Ematologia-Centro della Microcitemia e delle Anemie Congenite, Ospedale Galliera, Genoa, Italy.
Haematologica. 2013 Apr;98(4):555-9. doi: 10.3324/haematol.2012.076240. Epub 2012 Dec 14.
Several studies have established an association between iron chelation therapy with deferasirox and hematopoietic improvement in patients with myelodysplastic syndromes. There are no data from patients with β-thalassemia major. In a cross-sectional study, we evaluated the absolute number of several hematopoietic peripheral progenitors (colony-forming unit-granulocyte/macrophage, erythroid burst-forming units, colony-forming unit-granulocyte/erythrocyte/macrophage/megakaryocyte, and long-term culture-initiating cells) in 30 patients with β-thalassemia major (median age 29.5 years, 40% males) and 12 age-matched controls. For the β-thalassemia major patients, data on splenectomy status, the type of iron chelator used, and serum ferritin levels reflecting changes in iron status on the chelator were also retrieved. All patients had to be using the same iron chelator for at least 6 months with >80% compliance. The absolute number of all hematopoietic peripheral progenitors was higher in β-thalassemia major patients than in controls, and varied between splenectomized and non-splenectomized patients (lower number of erythroid burst-forming units and higher numbers of colony-forming unit-granulocyte/macrophage, colony-forming unit-granulocyte/erythrocyte/macrophage/megakaryocyte, and long-term culture-initiating cells). The number of erythroid burst-forming units was significantly higher in patients taking deferasirox (n=10) than in those taking either deferoxamine (n=10) or deferiprone (n=10) (P<0.05). After adjusting for age, sex, splenectomy status, and serum ferritin changes, the association between a higher absolute number of erythroid burst-forming units in deferasirox-treated patients than in patients taking deferoxamine or deferiprone remained statistically significant (P=0.011). In conclusion, in β-thalassemia major patients, compared with other iron chelators, deferasirox therapy is associated with higher levels of circulating erythroid burst-forming units. This variation is independent of iron status changes and is more likely to be due to the type of chelator.
几项研究已经证实，使用地拉罗司进行铁螯合疗法与骨髓增生异常综合征患者的造血改善有关。但在重型β地中海贫血患者中没有相关数据。在一项横断面研究中，我们评估了 30 名重型β地中海贫血患者（中位年龄 29.5 岁，40%为男性）和 12 名年龄匹配的对照者外周造血祖细胞（粒巨噬细胞集落形成单位、红系爆式集落形成单位、粒巨噬细胞/红细胞/巨核细胞集落形成单位和长期培养起始细胞）的绝对数量。还收集了关于脾切除术状态、使用的铁螯合剂类型以及反映螯合剂中铁状态变化的血清铁蛋白水平的数据。所有患者必须至少使用相同的铁螯合剂 6 个月，且符合率>80%。与对照组相比，重型β地中海贫血患者的所有外周造血祖细胞的绝对数量更高，且在脾切除术患者和非脾切除术患者之间存在差异（红系爆式集落形成单位数量较低，而粒巨噬细胞集落形成单位、粒巨噬细胞/红细胞/巨核细胞集落形成单位和长期培养起始细胞的数量较高）。使用地拉罗司（n=10）的患者的红系爆式集落形成单位数量明显高于使用去铁胺（n=10）或地拉罗酮（n=10）的患者（P<0.05）。在校正年龄、性别、脾切除术状态和血清铁蛋白变化后，与使用去铁胺或地拉罗酮的患者相比，地拉罗司治疗组患者的红系爆式集落形成单位绝对数量较高的关联仍然具有统计学意义（P=0.011）。总之，与其他铁螯合剂相比，在重型β地中海贫血患者中，地拉罗司治疗与循环中红系爆式集落形成单位水平升高相关。这种差异独立于铁状态变化，更可能是由于螯合剂的类型所致。
