Long‐term efficacy and safety of thalidomide treatment in children with β$\ubeta$‐thalassemia major

Xiao, Jian; Liu, Xiaodong; Peng, Wei; Li, Lan; Hua, Fang; Chen, Kun; Zhang, Jin; Luo, Shan; Yang, Kun; Wu, Yu

doi:10.1002/pbc.30391

Cited by 5 publications

(2 citation statements)

References 13 publications

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“…Of the 34 patients with TDT, 21 (61.8%) achieved transfusion independence and were rated as having achieved the MaR response. This nding was similar to the rate of 63.6% reported in 22 patients with TDT who achieved the MaR in the study by Xiao et al [11] .…”

Section: Evaluating the E Cacy Based On Hb Levelssupporting

confidence: 91%

Clinical efficacy of thalidomide for various genotypes of beta thalassemia

Yang,

Kang,

Zhou

et al. 2024

Preprint

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Objective The objective of this study was to investigate the therapeutic efficacy of thalidomide across various genotype presentations of β-thalassemia so as to facilitate the early screening of thalidomide-sensitive thalassemia cases and to understand the impact of iron overload on thalidomide. Methods From our initial sample of 52 patients, we observed 48 patients with β-thalassemia for two years after administration of thalidomide. This cohort included 34 patients with transfusion-dependent thalassemia (TDT) and 14 patients with non-transfusion-dependent thalassemia (NTDT). We recorded the values of hemoglobin (Hb), fetal hemoglobin (HbF), and serum ferritin (SF) in the baseline period and at 1, 3, 6, 12, 18, and 24 months after enrollment, as well as the pre- and post-treatment blood transfusion volume in all 48 cases. According to the increase in Hb levels from baseline during the 6-month observation period, the response to thalidomide was divided into four levels: main response (MaR), minor response (MiR), slow response (SLR), and no response (NR). A decrease in serum ferritin levels compared to baseline was considered alleviation of iron overload. We calculated the overall response rate (ORR) as follows: ORR = MaR + MiR + SLR/number of observed cases. Results The ORR was 91.7% (44/48 cases), and 72.9% showed MaR (35/48 cases). Among the 34 patients with TDT, 21 patients (61.8%) were free of blood transfusion, and the remaining 13 patients still required blood transfusion, but their total blood transfusion volume reduced by 31.3% when compared to the baseline. We found a total of 33 cases with 10 combinations of advantageous genes, which included 5 cases with βCD41-42/βCD17 and 6 cases with βCD41-42/β-28. Based on the treatment outcomes among the 48 cases in the observation group, there were 33 cases in the MaR group and 15 cases in the SLR/NR group. There was a difference in HbF between the two groups at baseline (P = 0.041). There were significant differences between the two groups in Hb and HbF at the time points of 6 and 12 months, respectively (P < 0.001). Compared to the baseline measurement, there was a significant decrease in the level of SF at months 12 and 24 (P < 0.001). Conclusion In this study, we identified 10 β-thalassemia gene combinations that were sensitive to thalidomide. These gene combinations can be used for initial screening and to predict the therapeutic effect of thalidomide in clinical practice. We examined the therapeutic response to thalidomide and found that the administration of thalidomide in combination with standardized iron removal was more beneficial in reducing iron overload.

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Section: Evaluating the E Cacy Based On Hb Levelssupporting

confidence: 91%

Clinical efficacy of thalidomide for various genotypes of beta thalassemia

Yang,

Kang,

Zhou

et al. 2024

Preprint

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“…5 Among the previous studies, except for peripheral neurotoxicity, central venous thromboses, autoimmune hemolytic anemia, cerebrovascular accidents, and seizures in a few patients, the reported adverse effects were mild, and the common adverse effects included constipation, somnolence, arthralgias, and rashes. 3,[6][7][8] Hardly any report of secondary amenorrhea following thalidomide therapy have been reported in patients with β-thalassemia. Amenorrhea, as an adverse effect of thalidomide, was first reported in 1998 by Ordi et al 9 Amenorrhea during treatment with thalidomide is considered a rare side effect with a prevalence of 0.02%.…”

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confidence: 99%

Secondary amenorrhea in a β‐thalassemia major patient treated with thalidomide

Yu,

Yang

2024

Pediatric Blood & Cancer

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Recent evidence suggests a possible role of thalidomide in the treatment of thalassemia. By promoting expression of the γ-globin gene, thalidomide increases fetal hemoglobin (HbF) levels, and can significantly reduce the need for blood transfusions. 1 Moreover, thalidomide ameliorates erythropoiesis and iron homeostasis in patients with β-thalassemia. 2 Severe teratogenicity, peripheral neuropathy, and increased risk of thromboembolic events are among the main side effects of thalidomide; other common side effects include drowsiness, digestive disorders, skin eruptions, vertigo, headaches, and dizziness. 1,3 Compared to the incidence of the common side effects associated with thalidomide, reports of amenorrhea are limited.

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Thalidomide

2023

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Long‐term efficacy and safety of thalidomide treatment in children with β$\ubeta$‐thalassemia major

Cited by 5 publications

References 13 publications

Clinical efficacy of thalidomide for various genotypes of beta thalassemia

Clinical efficacy of thalidomide for various genotypes of beta thalassemia

Secondary amenorrhea in a β‐thalassemia major patient treated with thalidomide

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