影响沙利度胺治疗输血依赖型地中海贫血的疗效及安全性的因素
Factors Influencing the Efficacy and Safety of Thalidomide in the Treatment of Transfusion-Dependent Thalassaemia
摘要: 沙利度胺作为HbF诱导剂在治疗输血依赖型地中海贫血(TDT)上取得了临床疗效，但其便秘、嗜睡、皮疹和周围神经病变不良反应受到关注。目前认为影响沙利度胺治疗TDT疗效的因素有HbF比率、基因多态性(SNP)、转录因子等。沙利度胺治疗前后Hb差值与治疗前HbF比率正相关，多项研究认为基线HbF比率有望成为预测、评估疗效及指导预后的独立指标。同时沙利度胺治疗的安全性与其每日剂量及其使用总剂量相关，每日剂量低于50 mg的情况下不良反应的发生率较低。
Abstract: Thalidomide has achieved clinical efficacy as an HbF inducer in the treatment of transfu-sion-dependent thalassaemia (TDT), but concerns have been raised about its side effects of contipa-tion, lethargy, rash and peripheral neuropathy. Factors currently thought to influence the efficacy of thalidomide in the treatment of TDT include the Hb/F ratio, gene polymorphisms (SNPs) and tran-scription factors. The difference in Hb between before and after thalidomide treatment is positively correlated with the pretreatment HbF ratio, and several studies have suggested that the baseline HbF ratio may become an independent indicator for predicting and evaluating treatment efficacy and guiding prognosis. Meanwhile, the safety of thalidomide treatment is related to the daily dose and the total dose used, with a lower incidence of adverse reactions at daily doses below 50 mg.
文章引用：戴晓琳, 黄月艳, 张晓旭, 丘明华, 倪艺齐, 陆莹, 文豪. 影响沙利度胺治疗输血依赖型地中海贫血的疗效及安全性的因素[J]. 临床医学进展, 2023, 13(11): 17050-17056. https://doi.org/10.12677/ACM.2023.13112388

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