Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia:A case report  

作　　者：Yuan Li Lei Ye Kang Zhou Hui-Hui Fan Jian-Ping Li You-Zhen Xiong Yang Yang Guang-Xin Peng Wen-Rui Yang Xin Zhao Li-Ping Jing Li Zhang Feng-Kui Zhang 

机构地区：[1]Anemia Therapeutic Center,Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Science,Peking Union Medical College,Tianjin 300020,China

出　　处：《World Journal of Clinical Cases》2024年第19期3978-3984,共7页世界临床病例杂志

基　　金：National Natural Science Foundation of China,No.81890992.

摘　　要：BACKGROUND Congenital sideroblastic anemia(CSA)is a rare and heterogeneous group of genetic disorders.Conventional treatment include pyridoxine(vitamin B6)and allogeneic hematopoietic stem cell transplantation(allo-HSCT),and can alleviate anemia in the majority of cases.Nevertheless,some CSA cases remain unresponsive to pyridoxine or are unable to undergo allo-HSCT.Novel management approaches is necessary to be developed.To explore the response of luspatercept in treating congenital sideroblastic anemia.CASE SUMMARY We share our experience in luspatercept in a 4-year-old male patient with CSA.Luspatercept was administered subcutaneously at doses of 1.0 mg/kg/dose to 1.25 mg/kg/dose every 3 wk,three consecutive doses,evaluating the hematological response.Luspatercept leading to a significant improvement in the patient's anemia.The median hemoglobin during the overall treatment with three doses of luspatercept was 90(75-101)g/L,the median absolute reticulocyte count was 0.0593(0.0277-0.1030)×10^(12)/L,the median serum ferritin was 304.3(234.4-399)ng/mL,and the median lifespan of mature red blood cells was 80(57-92)days.Notably,no adverse reactions,such as headaches,dizziness,vomiting,joint pain,or back pain,were observed during the treatment period.CONCLUSION We believe that luspatercept might emerge as a viable therapeutic option for the maintenance treatment of CSA or as a bridging treatment option before hematopoietic stem cell transplantation.

关 键 词：ANEMIA Sideroblastic anemia Luspatercept CONGENITAL Lifespan of mature red blood cells Microcytic anemia Case report 

分 类 号：R733.71[医药卫生—肿瘤] R737.9[医药卫生—临床医学]