Lynch Emilie J, Citta Autumn, Alford Constance, Ligon John A, Dalal Mansi, Castillo Paul, Horn Biljana, Dotson Natalie, Moore-Higgs Giselle, Milner Jordan
Division of Pediatric Hematology, Oncology, Stem Cell Transplantation, University of Florida, Shands Children's Hospital, Gainesville, FL, USA.
Leuk Res Rep. 2024 Jul 24;22:100472. doi: 10.1016/j.lrr.2024.100472. eCollection 2024.
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/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/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.
接受异基因造血干细胞移植（alloHSCT）的患者有发生持续性血小板减少症的风险。在此，我们描述了一名儿科患者在alloHSCT后出现慢性、严重、依赖输血的血小板减少症（<10×10⁹/µL），尽管使用了另一种血小板生成素受体激动剂罗米司亭进行治疗，但血小板减少症仍持续存在，随后使用血小板生成素受体激动剂阿伐曲泊帕进行治疗的情况。在获得同情用药研究性新药授权后，开始使用阿伐曲泊帕治疗，患者的血小板计数增加。迄今为止，该患者的血小板计数维持在>100×10⁹/µL。未报告不良事件或药物毒性，并且他已恢复alloHSCT前的活动。
