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病例报告:阿伐曲泊帕成功治疗两例抗程序性死亡受体1(PD-1)抗体诱导的获得性无巨核细胞性血小板减少症

Case Report: Successful Avatrombopag Treatment for Two Cases of Anti-PD-1 Antibody-Induced Acquired Amegakaryocytic Thrombocytopenia.

作者信息

Tu Xiaofang, Xue Ali, Wu Suye, Jin Mengmeng, Zhao Pu, Zhang Hao

机构信息

Department of Hematology, The Third Affiliated Hospital of Wenzhou Medical University, Wenzhou, China.

出版信息

Front Pharmacol. 2022 Jan 27;12:795884. doi: 10.3389/fphar.2021.795884. eCollection 2021.

Abstract

Anti-PD-1/PD-L1 immunotherapy has achieved impressive responses in multiple types of malignancies in recent years. However, immune-related adverse events (irAEs) occur and limit their continuous clinical use. Among these irAEs, acquired amegakaryocytic thrombocytopenia (AAT) is rare but often clinically serious, life-threatening and refractory to multiple treatment approaches. We reported for the first time the successful treatment of avatrombopag in two cases of anti-PD1 antibody-induced AAT (in particular, one case had progressed to aplastic anemia), which was refractory or intolerant to glucocorticoids, ciclosporin, intravenous immunoglobulin (IVIG), recombinant human thrombopoietin (rh-TPO) and even TPO receptor agonist (TPO-RA) eltrombopag. To date, the two cases manifested as normal platelet counts and are independent of transfusion. Anti-PD1 antibody-induced AAT occurs with low frequency but is often serious and difficult to manage, for which this study proposed vatrombopag as a potential curative and safe approach.

摘要

近年来,抗PD-1/PD-L1免疫疗法在多种恶性肿瘤中取得了令人瞩目的疗效。然而,免疫相关不良事件(irAEs)的发生限制了其在临床上的持续应用。在这些irAEs中,获得性无巨核细胞性血小板减少症(AAT)虽罕见,但临床上往往较为严重,危及生命,且对多种治疗方法均难起效。我们首次报道了阿伐曲泊帕成功治疗两例抗PD1抗体诱导的AAT(特别是其中一例已进展为再生障碍性贫血),这两例患者对糖皮质激素、环孢素、静脉注射免疫球蛋白(IVIG)、重组人血小板生成素(rh-TPO)甚至血小板生成素受体激动剂(TPO-RA)艾曲泊帕均难治或不耐受。迄今为止,这两例患者血小板计数恢复正常,且无需输血。抗PD1抗体诱导的AAT发生率较低,但往往病情严重且难以处理,本研究提出阿伐曲泊帕是一种潜在的有效且安全的治疗方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1d2c/8830913/ed61dca68db8/fphar-12-795884-g001.jpg

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