Safety and efficacy of thalidomide in patients with transfusion-dependent β-thalassemia: a randomized clinical trial
Signal transduction and targeted therapy. 2021;6(1):405
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.
A randomized controlled and multicenter clinical study of ferrous L-threonate in treatment of iron deficiency anemia . Chinese
Chung-Hua Nei Ko Tsa Chih Chinese Journal of Internal Medicine. 2005;44((11):):844-7.
OBJECTIVE To study the clinical efficacy and side effects of ferrous L-threonate for treatment of iron deficiency anemia (IDA). METHODS It is a multicentral, randomized, double blind, double placebo and paralled comparative study with positive control. One hundred and forty IDA patients diagnosed according to the standard criteria in three hospitals were randomly divided into a test group (ferrous L-threonate plus placebo ferrous succinate) and a positive control group (ferrous succinate plus placebo ferrous L-threonate). Some iron parameters were examined 1, 4 and 8 weeks after medication. Hemoglobin, reticulocyte and other parameters for safety observation were collected every two weeks. RESULTS For the 2 groups, self comparison showed significant difference (P < 0.01). The total efficacy is 98.44% and 97.01% respectively with no difference. Hemoglobin rised rapidly and gradually and reached a peak in week 8, the change was statistically significant (P < 0.01). Changes of iron parameters also showed significant difference. Side-effects were similar in both groups (13.85% and 14.71%, P > 0.05). CONCLUSION The effect of ferrous L-threonate in IDA treatment is significant and rapid. Side-effects are few and minimal.