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Eltrombopag in the Treatment of Immune Thrombocytopenia: Two-Center Experience from Istanbul
Indian Journal of Hematology and Blood Transfusion ( IF 0.7 ) Pub Date : 2021-07-05 , DOI: 10.1007/s12288-021-01462-5
Esma Evrim Dogan 1 , Esra Turan Erkek 2 , Tugrul Elverdi 3 , Sule Celik Kamaci 4 , Ugur Ciftci 4 , Naciye Demirel 1 , Demet Aydin 1 , Rafet Eren 1
Affiliation  

Immune Thrombocytopenia (ITP) is a disease caused by autoantibodies forming against platelets and T cell dysregulation and is characterized by platelet count falling below 100 × 109/L. Corticosteroids remain as the first-line of treatment, but in the light of recent developments, thrombopoietin receptor agonists are gradually replacing splenectomy in steroid-dependent or refractory patients. In this study, it was aimed to retrospectively evaluate the efficacy, safety and side-effect profile of eltrombopag treatment for chronic ITP. A total of 23 chronic ITP patients treated with eltrombopag from two health institutions in Istanbul were evaluated retrospectively. Overall response rate (partial or complete) was 87%, complete response rate was 78.3%, and the median time from treatment until reaching platelet counts above 50 × 109/L was 14 days (min–max: 4–126). Treatment was discontinued in four patients due to persistent response, two of these were still fully responsive. During treatment, one patient developed basal cell carcinoma, and another developed chronic myelomonocytic leukemia. Although its long-term side effects are not yet known, eltrombopag is a very effective treatment option in ITP and may provide favorable outcomes in patients.



中文翻译:

艾曲波帕治疗免疫性血小板减少症:来自伊斯坦布尔的双中心经验

免疫性血小板减少症 (ITP) 是一种由针对血小板的自身抗体形成和 T 细胞失调引起的疾病,其特征是血小板计数低于 100 × 10 9/L。皮质类固醇仍然是一线治疗,但鉴于最近的发展,血小板生成素受体激动剂正逐渐取代类固醇依赖或难治性患者的脾切除术。在本研究中,旨在回顾性评估艾曲波帕治疗慢性 ITP 的疗效、安全性和副作用情况。对来自伊斯坦布尔两家卫生机构的 23 名接受艾曲波帕治疗的慢性 ITP 患者进行了回顾性评估。总体缓解率(部分或完全)为 87%,完全缓解率为 78.3%,从治疗到血小板计数达到 50 × 10 9以上的中位时间/L 为 14 天(最小值-最大值:4-126)。由于持续反应,四名患者停止治疗,其中两名患者仍然完全反应。在治疗期间,一名患者发展为基底细胞癌,另一名患者发展为慢性粒单核细胞白血病。尽管其长期副作用尚不清楚,但艾曲波帕是 ITP 中非常有效的治疗选择,并可能为患者提供良好的结果。

更新日期:2021-07-06
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