Samal Priyanka, Paul Anindita, Bahirat Harshwardhan, Bishoyi Ajit Kumar, Epari Venkatarao
Department of Clinical Hematology, Institute of Medical Sciences & Sum Hospital, Siksha 'O' Anusandhan Deemed to Be University, Bhubaneswar, Odisha, 751003, India.
Department of Community Medicine, Institute of Medical Sciences & Sum Hospital, Siksha 'O' Anusandhan Deemed to Be University, Bhubaneswar, Odisha, 751003, India.
Blood Res. 2025 Apr 1;60(1):21. doi: 10.1007/s44313-025-00068-4.
The clinical course of sickle cell anemia (SCA) is variable, with chronic hemolysis and end-organ damage caused by microvascular occlusion. We evaluated the efficacy and safety of thalidomide plus hydroxyurea (HU) compared with HU alone to determine whether the combination provides a superior clinical benefit and safety profile.
This was an open-label quasi-experimental clinical trial (Clinical Trials Registry of India, CTRI Registration Number 2023/04/065682). Patients with SCA aged > 12 years and postmenopausal females aged > 45 years were allocated 1:1 to receive either HU (20 mg/kg/day) and thalidomide (50 mg/day) in Group A or HU (20 mg/kg/day) only in Group B.
The frequency of vaso-occlusive crises (VOCs), transfusion requirements, variations in hematological parameters (hemoglobin [Hb], fetal hemoglobin [HbF], and sickle hemoglobin [HbS]), and side effects between the groups were assessed over 12 months. Repeated-measures analysis of variance was used to determine changes across the observation period. The mean age of the 66 patients diagnosed with SCA (homozygous HbS mutation) was 32.9 (standard deviation ± 11.5) years, and 57.6% were males. Over the 12-month observation period, Group A had significantly fewer VOCs (3.48 ± 2.81) and packed red blood cell transfusions (3.61 ± 2.19) than Group B (11.36 ± 4.20 VOCs; 13.27 ± 3.70 transfusions) (p = 0.0001). There was a significant increase in Hb (8.2 ± 1.8 to 11.8 ± 1.2 g/dL), a decrease in HbS% (72.5 ± 5.5 to 64.5 ± 5.4), and a rise in HbF% (18.9 ± 5.1 to 28.4 ± 5.6) (p < 0.0001) in Group A.
Combining thalidomide with HU significantly reduced VOCs and transfusion requirements, improved Hb and HbF%, and decreased HbS levels.
镰状细胞贫血（SCA）的临床病程具有变异性，微血管阻塞会导致慢性溶血和终末器官损伤。我们评估了沙利度胺联合羟基脲（HU）与单用HU的疗效和安全性，以确定联合用药是否能带来更好的临床益处和安全性。
这是一项开放标签的准实验性临床试验（印度临床试验注册中心，CTRI注册号2023/04/065682）。年龄大于12岁的SCA患者和年龄大于45岁的绝经后女性按1:1分配，A组接受HU（20mg/kg/天）和沙利度胺（50mg/天），B组仅接受HU（20mg/kg/天）。
在12个月内评估了两组之间血管闭塞性危机（VOCs）的发生频率、输血需求、血液学参数（血红蛋白[Hb]、胎儿血红蛋白[HbF]和镰状血红蛋白[HbS]）的变化以及副作用。采用重复测量方差分析来确定观察期内的变化。66例被诊断为SCA（纯合子HbS突变）的患者平均年龄为32.9（标准差±11.5）岁，57.6%为男性。在12个月的观察期内，A组的VOCs（3.48±2.81）和浓缩红细胞输血次数（3.61±2.19）明显少于B组（11.36±4.20次VOCs；13.27±3.70次输血）（p = 0.0001）。A组的Hb显著升高（从8.2±1.8 g/dL升至11.8±1.2 g/dL），HbS%降低（从72.5±5.5降至64.5±5.4），HbF%升高（从18.9±5.1升至28.4±5.6）（p < 0.0001）。
沙利度胺与HU联合使用可显著降低VOCs和输血需求，提高Hb和HbF%，并降低HbS水平。
