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, Autumn Citta, Constance Alford, John A. Ligon, Mansi Dalal, Paul Castillo, Biljana Horn, Natalie Dotson, Giselle Moore-Higgs, 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 (<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 >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, Autumn Citta, Constance Alford, John A. Ligon, Mansi Dalal, Paul Castillo, Biljana Horn, Natalie Dotson, Giselle Moore-Higgs, 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 (<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 >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}
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.