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地拉罗司与去铁酮治疗骨髓增生异常综合征患者铁过载的对比研究。

A comparative study of deferasirox and deferiprone in the treatment of iron overload in patients with myelodysplastic syndromes.

机构信息

Institute of Hematology and Blood Transfusion, U nemocnice 1, 128 20 Praha, Prague, Czech Republic.

出版信息

Leuk Res. 2013 Dec;37(12):1612-5. doi: 10.1016/j.leukres.2013.07.021. Epub 2013 Aug 9.

Abstract

One hundred thirteen patients with myelodysplastic syndromes (MDS) with <10% of bone marrow blasts received either deferiprone in a daily dose of 40-90 mg/kg (48 patients) or deferasirox in a daily dose of 10-40 mg/kg (65 patients). Median duration of treatment was 10,9 months for deferiprone and 13,7 months for deferasirox. A substantial reduction of iron stores evaluated as a decrease in serum ferritin of more than 50% of pretreatment level was achieved in 18 patients in deferasirox group (27.7%) but not in any patient treated with deferiprone, The incidence of adverse effects (mostly gastrointestinal symptoms) was similar after administration of both the drugs. The symptoms of deferasirox toxicity were mild and mostly transient and no drug related myelosuppresive effect was observed in contrast to deferiprone where agranulocytosis occurred in 4% of patients and the treatment had to be discontinued due to side effects in 20% of patients. The results confirmed the usefulness of deferasirox as an effective and safe iron chelator in MDS patients and indication of deferiprone as an alternative treatment only in patients with mild or moderate iron overload clearly not indicated for deferasirox.

摘要

113 例骨髓增生异常综合征(MDS)患者骨髓原始细胞<10%,接受每日 40-90mg/kg 剂量的去铁酮(48 例)或每日 10-40mg/kg 剂量的地拉罗司(65 例)治疗。去铁酮组中位治疗持续时间为 10.9 个月,地拉罗司组为 13.7 个月。地拉罗司组 18 例(27.7%)患者的铁储存显著减少,血清铁蛋白较治疗前下降>50%,而接受去铁酮治疗的患者中无一例达到这一水平。两种药物不良反应(主要为胃肠道症状)发生率相似。与去铁酮相比,地拉罗司的毒性症状较轻且多为一过性,未观察到与药物相关的骨髓抑制作用,而去铁酮组有 4%的患者发生粒细胞缺乏症,20%的患者因不良反应而停止治疗。研究结果证实了地拉罗司作为 MDS 患者有效且安全的铁螯合剂的用途,以及去铁酮仅作为轻度或中度铁过载患者的替代治疗的适应证,而这些患者对地拉罗司并不适用。

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