阿伐曲泊帕用于成人免疫性血小板减少症：西班牙马德里的一项多中心真实世界观察性研究（AVAMAD研究）。
Avatrombopag in adults with immune thrombocytopenia: A multicentre real-life observational study in Madrid, Spain (AVAMAD study).
作者信息
Pascual-Izquierdo Cristina, Llacer-Ferrandis Mª Jose, de-la-Iglesia Almudena, Monsalvo-Saornil Silvia, Menor-Gómez María, Gil-Fernández Juan Jose, Chica-Gullon Esther, Álvarez-Román María Teresa, Perez-Segura Gloria, Zafra Denis, Ortuzar-Pasalodos Ariana, González-Gascón-Y-Marín Isabel Teresa, Moreno Gemma, Arquero-Portero Teresa, Moreno-Carbonell Marta, Revilla Nuria
机构信息
Hematología, Hospital General Universitario Gregorio Marañon, Instituto de Investigación Gregorio Marañon, Madrid, Spain.
Hematología, Hospital Universitario Puerta de Hierro, Madrid, Spain.
出版信息
Br J Haematol. 2025 Feb;206(2):652-656. doi: 10.1111/bjh.19975. Epub 2025 Jan 5.
Immune thrombocytopenia (ITP) is a rare acquired disorder where thrombopoietin-receptor agonists have become mainstays of ITP treatment. With the recent approval of avatrombopag (AVA), real-world studies are essential to evaluate its efficacy and safety. Our study of 66 adult ITP patients treated with AVA showed a high response rate. After starting AVA, 74.2% did not require rescue medications, with minimal adverse effects. Additionally, 56.0% of patients reduced other ITP medications, and all patients aged ≥65 years responded to AVA. Results should be confirmed in larger studies, but AVA appears to be an effective and safe treatment for ITP patients.
免疫性血小板减少症（ITP）是一种罕见的获得性疾病，血小板生成素受体激动剂已成为ITP治疗的主要手段。随着阿伐曲泊帕（AVA）最近获批，开展真实世界研究对于评估其疗效和安全性至关重要。我们对66例接受AVA治疗的成年ITP患者的研究显示出高缓解率。开始使用AVA后，74.2%的患者不需要救援药物，且不良反应极小。此外，56.0%的患者减少了其他ITP药物的使用，所有年龄≥65岁的患者对AVA均有反应。结果应在更大规模的研究中得到证实，但AVA似乎是ITP患者一种有效且安全的治疗方法。
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