Leukemia Res:阿那格雷在真性红细胞增多症中用于血小板定向细胞减少:从大型多中心数据库中洞察实用性和安全性结果

2022-09-11 将军的九分裤 MedSci原创

阿那格雷 (ANA) 是一种血小板特异性细胞减少剂,多用于高危原发性血小板增多症的指南指导管理。尽管耐受性有限,ANA仍是 PV 患者选择性血小板控制的有效选择

阿那格雷 (ANA) 是一种血小板特异性细胞减少剂,多用于高危原发性血小板增多症的指南指导管理在真性红细胞增多症 (PV) 的背景下,ANA 偶尔用于临床实践,虽然不是指南指导的 PV 治疗的一部分,但 ANA 经常在临床实践中用作血小板降低剂,用于治疗伴有血小板增多症的 PV 患者。然而,这种方法的安全性和有效性尚未得到正式研究。此次报道的研究的目的是利用一个完善的、大型、多中心的 PV 患者数据库来回顾性描述接受 ANA 治疗的患者的特征和结果。

研究利用了来自美国 10 家机构的 527 名PV患者的既定数据库。对于初始数据库纳入,患者必须已被诊断为 2016 年世界卫生组织 (WHO) 标准所定义的 PV,并且已年满 18 岁。在 527 名 PV 患者的多中心队列中,48 名接受了 ANA(9 名因缺乏数据而被排除)。27 人 (69.2%) 有高危 PV,10 人 (25.6%) 曾有血栓形成,没有人有严重的血小板增多症、获得性血管性血友病和/或对羟基脲的耐药性。

图1:流程图

图 2:阿那格雷停药时间的 Kaplan-Meier 曲线。

虽然 ANA 有效地降低了中位血小板计数,但 43。5% 的患者在 ANA 停药时有未解决的血小板增多症。治疗中出现的不良事件——包括头痛、心悸和心律失常、恶心、呕吐和/或腹泻——导致 76.9% 的患者停用 ANA。此外,三名患者在 ANA 治疗的中位持续时间为 27.5 个月期间经历了动脉血栓形成。

表:导致ANA剂量减少或停用的诱发事件。

总体而言,目前的研究表明,尽管耐受性有限,ANA仍是 PV 患者选择性血小板控制的有效选择。在接受 ANA 治疗的 PV 患者队列中,血栓并发症(7.7%)和因毒性而停止治疗的发生率(76.9%)值得注意。目前的数据表明,将血小板目标作为 PV 的治疗终点缺乏明确的益处,相反,PV患者可以通过持续使用低剂量阿司匹林来降低血栓风险,从而靶使PV相关的血栓烷生物合成增加。在评估 ANA 在 PV 中的作用的大型前瞻性研究之前,研究人员建议考虑到伴有出血风险和症状性血小板增多症的 PV 患者,可使用血小板定向细胞减灭术。即使在这些情况下,他们也提倡使用非选择性细胞减灭剂,例如 HU 和 IFN-α

 

原始出处:

Rippel N, Tremblay D, Zubizarreta N, Podoltsev N, Gotlib J, Heaney M, Kuykendall A, O'Connell C, Shammo JM, Fleischman A, Kremyanskaya M, Hoffman R, Mesa R, Yacoub A, Mascarenhas J. Anagrelide for platelet-directed cytoreduction in polycythemia vera: Insights into utility and safety outcomes from a large multi-center database. Leuk Res. 2022 Aug;119:106903. doi: 10.1016/j.leukres.2022.106903. Epub 2022 Jun 16. PMID: 35717689.

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Lancet Haematol:鲁索替尼治疗真性红细胞增多症的5年随访结果

鲁索替尼可作为无脾肿大的控制欠佳的真性红细胞增多症患者的二线治疗选择