艾曲波帕治疗重组人血小板生成素无效或复发的结缔组织病相关血小板减少症4例并文献复习

Efficacy of eltrombopag on recurrent connective tissue disease related thrombocytopenia patients who failed to respond to rhTPO or recombinant human thrombopoietin:a report of 4 cases and literature review

目的评价艾曲泊帕对标准治疗联合重组人血小板生成素(rhTPO)治疗无效或停用后复发的结缔组织病(CTD)相关血小板减少的患者疗效与安全性。方法回顾性分析2017年11月至2022年6月新疆维吾尔自治区人民医院风湿免疫科收治的艾曲波帕治疗rhTPO无效或复发的4例CTD相关血小板减少患者的临床资料,并对艾曲波帕治疗结缔组织病相关血小板的文献进行复习。结果4例患者使用艾曲波帕2周后血小板计数均可升至30×10^(9)/L以上,血小板计数恢复正常的中位时间为32(14,41)d,此后艾曲波帕逐渐减量仍能维持血小板计数正常,有助于减量糖皮质激素及免疫抑制剂,用药期间未见不良反应。结论艾曲波帕对rhTPO治疗无效或停用后复发的CTD相关血小板减少的患者安全有效。

Objective To evaluate the efficacy and safety of eltrombopag standard therapy in the treatment of patients with connective tissue disease(CTD)associated with thrombocytopenia who were non-responder to recombinant human thrombopoietin(rhTPO)or relapsed after discontinuation of rhTPO.Methods Retrospectively analysis of clinical data of 4 cases presented with CTD associated with thrombocytopenia who were non-responder to rhTPO or relapsed after discontinuation of rhTPO,and then treated with eltrombopag from November 2017 to June 2022.The literature were reviewed on eltrombopag in the treatment of CTD associated with thrombocytopenia.Results The platelet count of 4 patients increased to more than 30×10^(9)/L after 2 weeks administration of eltrombopag.The median time was 32(14,41)days for platelet counts returning to normal level.The dosage of eltrombopag was gradually reduced to maintain normal platelet count,which was helpful for tapering of glucocorticoid and other immunosuppressant.No adverse event was observed during the treatment.Conclusion Eltrombopag is safe and effective for CTD associated with thrombocytopenia,for patients who are non-responders to rhTPO or relapsed after discontinuation of rhTPO treatment in particular.