Li Y, Ye L, Zhou K, Fan HH, Li JP, Xiong YZ, Yang Y, Peng GX, Yang WR, Zhao X, Jing LP, Zhang L, Zhang FK. Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia: A case report. World J Clin Cases 2024; 12(19): 3978-3984 [PMID: 38994307 DOI: 10.12998/wjcc.v12.i19.3978]
Corresponding Author of This Article
Feng-Kui Zhang, PhD, Professor, Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science, Peking Union Medical College, No. 288 Nanjing Road, Heping District, Tianjin 300020, China. fkzhang@ihcams.ac.cn
Research Domain of This Article
Hematology
Article-Type of This Article
Case Report
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
World J Clin Cases. Jul 6, 2024; 12(19): 3978-3984 Published online Jul 6, 2024. doi: 10.12998/wjcc.v12.i19.3978
Luspatercept enhances hemoglobin levels in a Chinese boy with congenital sideroblastic anemia: A case report
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
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, Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science, Peking Union Medical College, Tianjin 300020, China
Author contributions: Li Y analyzed the data and wrote the paper; Ye L, Zhou K, Fan HH, Li JP, Xiong YZ, Yang Y, and Peng GX contributed to data collection and revised the manuscript; Yang WR, Zhao X, Jing LP, Zhang L performed and supervised the research; Zhang FK designed the study, reviewed the data and revised the manuscript.
Supported byNational Natural Science Foundation of China, No. 81890992.
Informed consent statement: Informed written consent was obtained from the patient for publication of this report and any accompanying images.
Conflict-of-interest statement: All authors declare that they have no conflict of interest to report.
CARE Checklist (2016) statement: The authors have read the CARE Checklist (2016), and the manuscript was prepared and revised according to the CARE Checklist (2016).
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Feng-Kui Zhang, PhD, Professor, Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Science, Peking Union Medical College, No. 288 Nanjing Road, Heping District, Tianjin 300020, China. fkzhang@ihcams.ac.cn
Received: February 28, 2024 Revised: April 23, 2024 Accepted: May 10, 2024 Published online: July 6, 2024 Processing time: 122 Days and 8.9 Hours
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) × 1012/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.
Core Tip: 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. We present a case of luspatercept administered to enhance hemoglobin Levels.
