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Clinical Trial
. 2021 Nov 18;6(1):405.
doi: 10.1038/s41392-021-00811-0.

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

Jiang-Ming Chen #  1 , Wei-Jian Zhu #  2 , Jie Liu #  3   4 , Gui-Zhen Wang #  3 , Xiao-Qin Chen #  5 , Yun Tan #  6 , Wei-Wei Xu  7 , Li-Wei Qu  3   8 , Jin-Yan Li  7 , Huan-Ju Yang  7 , Lan Huang  7 , Ning Cai  7 , Wei-Da Wang  5 , Ken Huang  9 , Jian-Quan Xu  10 , Guo-Hui Li  11 , Sheng He  12 , Tian-Ying Luo  7 , Yi Huang  13 , Song-Hua Liu  14 , Wen-Qiang Wu  15 , Qi-Yang Lu  7 , Mei-Guang Zhou  7 , Shu-Ying Chen  7 , Rong-Lan Li  7 , Mei-Ling Hu  7 , Ying Huang  7 , Jin-Hua Wei  16 , Jun-Min Li  6 , Sai-Juan Chen  17 , Guang-Biao Zhou  18
Affiliations
Clinical Trial

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

Jiang-Ming Chen et al. Signal Transduct Target Ther. .

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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Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Study design
Fig. 2
Fig. 2
Changes in Hb, transfusion volume, and Hb F in patients upon placebo or thalidomide treatment. The levels of Hb (a, b) and transfusion volume (c) were expressed as mean ± standard deviation (SD), and P values determined by Student’s t test (*) and mixed-effects regression analyses (#) were provided. ***P < 0.001, ****P < 0.0001; ####P < 0.0001. The percentages of Hb F (d) were expressed as median (interquartile range), and P value was determined by Mann–Whitney U test. $$$P < 0.001
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