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艾曲泊帕治疗难治性原发性免疫性血小板减少症的疗效与安全性:一项回顾性研究。

Efficacy and safety of eltrombopag in treatment-refractory primary immune thrombocytopenia: a retrospective study.

作者信息

Eser Ali, Toptas Tayfur, Kara Osman, Sezgin Aslihan, Noyan-Atalay Figen, Yilmaz Guven, Ozgumus Toluy, Pepedil-Tanrikulu Funda, Kaygusuz-Atagunduz Isik, Firatli-Tuglular Tulin

机构信息

aDepartment of Hematology, Marmara University HospitalbDepartment of Hematology, Baskent University HospitalcKartal Lutfi Kirdar Training and Research Hospital, Hematology Clinic, Istanbul, Turkey.

出版信息

Blood Coagul Fibrinolysis. 2016 Jan;27(1):47-52. doi: 10.1097/MBC.0000000000000380.

DOI:10.1097/MBC.0000000000000380
PMID:26258668
Abstract

Eltrombopag was used in patients with chronic primary immune thrombocytopenia (ITP) who did not tolerate or were refractory to two or more previous treatments. The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment. Data were extracted from medical chart records retrospectively. Platelet count of at least 50 000/μl at any time point during the treatment was defined as the 'response'. Median duration of eltrombopag treatment was 29 weeks (11-74). The number of patients who had a platelet count of at least 50 000/μl at any time point was 26 (83.9%). The response was achieved by the second week in most of the patients. Concomitant ITP medications were withdrawn in nine out of the 11 patients. Eltrombopag was discontinued in one patient due to sustained response despite discontinuation of the treatment. Age, sex, concomitant ITP treatments, and previous ITP treatment failures had no impact on the treatment response. The treatment was discontinued due to thrombosis in only four patients. Four patients experienced a minor bleeding event. Hepatotoxicity and all other adverse events were mild and manageable. Eltrombopag is effective, safe, and well tolerated in the long-term treatment of chronic ITP patients.

摘要

艾曲泊帕用于对两种或更多种先前治疗不耐受或难治的慢性原发性免疫性血小板减少症(ITP)患者。该研究的主要目的是确定长期使用艾曲泊帕治疗的疗效和安全性。数据通过回顾性地从病历记录中提取。治疗期间任何时间点血小板计数至少为50000/μl被定义为“反应”。艾曲泊帕治疗的中位持续时间为29周(11 - 74周)。在任何时间点血小板计数至少为50000/μl的患者有26例(83.9%)。大多数患者在第二周时达到反应。11例患者中有9例停用了伴随的ITP药物。1例患者尽管停止治疗仍有持续反应,因此停用了艾曲泊帕。年龄、性别、伴随的ITP治疗以及先前的ITP治疗失败对治疗反应均无影响。仅4例患者因血栓形成而停止治疗。4例患者发生轻微出血事件。肝毒性和所有其他不良事件均较轻且可控。艾曲泊帕在慢性ITP患者的长期治疗中有效、安全且耐受性良好。

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