Efficacy and safety of avatrombopag in the treatment of chemotherapy-induced thrombocytopenia in children with acute lymphoblastic leukemia: a single-center retrospective study.

IF 3.4 3区 医学 Q2 HEMATOLOGY
Therapeutic Advances in Hematology Pub Date : 2024-12-06 eCollection Date: 2024-01-01 DOI:10.1177/20406207241304300
Huiyan Yang, Jingyu Gao, Yongsheng Ruan, Zhaokun Chen, Ruihan Fang, Lei Zhang, Zhibiao Wang, Tiantian Yi, Qian Zhang, Yang Luo, Libai Chen, Xuedong Wu
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引用次数: 0

Abstract

Background: Chemotherapy-induced thrombocytopenia (CIT) commonly exacerbates the difficulty of cancer treatment, increasing bleeding risks and potentially reducing chemotherapy dosage, ultimately impacting its efficacy. However, there are limited studies about avatrombopag application in acute lymphoblastic leukemia (ALL) CIT.

Objectives: We aimed to evaluate the efficacy and safety of avatrombopag in treating CIT patients diagnosed with ALL.

Design: This retrospective study, using propensity score matching, included 42 pairs of cases treated with and without avatrombopag (CAT: 54 cases, CAT+: 30 cases).

Methods: Data of CIT-ALL children were retrospectively collected. The primary endpoint was platelet count (PC) response rate on day 10 ± 2 (defined as an increase of PC to ⩾75 × 109/L with the exclusion of platelet transfusion). Secondary efficacy endpoints, safety endpoints, and factors that predict PC response were also analyzed.

Results: In the avatrombopag group, the PC response rate was prominently higher on day 10 ± 2 (89.1%) versus the control group (56.4%, p = 0.005). On day 10 ± 2, the difference in median PC change from baseline was predominantly distinct in the avatrombopag group compared to the control group (p = 0.001). In the avatrombopag group, platelet recovery to ⩾25 and ⩾50 × 109/L was faster (p = 0.001, p = 0.002), and quicker platelet reaching ⩾75 × 109/L and ⩾100 × 109/L was achieved (p = 0.023, p = 0.011). The avatrombopag group not only increased the nadir PC (p = 0.009) but also reduced the total platelet transfusion compared to the control group (p = 0.047). Only one case (2.4%) experienced bleeding events after medication. Nine cases of secondary thrombocythemia were noted without other adverse events. There was no difference in event-free survival between the two groups (p = 0.648). Drug administration was prediction factor for PC response.

Conclusion: Avatrombopag is a potentially safe and effective treatment option for CIT in pediatric ALL.

阿伐波帕治疗急性淋巴细胞白血病儿童化疗性血小板减少症的疗效和安全性:单中心回顾性研究
背景:化疗诱导的血小板减少症(CIT)通常会加剧癌症治疗的难度,增加出血风险,并可能减少化疗剂量,最终影响其疗效。然而,关于阿伐波帕在急性淋巴细胞白血病(ALL) CIT中的应用研究有限。目的:评价阿伐波帕治疗ALL诊断的CIT患者的有效性和安全性。设计:本回顾性研究采用倾向评分匹配法,纳入42对使用和不使用阿伐罗巴格的病例(CAT: 54例,CAT+: 30例)。方法:回顾性收集CIT-ALL患儿的资料。主要终点是第10±2天的血小板计数(PC)反应率(定义为PC增加到小于或等于75 × 109/L,排除血小板输注)。次要疗效终点、安全性终点和预测PC反应的因素也进行了分析。结果:阿伐隆巴格组患者在第10±2天的PC有效率(89.1%)显著高于对照组(56.4%,p = 0.005)。在第10±2天,与对照组相比,阿伐龙帕格组中位PC变化与基线的差异显著(p = 0.001)。在avatrombopag组中,血小板恢复到小于或等于25和大于或等于50 × 109/L更快(p = 0.001, p = 0.002),并且血小板更快达到大于或等于75 × 109/L和大于或等于100 × 109/L (p = 0.023, p = 0.011)。与对照组相比,阿伐波帕组不仅提高了最低血小板输注(p = 0.009),而且降低了血小板输注总量(p = 0.047)。用药后仅1例(2.4%)出现出血事件。9例继发性血小板增多症无其他不良事件。两组无事件生存率无差异(p = 0.648)。药物给药是PC反应的预测因素。结论:Avatrombopag是一种安全有效的治疗小儿ALL CIT的方法。
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来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
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