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
{"title":"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","authors":"Emilie J. Lynch,&nbsp;Autumn Citta,&nbsp;Constance Alford,&nbsp;John A. Ligon,&nbsp;Mansi Dalal,&nbsp;Paul Castillo,&nbsp;Biljana Horn,&nbsp;Natalie Dotson,&nbsp;Giselle Moore-Higgs,&nbsp;Jordan Milner","doi":"10.1016/j.lrr.2024.100472","DOIUrl":null,"url":null,"abstract":"<div><p>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 (&lt;10 × 10<sup>3</sup>/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 &gt;100 × 10<sup>3</sup>/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100472"},"PeriodicalIF":0.7000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000621/pdfft?md5=3bf11fb769b63e12128df91f0b55035b&pid=1-s2.0-S2213048924000621-main.pdf","citationCount":"0","resultStr":"{\"title\":\"Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report\",\"authors\":\"Emilie J. Lynch,&nbsp;Autumn Citta,&nbsp;Constance Alford,&nbsp;John A. Ligon,&nbsp;Mansi Dalal,&nbsp;Paul Castillo,&nbsp;Biljana Horn,&nbsp;Natalie Dotson,&nbsp;Giselle Moore-Higgs,&nbsp;Jordan Milner\",\"doi\":\"10.1016/j.lrr.2024.100472\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>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 (&lt;10 × 10<sup>3</sup>/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 &gt;100 × 10<sup>3</sup>/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.</p></div>\",\"PeriodicalId\":38435,\"journal\":{\"name\":\"Leukemia Research Reports\",\"volume\":\"22 \",\"pages\":\"Article 100472\"},\"PeriodicalIF\":0.7000,\"publicationDate\":\"2024-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.sciencedirect.com/science/article/pii/S2213048924000621/pdfft?md5=3bf11fb769b63e12128df91f0b55035b&pid=1-s2.0-S2213048924000621-main.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Leukemia Research Reports\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2213048924000621\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Leukemia Research Reports","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2213048924000621","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"HEMATOLOGY","Score":null,"Total":0}
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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
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.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
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
期刊最新文献
Late-onset Familial Hemophagocytic Lymphohistiocytosis in a survivor of Hodgkin's Lymphoma Posterior reversible encephalopathy syndrome (PRES) and myeloma IDH2-mutated near ETP-ALL with aggressive leukemia cutis and brisk response to venetoclax and decitabine Genomic heterogeneity within B/T mixed phenotype acute leukemia in a context of an immunophenotype Avapritinib treatment of aggressive systemic mastocytosis with a novel KIT exon 17 mutation
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1