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口服螯合剂L1(去铁酮)治疗铁过载状态

[Treatment of iron overload states with oral administration of the chelator agent, L1 (Deferiprone)].

作者信息

Cermák J, Brabec V

机构信息

Ustav hematologie a krevní transfuze Praha.

出版信息

Vnitr Lek. 1994 Sep;40(9):586-90.

PMID:7975362
Abstract

Nine iron overloaded patients were treated with L1--Deferiprone (1,2-dimethyl-3-hydroxypyrid-4-one) in daily dose 3 g (40-50 mg/kg) for 12 weeks. In 7 patients the efficiency of L 1 treatment was compared to the therapeutic effect of the same dose of desferrioxamine (Desferal). A significant increase in urinary iron excretion was observed after administration of both chelating agents. Iron excretion after L 1 treatment was approximately 65% of that obtained with Desferal. The amount of excreted iron correlated with the amount of iron stores before chelation. A significant decrease in transferrin saturation, serum and red cell ferritin was observed after treatment with Desferal, L 1 administration caused a significant decrease only in serum ferritin level. However, all the parameters reflecting iron stores remained increased when compared to normal values after 12 weeks of chelation therapy. An incomplete absorption from gut and some reutilization of chelated iron may be responsible for less potent iron chelation by L 1 in comparison to Desferal. A low tolerance of the drug together with repeated nausea and vomiting were the most frequent adverse effects observed in the course of L1 administration. L 1 treatment had to be discontinued due to repeated vomiting in one patient and due to progressive granulocytopenia and thrombocytopenia in another patient. Because of the side effects more clinical studies with L 1 are needed before its introduction in wide clinical practice.

摘要

9名铁过载患者接受L1(去铁酮,1,2 - 二甲基 - 3 - 羟基吡啶 - 4 - 酮)治疗,日剂量为3 g(40 - 50 mg/kg),持续12周。在7名患者中,将L1治疗的效果与相同剂量去铁胺(去铁敏)的治疗效果进行了比较。两种螯合剂给药后均观察到尿铁排泄显著增加。L1治疗后的铁排泄量约为去铁胺治疗后铁排泄量的65%。排泄的铁量与螯合前铁储存量相关。去铁胺治疗后转铁蛋白饱和度、血清和红细胞铁蛋白显著降低,而L1给药仅使血清铁蛋白水平显著降低。然而,与螯合治疗12周后的正常值相比,所有反映铁储存的参数仍升高。与去铁胺相比,L1的铁螯合作用较弱可能是由于肠道吸收不完全和螯合铁的部分再利用。药物耐受性差以及反复出现恶心和呕吐是L1给药过程中最常见的不良反应。1名患者因反复呕吐、另1名患者因进行性粒细胞减少和血小板减少而不得不停止L1治疗。由于这些副作用,在广泛应用于临床实践之前,还需要对L1进行更多的临床研究。

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