 7. Platelet-transfusion independence rate in Ava was higher than BATs group. The median cumulative platelet transfusion unit in Ava was lower than that of BATs group. The OS and bleeding events-free EFS rate of Ava group improved within 3 months as compared to BATs group. Cox proportional hazards regression analysis revealed that Ava therapy was a protective factor for the OS and EFS. No primary disease progression or termination of
avatrombopag was observed due to intolerability.
UNASSIGNED: Our study suggests that
avatrombopag is an effective and safe treatment option for refractory PTR patients.
Avatrombopag in platelet transfusion refractoriness PTR is a challenging clinical issue in patients with hematologic disorders which increases early death and hospitalization costs. Thrombopoietin receptor agonists have shown inspiring effects in treating thrombocytopenia. However, there are few studies focused on the application of these drugs in PTR patients. In this study, we investigated 71 patients with PTR in which 30 patients received the best available therapies, while 41 patients received avatrombopag treatment. We found that
avatrombopag increases platelet response rate, reduces platelet transfusions dependence and occurrence of severe bleeding events, as well as improves overall survival rate and event free survival in PTR patients.
Avatrombopag also exhibited good tolerance and safety. We reported for the first time that avatrombopag was an effective and safe treatment in PTR, which may also help to expand the clinical application of TPO-RAs.