Fragomeno Concetta, Roccabruna Emilio, D'Ascola Domenico Giuseppe
SSD Microcythemia Center, Hospital Bianchi Melacrino Morelli, Reggio Calabria, Italy.
SSD Microcythemia Center, Hospital Bianchi Melacrino Morelli, Reggio Calabria, Italy.
Blood Cells Mol Dis. 2015 Dec;55(4):382-6. doi: 10.1016/j.bcmd.2015.04.004. Epub 2015 Apr 22.
Patients with haematopoietic disorders requiring long-term blood transfusions are at risk of iron overload. This study aimed to investigate the efficacy and safety of long-term deferasirox monotherapy in patients with transfusion-dependent anaemia in the routine clinical practice setting.
This was a retrospective analysis of patients who commenced deferasirox therapy at the Hospital Bianchi Melacrino Morelli in Reggio Calabria, Italy. Data collected included cardiac and hepatic iron load (assessed by magnetic resonance imaging); left ventricular ejection fraction (LVEF). Patients were divided into two groups for analysis: group A (baseline information collected prior to deferasirox initiation) and group B (baseline information collected after deferasirox initiation).
Forty-six patients were included (group A: n=25; group B: n=21). The overall population was 63% male, with a mean age of 33 years. The majority of patients (65%) had thalassaemia major. In the overall population, cardiac iron levels between the baseline and first follow-up visits improved in both groups A and B (29.2 vs. 32.5 ms; p=0.04 and 28.4 vs. 31.4 ms; p=0.038). Liver iron levels improved significantly from baseline to visit 1 in group A (7.2 vs. 12.1 ms; p<0.004) and from baseline to visit 3 (6.9 vs. 10.7; p=0.049) in group B. Generally, there was no correlation between cardiac and liver iron levels. LVEF remained stable throughout the study period. Deferasirox was well tolerated and was not associated with significant adverse events.
Long-term treatment with deferasirox is effective and safe in patients with transfusion-dependent haemoglobinopathies monitored in the clinical practice setting.
需要长期输血的造血系统疾病患者存在铁过载风险。本研究旨在调查在常规临床实践环境中，长期使用地拉罗司单药治疗对依赖输血性贫血患者的疗效和安全性。
这是一项对在意大利雷焦卡拉布里亚的比安基·梅拉克里诺·莫雷利医院开始接受地拉罗司治疗的患者进行的回顾性分析。收集的数据包括心脏和肝脏铁负荷（通过磁共振成像评估）；左心室射血分数（LVEF）。患者分为两组进行分析：A组（在开始使用地拉罗司之前收集基线信息）和B组（在开始使用地拉罗司之后收集基线信息）。
共纳入46例患者（A组：n = 25；B组：n = 21）。总体人群中63%为男性，平均年龄33岁。大多数患者（65%）患有重型地中海贫血。在总体人群中，A组和B组从基线到首次随访时心脏铁水平均有所改善（29.2对32.5毫秒；p = 0.04以及28.4对31.4毫秒；p = 0.038）。A组从基线到第1次随访时肝脏铁水平显著改善（7.2对12.1毫秒；p < 0.004），B组从基线到第3次随访时肝脏铁水平显著改善（6.9对10.7；p = 0.049）。一般来说，心脏和肝脏铁水平之间无相关性。在整个研究期间LVEF保持稳定。地拉罗司耐受性良好，未发生显著不良事件。
在临床实践环境中监测的依赖输血性血红蛋白病患者中，长期使用地拉罗司治疗有效且安全。
