艾曲波帕治疗儿童异基因造血细胞移植后血小板减少症:单中心经验。

Eltrombopag for treatment of thrombocytopenia after allogeneic hematopoietic cell transplantation in children: Single-centre experience.

机构信息

Pediatric BMT Unit, Istanbul Medipol Universitesi, Istanbul, Turkey.

Pediatric Oncology, Istanbul Medipol Universitesi, Istanbul, Turkey.

出版信息

Pediatr Transplant. 2021 Aug;25(5):e13962. doi: 10.1111/petr.13962. Epub 2021 Jan 16.

Abstract

Delayed recovery of thrombocytopenia is a well-known complication after allogeneic HSCT. Eltrombopag (ELT), a thrombopoietin receptor agonist (TRAs), induces platelet maturation and release. Mostly conducted in adults, some of the previous studies have shown that ELT seems to enhance platelet recovery for post-allogeneic HSCT thrombocytopenia, appears efficacious, and offers transfusion independence. To evaluate the safety and efficacy of ELT in pediatric patients with prolonged isolated thrombocytopenia (PIT) or secondary failure of platelet recovery (SFPR) after alloHSCT. Retrospective analysis of childhood patients who received treatment with ELT for persistent thrombocytopenia after alloHSCT between May 2016 and August 2019. We evaluated the safety and efficacy of ELT in 18 childhood patients with PIT or SFPR after alloHSCT. Eltrombopag (50 mg/d) treatment was started in all patients, above 6 years of age and 20 kg weight, who had thrombocytopenia despite neutrophil engraftment on the 30th day of HSCT. Our objective was to decrease the need for platelet transfusion and have a platelet count of more than 50 000/µL. The overall response rate was 77.7%. The median time to achieve a platelet level above 30 000/µL and 50 000/µL was 21 and 44 days, respectively. In four patients, platelet count never reached 30 000/mm . In two patients, the treatment was discontinued due to grade 3 hepatotoxicity. Our study supports the efficacy and relative safety of ELT use for the treatment of PIT and SFPR seen after alloHSCT in children.

摘要

血小板减少症延迟恢复是异基因 HSCT 后的一种已知并发症。血小板生成素受体激动剂(TRAs)艾曲泊帕(ELT)可诱导血小板成熟和释放。虽然之前的一些研究主要在成人中进行,但已经表明 ELT 似乎可以增强异基因 HSCT 后血小板减少症的血小板恢复,具有疗效,并可实现输血独立性。评估 ELT 在儿童异基因 HSCT 后持续性孤立性血小板减少症(PIT)或血小板恢复继发失败(SFPR)患者中的安全性和有效性。对 2016 年 5 月至 2019 年 8 月期间接受 ELT 治疗以治疗异基因 HSCT 后持续性血小板减少症的儿童患者进行回顾性分析。我们评估了 ELT 在 18 例异基因 HSCT 后发生 PIT 或 SFPR 的儿童患者中的安全性和有效性。所有患者均开始使用 ELT 治疗,年龄超过 6 岁且体重超过 20kg,在 HSCT 第 30 天中性粒细胞植入后仍存在血小板减少症。我们的目标是减少血小板输注的需求,并使血小板计数超过 50000/µL。总体缓解率为 77.7%。达到血小板计数超过 30000/µL 和 50000/µL 的中位时间分别为 21 天和 44 天。有 4 名患者的血小板计数从未达到 30000/mm。有 2 名患者因 3 级肝毒性而停止治疗。我们的研究支持在儿童中使用 ELT 治疗异基因 HSCT 后出现的 PIT 和 SFPR 的疗效和相对安全性。

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