Campus of Haematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, 90142 Palermo, Italy.
Molecular and Clinical Hematology Branch, National Institute of Diabetes and Digestive and Kidney Diseases/National Heart, Lung, and Blood Institute, Bethesda, MD 20814, USA.
Int J Mol Sci. 2018 Feb 28;19(3):681. doi: 10.3390/ijms19030681.
In sickle cell disease (SCD), hydroxyurea (HU) treatment decreases the number of vaso-occlusive crisis (VOC) and acute chest syndrome (ACS) by increasing fetal hemoglobin (HbF). Data are lacking regarding the frequency of HU dose modification or whether sub-therapeutic doses (<15 mg/kg/day) are beneficial. We reviewed the medical records of 140 patients from 2010 to 2014. The laboratory parameters and SCD complications were compared between the first and last visits based on HU use. Fifty patients (36%) never took HU or suspended HU ("no HU" group). Among patients taking <15 mg/kg/day HU on their first visit, half remained at the same dose, and the other half increased to ≥15 mg/kg/day. Among patients taking ≥15 mg/kg/day, 17% decreased to <15 mg/kg/day, and 83% stayed at ≥15 mg/kg/day. The "no HU" group had fewer episodes of VOC and ACS. Both HU treatment groups had a reduction in both complications ( < 0.0001). This improvement was observed in all SCD phenotypes. The white blood cell (WBC) counts were found to be lower, and HbF increased in both HU groups ( = 0.004, 0.001). The maximal HbF response to HU in HbS/β⁺-thalassemia was 20%, similar to those observed for HbSS (19%) and HbS/β⁰-thalassemia (22%). HbS/β⁺-thalassemia could have a similar disease severity as HbSS or HbS/β⁰-thalassemia. Patients with HbS/β⁰-thalassemia or HbS/β⁺-thalassemia phenotypes responded to HU.
在镰状细胞病（SCD）中，羟基脲（HU）通过增加胎儿血红蛋白（HbF）来减少血管阻塞性危象（VOC）和急性胸部综合征（ACS）的发生次数。关于 HU 剂量调整的频率，或者亚治疗剂量（<15mg/kg/天）是否有益，尚缺乏数据。我们回顾了 2010 年至 2014 年间 140 例患者的病历。根据 HU 的使用情况，比较了首次和末次就诊时的实验室参数和 SCD 并发症。50 例患者（36%）从未服用 HU 或暂停 HU（“无 HU”组）。在首次就诊时接受<15mg/kg/天 HU 的患者中，有一半保持相同剂量，另一半增加到≥15mg/kg/天。在接受≥15mg/kg/天 HU 的患者中，17%减少到<15mg/kg/天，83%保持在≥15mg/kg/天。“无 HU”组 VOC 和 ACS 发作次数较少。两组 HU 治疗组均减少了两种并发症（<0.0001）。所有 SCD 表型均观察到这种改善。HU 治疗组的白细胞（WBC）计数降低，HbF 升高（=0.004，0.001）。在 HbS/β⁺-地中海贫血中，HU 的最大 HbF 反应为 20%，与 HbSS（19%）和 HbS/β⁰-地中海贫血（22%）观察到的相似。在 HbS/β⁺-地中海贫血中，疾病严重程度可能与 HbSS 或 HbS/β⁰-地中海贫血相似。具有 HbS/β⁰-地中海贫血或 HbS/β⁺-地中海贫血表型的患者对 HU 有反应。
