Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia
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To investigate the efficacy and safety of thalidomide in patients with thalassemia intermedia (TI). Patients with a confirmed diagnosis of TI who met the trial criteria and signed consent forms were prescribed oral thalidomide 50 mg qn for 3 months from February 2017. Complete blood counts, Hb analysis, and liver and kidney functions were monitored monthly during treatment and any differences were compared before and after treatment. Patients with Hb increments > 2.0 g/dL were termed main responders (MaR), and those with Hb increments between 1.0 and 2.0 g/dL as minor responders (MiR), otherwise they were termed non-responders. Relevance analysis was performed to explore parameters predicting Hb increments after treatment. Adverse effects during treatment were carefully recorded. The overall response rate (ORR = MaR + MiR) and MaR rates were 78.6 and 50% after 1 month of treatment, respectively, and 85.7 and 71.4% after 3 months treatment. At the end of the treatment period, Hb and HbF increased by 2.5 ± 1.8 g/dL and 2.5 ± 1.6 g/dL, while bilirubin, lactate dehydrogenase, and the nucleated red blood cell count (NRBC) were significantly decreased, while the reticulocyte count significantly increased. Correlation analysis showed that the Hb increments correlated significantly with the ratio of HbF before treatment (r = 0.683, P = 0.007) rather than age, Hb, reticulocyte count, and NRBC before treatment. Adverse events during treatment were mild, and drug reduction or withdrawal from the trial was not required. Thalidomide had rapid and significant effects in patients with TI, and also, it is safe and convenient. But larger scale clinical trials will be required to confirm our conclusions. Trial Registration: NCT02995707, https://www.clinicaltrials.gov/ct2/show/NCT03184844?term=thalidomide+thalassemia&rank=1.
KeywordsThalassemia intermedia Thalidomide Fetal hemoglobin synthesis Clinical efficacy
Xiao-Lin Yin designed the study; Li Wang, Yong-Sheng Chen, and Ya-Li Zhou presented the patients; Ping-Ping Li and Yan-Ni Ma performed the laboratory analyses; Quan Ren wrote the first draft of the manuscript. All authors approved the final version of the manuscript.
This study was supported by projects from the National Natural Science Foundation of China (31201103) and the Natural Science Foundation of Guangxi, China (2015GXNSFAA139164 and 2014GXNSFBA118187).
Compliance with ethical standards
Our study was approved by the medical ethics committee of the 303rd Hospital of the People’s Liberation Army and registered at ClinicalTrials.gov, registration number: NCT02995707. Based on the Declaration of Helsinki, written informed consent was obtained from all patients included in the trial.
Conflict of interest
The authors declare that they have no conflicts of interest.
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