Luspatercept Enhances Hemoglobin Levels in a Chinese Boy With Congenital Sideroblastic Anemia: A Case Report

Authors

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

Publication Date

7-6-2024

Journal

World Journal of Clinical Cases

Abstract

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

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.

Keywords

Anemia, Sideroblastic anemia, Luspatercept, Congenital, Lifespan of mature red blood cells, Microcytic anemia, Case report

Comments

PMID: 38994307