Journal Article

Effects of Thalidomide on Metabolism and Lifespan of Red Blood Cell in Patients With β-Thalassemia Major: A Post Hoc Analysis of a Randomized Controlled Trial

Mo, Yongquan, Wu, Zhengwei, Yang, Huanju, Chen, Shuying, Wang, Guizhen, He, Yanfang, Zhu, Weijian, Luo, Tianying, Han, Sichong, Zhou, Meiguang, Zhou, Guangbiao, Huang, Minjie, Chen, Jiangming, Dang, Lujie, Li, Jinyan, Liao, Yangdong, Huang, Lan, Cai, Ning, Xu, Jianquan, He, Sheng, Lu, Qiyang

2025

Overview

•This study was a post hoc analysis of a randomized controlled trial with 100 patients with β-thalassemia major.•Thalidomide can improve the lifespan and reduce hemolysis of red blood cells in patients with β-thalassemia major.•Provide a new treatment alternative for improving the quality of life of patients with β-thalassemia major. Recent studies have shown the thalidomide's therapeutic potential in treatment of patients with β-thalassemia major. However, the effect of thalidomide on metabolism and lifespan of red blood cells (RBCs) is rarely reported. This study was a post hoc analysis of a randomized controlled trial (Chinese Clinical Trial Registry, ChiCTR1800015702). One hundred patients with β-thalassemia major were randomly assigned 1:1 to treatment with a placebo or thalidomide. The primary outcomes were the differences in RBC lifespan, reticulocyte count, and peripheral nucleated RBC count of patients after treatment of 12 weeks. Other indicators of hemolytic reaction were also analyzed. Compared with the placebo group after treatment of 12 weeks, the thalidomide group showed a longer RBC lifespan (16.29 ± 6.42 vs 12.90 ± 4.98 days; P = 0.004), smaller mean corpuscular volume (68.34 ± 7.79 vs 78.01 ± 6.33 fl; P < 0.001), smaller mean corpuscular hemoglobin (21.62 ± 2.85 vs 24.68 ± 2.69 pg; P < 0.001), and lower lactate dehydrogenase (190.00 [148.00 - 305.00] vs 251.00 [199.20 - 327.80]; P = 0.014). Meanwhile, thalidomide significantly increased the RBC lifespan at 24 weeks (21.24 ± 8.30 days; P < 0.001) and 48 weeks (23.21 ± 8.42 days; P < 0.001) when compared with baseline (12.8 ± 6.0 days). Thalidomide increases the RBC lifespan and reduces hemolytic reactions in patients with β-thalassemia major. Chinese Clinical Trial Registry identifier: ChiCTR1800015702.

• Publisher: Elsevier Inc,Elsevier Limited
• Subject: Adolescent / Adult / Anemia / beta-Thalassemia - blood / beta-Thalassemia - drug therapy / Blood / Blood diseases / Blood transfusions / Child / Clinical outcomes / Clinical trials / Double-Blind Method / Erythrocyte Indices - drug effects / Erythrocytes / Erythrocytes - drug effects / Erythrocytes - metabolism / FDA approval / Female / Hemoglobin / Hemolysis - drug effects / Hospitals / Humans / Internal Medicine / Iron / L-Lactate dehydrogenase / Life span / Male / Medical prognosis / Metabolism / Normal distribution / Patients / Placebos / Red blood cells / Reticulocyte Count / Thalassemia / Thalidomide / Thalidomide - adverse effects / Thalidomide - pharmacology / Thalidomide - therapeutic use / Treatment Outcome / Young Adult / β-Thalassemia