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成人生理性免疫性血小板减少症患者中血小板生成素受体激动剂的替代应用:来自意大利血液中心的回顾性合作调查。

Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers.

机构信息

Hematology and Oncology Department, Niguarda Cancer Center, ASST Grande Ospedale Metropolitano Niguarda, Milan.

Hematology and Transplant Unit, ASST San Gerardo Hospital, Monza.

出版信息

Am J Hematol. 2018 Jan;93(1):58-64. doi: 10.1002/ajh.24935. Epub 2017 Nov 9.

DOI:10.1002/ajh.24935
PMID:28983953
Abstract

Sequential use of the TPO-RAs romiplostim and eltrombopag in ITP patients failing either agent was retrospectively evaluated to assess efficacy and impact of clinical characteristics on outcome. Patients were grouped into 5 categories: efficacy issues: 1 TPO-RA failure; loss of response; non-efficacy issues: platelet fluctuations; patient's preference; adverse event development. Either one TPO-RA sequence was analyzed at 3 month and at last follow-up. 106/546 patients on TPO-RA underwent switch and 65% achieved, regained or maintained a short- term response independent of switch sequence, gender or age; lower response rates were associated with lines of previous therapy; disease duration lowers probability to respond. Clinically, patients switched for efficacy issue did not differ from those switched for non-efficacy issues. Response was achieved/regained in 57.8% of patients switched for efficacy issues, the lowest response rates were observed in non-responders to 1 TPO-RA; 80% of patients switched for non-efficacy issues maintained a response. Platelet fluctuation resolved in 44.4%. Of the 49 patients evaluable for long-term outcome, 27 were in response on therapy; 16 discontinued the TPO-RA for reasons other than efficacy, while only 6 were non responders. We confirm the efficacy of TPO-RA switch; once achieved, response to the 2 TPO-RA seems durable.

摘要

回顾性评估了在任一 TPO-RA 治疗失败的 ITP 患者中序贯使用 romiplostim 和 eltrombopag 的疗效,并分析了临床特征对疗效的影响。患者分为 5 组:疗效问题:1 种 TPO-RA 治疗失败;应答丧失;非疗效问题:血小板波动;患者意愿;不良反应发生。分析了任一 TPO-RA 序贯治疗 3 个月和末次随访时的情况。在接受 TPO-RA 治疗的 546 例患者中,有 106 例进行了药物转换,无论转换顺序如何,65%的患者在短期随访时均获得、恢复或维持了应答,且与转换顺序、性别或年龄无关;较低的应答率与既往治疗线数相关;疾病持续时间降低了应答的可能性。临床来看,因疗效问题而转换药物的患者与因非疗效问题而转换药物的患者无差异。因疗效问题而转换药物的患者中,57.8%的患者获得/恢复应答,应答率最低的是对 1 种 TPO-RA 无应答的患者;因非疗效问题而转换药物的患者中,80%的患者维持应答。血小板波动缓解率为 44.4%。在可评估长期结局的 49 例患者中,27 例患者仍有应答;16 例因除疗效以外的原因停止 TPO-RA 治疗,而仅有 6 例患者无应答。我们证实了 TPO-RA 转换的疗效;一旦获得应答,对这两种 TPO-RA 的应答似乎是持久的。

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