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Safety and efficacy of thalidomide in patients with transfusion-dependent β-thalassemia: a randomized clinical trial

Department of Hematology, Wuzhou Gongren Hospital, Wuzhou, 543001, Guangxi, China. jiangming.chen@126.com. Department of Hematology, Zhuhai People's Hospital (Zhuhai Hospital Affiliated with Jinan University), Zhuhai, 541000, Guangdong, China. State Key Laboratory of Molecular Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100021, China. Department of Reproductive Medicine, The First Affiliated Hospital of Hebei North University, Zhangjiakou, 075000, Hebei, China. State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine; Medical Oncology Department, Sun Yat-Sen University Cancer Center, Guangzhou, 510060, Guangdong, China. State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, National Research Center for Translational Medicine, Ruijin Hospital Affiliated with Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China. Department of Hematology, Wuzhou Gongren Hospital, Wuzhou, 543001, Guangxi, China. State Key Laboratory of Membrane Biology, Institute of Zoology, Chinese Academy of Sciences & University of Chinese Academy of Sciences, Beijing, 100101, China. Department of Pediatrics, Affiliated Hospital of Youjiang Medical University for Nationalities, Baise City, 533000, Guangxi, China. Department of Hematology, Yulin Guinan Hospital, Yulin, 537005, Guangxi, China. Department of neurology, Wuzhou Gongren Hospital, Wuzhou, 543001, Guangxi, China. Guangxi Key Laboratory of Basic Research on Birth Defects Prevention and Treatment, Guangxi Zhuang Autonomous Region Women and Children Health Care Hospital, Nanning, 530000, Guangxi, China. Department of Hematology, Guigang People's Hospital, Guigang, 537100, Guangxi, China. Department of Hematology, Hospital of Traditional Chinese Medicine of Wuzhou City, Wuzhou, 543002, Guangxi, China. Department of Hematology, Wuzhou Red Cross Hospital, Wuzhou, 543002, Guangxi, China. Department of Hematology, Hechi People's Hospital, Hechi City, 547000, Guangxi, China. State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, National Research Center for Translational Medicine, Ruijin Hospital Affiliated with Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China. sjchen@stn.sh.cn. State Key Laboratory of Molecular Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100021, China. gbzhou@ioz.ac.cn.

Signal transduction and targeted therapy. 2021;6(1):405
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Abstract
Thalidomide induces γ-globin expression in erythroid progenitor cells, but its efficacy on patients with transfusion-dependent β-thalassemia (TDT) remains unclear. In this phase 2, multi-center, randomized, double-blind clinical trial, we aimed to determine the safety and efficacy of thalidomide in TDT patients. A hundred patients of 14 years or older were randomly assigned to receive placebo or thalidomide for 12 weeks, followed by an extension phase of at least 36 weeks. The primary endpoint was the change of hemoglobin (Hb) level in the patients. The secondary endpoints included the red blood cell (RBC) units transfused and adverse effects. In the placebo-controlled period, Hb concentrations in patients treated with thalidomide achieved a median elevation of 14.0 (range, 2.5 to 37.5) g/L, whereas Hb in patients treated with placebo did not significantly change. Within the 12 weeks, the mean RBC transfusion volume for patients treated with thalidomide and placebo was 5.4 ± 5.0 U and 10.3 ± 6.4 U, respectively (P < 0.001). Adverse events of drowsiness, dizziness, fatigue, pyrexia, sore throat, and rash were more common with thalidomide than placebo. In the extension phase, treatment with thalidomide for 24 weeks resulted in a sustainable increase in Hb concentrations which reached 104.9 ± 19.0 g/L, without blood transfusion. Significant increase in Hb concentration and reduction in RBC transfusions were associated with non β0/β0 and HBS1L-MYB (rs9399137 C/T, C/C; rs4895441 A/G, G/G) genotypes. These results demonstrated that thalidomide is effective in patients with TDT.
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Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine