Tu Xiaofang, Xue Ali, Wu Suye, Jin Mengmeng, Zhao Pu, Zhang Hao
Department of Hematology, The Third Affiliated Hospital of Wenzhou Medical University, Wenzhou, China.
Front Pharmacol. 2022 Jan 27;12:795884. doi: 10.3389/fphar.2021.795884. eCollection 2021.
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.
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。
