Department of Hematology Rheumatology, Zhongshan Boai Hospital, Zhongshan, China.
Department of Electrocardiology, Zhongshan Boai Hospital, Zhongshan, China.
Medicine (Baltimore). 2024 Oct 25;103(43):e40328. doi: 10.1097/MD.0000000000040328.
To investigate the short-term and long-term efficacy and safety of thalidomide in the treatment of intermediate and severe β-thalassemia. We analyzed patients with intermediate and severe β-thalassemia treated at our hospital from February 2019 to February 2024. Patients who received treatment for more than 3 months were included. The efficacy of thalidomide was assessed by comparing changes in hemoglobin (Hb), ferritin, bilirubin, and Hb electrophoresis before and after treatment. Adverse drug reactions during treatment were also recorded. A total of 42 β-thalassemia patients were included, with thalidomide dosages ranging from 75 to 150 mg/d. The response rates at 1, 3, and 6 months of treatment were 73.8% (31/42), 75.0% (24/32), and 94.7% (18/19), respectively. The increase in Hb levels was primarily attributed to fetal hemoglobin (HbF). After 1 month of treatment, the HbF percentage increased from a baseline of 34.04 ± 27.58% to 56.25 ± 28.40% (P < .001). At 3 and 6 months, HbF further increased to 67.21 ± 27.12% (P < .001) and 73.93 ± 22.96% (P < .001), respectively. The average duration of thalidomide treatment was 25.3 ± 9.2 months (range: 4-60 months), with 6 patients treated for over 60 months and 18 patients for over 48 months. Two homozygous patients who failed thalidomide treatment achieved Hb levels above 100 g/L and discontinued transfusion therapy after 3 months of hydroxyurea treatment. The most common adverse reaction was somnolence, which was mild and tolerable. Thalidomide demonstrates significant and sustained therapeutic effects in β-thalassemia patients. The adverse reactions are mild and tolerable, allowing patients to continue treatment.
探讨沙利度胺治疗中间型和重型β地中海贫血的短期和长期疗效及安全性。
选取我院 2019 年 2 月至 2024 年 2 月接受治疗的中间型和重型β地中海贫血患者，纳入治疗时间超过 3 个月的患者。比较治疗前后血红蛋白（Hb）、铁蛋白、胆红素和 Hb 电泳的变化，评估沙利度胺的疗效。同时记录治疗期间的药物不良反应。
共纳入 42 例β地中海贫血患者，沙利度胺剂量为 75150 mg/d。治疗 1、3、6 个月的有效率分别为 73.8%（31/42）、75.0%（24/32）和 94.7%（18/19）。Hb 水平的升高主要归因于胎儿血红蛋白（HbF）。治疗 1 个月后，HbF 百分比从基线的 34.04±27.58%增加到 56.25±28.40%（P<0.001）。治疗 3、6 个月时，HbF 进一步增加至 67.21±27.12%（P<0.001）和 73.93±22.96%（P<0.001）。沙利度胺治疗的平均持续时间为 25.3±9.2 个月（460 个月），其中 6 例患者治疗时间超过 60 个月，18 例患者治疗时间超过 48 个月。2 例纯合子患者经沙利度胺治疗失败，在接受 3 个月羟基脲治疗后 Hb 水平达到 100 g/L 以上，停止输血治疗。最常见的不良反应是嗜睡，程度较轻，可耐受。
沙利度胺治疗β地中海贫血患者具有显著而持续的疗效，不良反应较轻，患者可耐受，可继续治疗。
