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[羟基脲治疗镰状细胞贫血患者:疗效与安全性]

[Hydroxyurea treatment in patients affected with sickle cell anemia: efficacy and safety].

作者信息

de Montalembert M

机构信息

Service de pédiatrie-générale, hôpital Necker-Enfants-Malades, 149, rue de Sèvres, 75015 Paris, France.

出版信息

Transfus Clin Biol. 2008 Feb-Mar;15(1-2):34-8. doi: 10.1016/j.tracli.2008.03.010. Epub 2008 May 21.

Abstract

Hydroxyurea is the unique drug having demonstrated an efficacy in preventing recurrences of painful crises, acute chest syndromes and in reducing transfusional needs in patients severely affected with sickle cell disease. However, there is a wide variation in the clinical response to hydroxyurea in sickle cell patients, with children generally experiencing greater benefits than adults. Short- and middle-term tolerances are good. Our uncertainties about long-term tolerance are mainly that we do not know the consequences of the drug on ulterior fertility in boys treated early and for long periods. Hydroxyurea has just been licensed for sickle cell adults and children in Europe. Its prescription for the moment must be restricted to severely affected patients, enrolled in long-term follow-up protocols.

摘要

羟基脲是唯一一种已被证明对预防镰状细胞病重症患者的疼痛性危象、急性胸部综合征复发以及减少输血需求有效的药物。然而,镰状细胞病患者对羟基脲的临床反应差异很大,儿童通常比成人受益更多。短期和中期耐受性良好。我们对长期耐受性的不确定性主要在于,我们不知道该药物对早期长期接受治疗的男孩日后生育能力的影响。羟基脲刚刚在欧洲获得针对镰状细胞病成人和儿童的许可。目前其处方必须限于纳入长期随访方案的重症患者。

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