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

Autor: Jiang-Ming Chen, Wei-Jian Zhu, Jie Liu, Gui-Zhen Wang, Xiao-Qin Chen, Yun Tan, Wei-Wei Xu, Li-Wei Qu, Jin-Yan Li, Huan-Ju Yang, Lan Huang, Ning Cai, Wei-Da Wang, Ken Huang, Jian-Quan Xu, Guo-Hui Li, Sheng He, Tian-Ying Luo, Yi Huang, Song-Hua Liu, Wen-Qiang Wu, Qi-Yang Lu, Mei-Guang Zhou, Shu-Ying Chen, Rong-Lan Li, Mei-Ling Hu, Ying Huang, Jin-Hua Wei, Jun-Min Li, Sai-Juan Chen, Guang-Biao Zhou
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Zdroj: Signal Transduction and Targeted Therapy, Vol 6, Iss 1, Pp 1-7 (2021)
Druh dokumentu: article
ISSN: 2059-3635
DOI: 10.1038/s41392-021-00811-0
Popis: 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
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