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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.

DOI:10.1111/bjh.19975
PMID:39756394
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11829131/
Abstract

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.

摘要

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。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3b96/11829131/c22c3fc9882b/BJH-206-652-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3b96/11829131/c22c3fc9882b/BJH-206-652-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3b96/11829131/c22c3fc9882b/BJH-206-652-g001.jpg

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本文引用的文献

1
Avatrombopag for adults with early versus chronic immune thrombocytopenia.阿伐曲泊帕治疗成人早期与慢性免疫性血小板减少症。
Am J Hematol. 2024 Feb;99(2):155-162. doi: 10.1002/ajh.27080. Epub 2023 Dec 8.
2
Recommendations for the Clinical Approach to Immune Thrombocytopenia: Spanish ITP Working Group (GEPTI).免疫性血小板减少症临床处理建议:西班牙免疫性血小板减少症工作组(GEPTI)
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Adults with immune thrombocytopenia who switched to avatrombopag following prior treatment with eltrombopag or romiplostim: A multicentre US study.
曾接受艾曲泊帕或罗米司亭治疗的成人免疫性血小板减少症患者换用阿伐曲泊帕治疗:一项多中心美国研究。
Br J Haematol. 2022 May;197(3):359-366. doi: 10.1111/bjh.18081. Epub 2022 Feb 18.
4
Thrombopoietin Receptor Agonists (TPO-RAs): Drug Class Considerations for Pharmacists.促血小板生成素受体激动剂(TPO-RAs):药师在药物类别方面的考虑因素。
Drugs. 2021 Jul;81(11):1285-1305. doi: 10.1007/s40265-021-01553-7. Epub 2021 Jun 23.
5
Real-world use of thrombopoietin receptor agonists in older patients with primary immune thrombocytopenia.血小板生成素受体激动剂在老年原发性免疫性血小板减少症患者中的真实世界应用。
Blood. 2021 Aug 19;138(7):571-583. doi: 10.1182/blood.2021010735.
6
Updated international consensus report on the investigation and management of primary immune thrombocytopenia.更新的原发性免疫性血小板减少症的调查和管理国际共识报告。
Blood Adv. 2019 Nov 26;3(22):3780-3817. doi: 10.1182/bloodadvances.2019000812.
7
Deciphering predictive factors for choice of thrombopoietin receptor agonist, treatment free responses, and thrombotic events in immune thrombocytopenia.解析免疫性血小板减少症中选择血小板生成素受体激动剂、无治疗反应和血栓事件的预测因素。
Sci Rep. 2019 Nov 13;9(1):16680. doi: 10.1038/s41598-019-53209-y.
8
Thrombopoietin receptor agonists: ten years later.血小板生成素受体激动剂:十年后。
Haematologica. 2019 Jun;104(6):1112-1123. doi: 10.3324/haematol.2018.212845. Epub 2019 May 9.
9
Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia.三期随机研究avatrombopag,一种新型的促血小板生成素受体激动剂,用于治疗慢性免疫性血小板减少症。
Br J Haematol. 2018 Nov;183(3):479-490. doi: 10.1111/bjh.15573. Epub 2018 Sep 7.