Division of Hematology, Massachusetts General Hospital, Boston, Massachusetts, USA.
Harvard Medical School, Boston, Massachusetts, USA.
Br J Haematol. 2022 May;197(3):359-366. doi: 10.1111/bjh.18081. Epub 2022 Feb 18.
Patients with immune thrombocytopenia (ITP) may respond to one thrombopoietin receptor agonist (TPO-RA) but not another. Limited data are available describing outcomes in patients who switched from romiplostim or eltrombopag to avatrombopag, a newer oral TPO-RA. We performed a retrospective observational study of adults with ITP who switched from eltrombopag or romiplostim to avatrombopag at four US tertiary ITP referral centres. Forty-four patients were included, with a mean ITP duration of 8.3 years and a median (range) of four prior ITP treatments. On avatrombopag, 41/44 patients (93%) achieved a platelet response (≥50 × 10 /l) and 38/44 patients (86%) achieved a complete response (≥100 × 10 /l). In all patients, the median platelet count on eltrombopag or romiplostim was 45 × 10 /l vs 114 × 10 /l on avatrombopag (p < 0.0001); in patients switched for ineffectiveness of romiplostim/eltrombopag, it was 28 × 10 /l on romiplostim/eltrombopag vs 88 × 10 /l on avatrombopag (p = 0.025). Fifty-seven percent of patients receiving concomitant ITP medications before switching discontinued them after switching, including 63% of patients receiving chronic corticosteroids. In a heavily pretreated chronic ITP population, avatrombopag was effective following therapy with romiplostim or eltrombopag, with high response rates even in patients with inadequate response to a prior TPO-RA.
患有免疫性血小板减少症 (ITP) 的患者可能对一种血小板生成素受体激动剂 (TPO-RA) 有反应,但对另一种没有反应。目前仅有有限的数据描述了从罗米司亭或艾曲泊帕转换为阿伐曲泊帕(一种新型口服 TPO-RA)的患者的结局。我们对四家美国三级 ITP 转诊中心的 44 名从艾曲泊帕或罗米司亭转换为阿伐曲泊帕的 ITP 成人患者进行了回顾性观察研究。44 名患者中,平均 ITP 病程为 8.3 年,中位数(范围)为 4 种既往 ITP 治疗方案。在阿伐曲泊帕治疗中,44 名患者中有 41 名(93%)达到血小板反应(≥50×10 /l),38 名(86%)达到完全反应(≥100×10 /l)。所有患者的艾曲泊帕或罗米司亭的血小板计数中位数为 45×10 /l,而阿伐曲泊帕的血小板计数中位数为 114×10 /l(p<0.0001);在因罗米司亭/艾曲泊帕无效而转换的患者中,罗米司亭/艾曲泊帕的血小板计数中位数为 28×10 /l,而阿伐曲泊帕的血小板计数中位数为 88×10 /l(p=0.025)。在转换后,57%接受 ITP 合并药物治疗的患者停用了这些药物,包括 63%接受慢性皮质类固醇治疗的患者。在接受罗米司亭或艾曲泊帕治疗的慢性 ITP 患者中,阿伐曲泊帕治疗效果良好,即使对既往 TPO-RA 反应不足的患者也有很高的反应率。