Ko Bor-Sheng, Chang Ming-Chih, Chiou Tzeon-Jye, Chang Te-Kau, Chen Yeu-Chin, Lin Sheng-Fung, Chang Cheng-Shyong, Lu Yin-Che, Yeh Su-Peng, Chen Tsai-Yun, Hwang Wei-Shou
a Department of Internal Medicine , National Taiwan University Hospital , Taipei , Taiwan.
b Division of Hematology and Oncology , Mackay Memorial Hospital , Taipei , Taiwan.
Hematology. 2019 Dec;24(1):247-254. doi: 10.1080/16078454.2018.1557860.
Patients with myelodysplastic syndromes (MDS), aplastic anemia (AA) or other rare anemia require chronic blood transfusions which can lead to iron overload and subsequent excess iron-mediated complications. Intensive iron chelation with deferasirox could remove excess iron and can alleviate these events; however, the long-term safety and efficacy in Chinese population are not clearly characterized. This study examined the long-term efficacy and safety of deferasirox in a real-world setting in Taiwan.
This observational, non-interventional, single-arm, multi-center, phase IV study was designed to collect the safety and clinical information about patients who were treated with deferasirox according to investigator's judgment and in accordance with the general clinical practice.
From 2009 to 2011, patients with MDS (N = 38), AA (N = 23), and other rare anemias (N = 18) were enrolled. The mean deferasirox exposure was 17.7 ± 4.02 mg/kg/day. The most common drug-related AEs were skin disorders (32.9%) and gastrointestinal disorders (30.4%), while grade 3-4 AEs were rare (5.1%). In the overall patient population, deferasirox effectively decreased serum ferritin levels at 1 year (P = 0.0154) and 3 years (P = 0.0424) from the baseline. Upon the use of deferasirox, 32.9% patients showed erythroid response and 16.7% patients had platelet response.
For patients with MDS, AA, and other rare anemias, the AEs observed in this 3-year surveillance study with deferasirox were mostly mild or moderate. In addition, the hematological response rate was higher than that in the EPIC study, which primarily enrolled Caucasian patients.
骨髓增生异常综合征（MDS）、再生障碍性贫血（AA）或其他罕见贫血患者需要长期输血，这可能导致铁过载及随后由铁过量介导的并发症。地拉罗司强化铁螯合可去除过量铁并减轻这些情况；然而，其在中国人群中的长期安全性和疗效尚未明确。本研究在台湾的实际临床环境中考察了地拉罗司的长期疗效和安全性。
本观察性、非干预性、单臂、多中心IV期研究旨在收集根据研究者判断并按照一般临床实践接受地拉罗司治疗的患者的安全性和临床信息。
2009年至2011年，纳入了MDS患者（N = 38）、AA患者（N = 23）和其他罕见贫血患者（N = 18）。地拉罗司的平均暴露量为17.7±4.02mg/kg/天。最常见的药物相关不良事件为皮肤疾病（32.9%）和胃肠道疾病（30.4%），而3 - 4级不良事件罕见（5.1%）。在总体患者人群中，地拉罗司在1年（P = 0.0154）和3年（P = 0.0424）时均有效降低了血清铁蛋白水平，相对于基线水平。使用地拉罗司后，32.9%的患者出现红系反应，16.7%的患者出现血小板反应。
对于MDS、AA和其他罕见贫血患者，在这项为期3年的地拉罗司监测研究中观察到的不良事件大多为轻度或中度。此外，血液学反应率高于主要纳入白种人的EPIC研究。
