Song Andrew B, Al-Samkari Hanny
Department of Medicine, Massachusetts General Hospital, Boston, MA, USA.
Harvard Medical School, Boston, MA, USA.
Expert Rev Clin Immunol. 2022 Aug;18(8):783-791. doi: 10.1080/1744666X.2022.2098119. Epub 2022 Jul 10.
Multiple agents are available for the management of chronic immune thrombocytopenia (ITP), including thrombopoietin-receptor agonists (TPO-RAs), rituximab, and fostamatinib. Although TPO-RAs are often selected as treatments for chronic ITP, when choosing between the TPO-RAs, clinicians must balance safety profile, dosing restrictions, and method of administration incorporating patient preference.
We provide an overview of the thrombopoietin receptor agonists with a particular focus on avatrombopag, the newest agent in this class. In phase II and III clinical trials, avatrombopag was shown to offer durable improvement in platelet counts. We also include recent real-world evidence describing avatrombopag effectiveness in patients with poor response to prior treatments (including other TPO-RAs).
Compared with other TPO-RAs used to treat ITP, avatrombopag offers practical oral dosing with a single pill strength, does not require long-term dietary restrictions, and has no warning for hepatotoxicity. It is frequently effective after use of other TPO-RAs in ITP. The primary downside with avatrombopag use at present is the lack of longer-term safety data in ITP that presently exists for romiplostim and eltrombopag.
有多种药物可用于治疗慢性免疫性血小板减少症（ITP），包括血小板生成素受体激动剂（TPO-RAs）、利妥昔单抗和福斯他替尼。尽管TPO-RAs常被选作慢性ITP的治疗药物，但在选择TPO-RAs时，临床医生必须在安全性、给药限制以及结合患者偏好的给药方法之间进行权衡。
我们概述了血小板生成素受体激动剂，特别关注该类最新药物阿伐曲泊帕。在II期和III期临床试验中，阿伐曲泊帕显示出能持久提高血小板计数。我们还纳入了近期真实世界证据，描述了阿伐曲泊帕在对先前治疗（包括其他TPO-RAs）反应不佳的患者中的有效性。
与用于治疗ITP的其他TPO-RAs相比，阿伐曲泊帕提供实用的口服给药方式，单一药丸强度，无需长期饮食限制，且无肝毒性警告。在ITP中，使用其他TPO-RAs后使用阿伐曲泊帕通常有效。目前使用阿伐曲泊帕的主要缺点是缺乏ITP方面像罗米司亭和艾曲泊帕那样现有的长期安全性数据。
