阿伐曲波帕治疗一名异基因造血干细胞移植后患有 ALL 的儿童患者的严重难治性血小板减少症:病例报告

IF 0.7 Q4 HEMATOLOGY Leukemia Research Reports Pub Date : 2024-01-01 DOI:10.1016/j.lrr.2024.100472
Emilie J. Lynch, Autumn Citta, Constance Alford, John A. Ligon, Mansi Dalal, Paul Castillo, Biljana Horn, Natalie Dotson, Giselle Moore-Higgs, Jordan Milner
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引用次数: 0

摘要

接受异基因造血干细胞移植(alloHSCT)的患者有可能患上持续性血小板减少症。在这里,我们描述了阿伐曲波帕(一种血小板生成素受体激动剂)对一名异体造血干细胞移植后慢性、严重、输血依赖性血小板减少症(10 × 103/µL)儿科患者的治疗。在获得研究性新药同情使用授权后,阿伐曲波帕开始治疗,患者的血小板计数也随之增加。迄今为止,患者的血小板计数一直保持在 100 × 103/µL。未报告任何不良事件或药物毒性,他已恢复了骨髓造血干细胞移植前的活动。
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Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report

Patients who receive allogeneic hematopoietic stem cell transplantation (alloHSCT) are at risk for developing persistent thrombocytopenia. Here, we describe treatment with avatrombopag, a thrombopoietin receptor agonist, in a pediatric patient with chronic, severe, transfusion-dependent thrombocytopenia (<10 × 103/µL) post-alloHSCT that was persistent despite treatment with romiplostim, another thrombopoietin receptor agonist. Following the granting of a compassionate use investigational new drug authorization, avatrombopag treatment was initiated, and the patient's platelet count increased. To date, the patient has maintained a platelet count >100 × 103/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.

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来源期刊
Leukemia Research Reports
Leukemia Research Reports Medicine-Oncology
CiteScore
1.70
自引率
0.00%
发文量
70
审稿时长
23 weeks
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