Dogan Esma Evrim, Turan Erkek Esra, Elverdi Tugrul, Celik Kamaci Sule, Ciftci Ugur, Demirel Naciye, Aydin Demet, Eren Rafet
Department of Hematology, Medical Science University Prof. Dr. Cemil Tascioglu City Hospital, Darulaceze Cad. No:27 Sisli, Istanbul, Turkey.
Department of Hematology, Medical Science University Kartal Lutfu Kirdar City Hospital, Istanbul, Turkey.
Indian J Hematol Blood Transfus. 2022 Apr;38(2):327-332. doi: 10.1007/s12288-021-01462-5. Epub 2021 Jul 5.
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 × 10/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 × 10/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.
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100×10⁹/L。皮质类固醇仍然是一线治疗药物，但鉴于最近的进展，血小板生成素受体激动剂正在逐渐取代脾切除术，用于治疗依赖类固醇或难治性患者。在本研究中，旨在回顾性评估艾曲泊帕治疗慢性ITP的疗效、安全性和副作用情况。对来自伊斯坦布尔两家医疗机构的23例接受艾曲泊帕治疗的慢性ITP患者进行了回顾性评估。总体缓解率（部分或完全缓解）为87%，完全缓解率为78.3%，从治疗至血小板计数达到50×10⁹/L以上的中位时间为14天（最小值-最大值：4-126天）。4例患者因持续缓解而停药，其中2例仍完全缓解。治疗期间，1例患者发生基底细胞癌，另1例发生慢性粒单核细胞白血病。尽管其长期副作用尚不清楚，但艾曲泊帕是ITP一种非常有效的治疗选择，可能为患者带来良好的治疗效果。
