Short-term oral corticosteroid therapy for acute haemarthrosis in haemophilia patients with high-titre inhibitors.
Short-term oral corticosteroid therapy for acute haemarthrosis in haemophilia patients with high-titre inhibitors.
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短期口服皮质类固醇治疗患有高滴度抑制剂的血友病患者的急性关节血肿。
DOI：
10.1111/j.1365-2516.2006.01410.x
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发表时间：
2007
期刊：
影响因子：
--
通讯作者：
Buchanan,GR
中科院分区：
文献类型：
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作者：
Medeiros,D;Laufenberg,JA;Miller,KL;Buchanan,GR
对于血友病和高滴度因子VIII(FVIII)抑制剂的患者，急性出血事件的治疗并不令人满意。为了确定短期皮质类固醇治疗是否能提高急性关节出血的体征和症状的缓解，我们对FVIII缺乏和接受F8抑制剂旁路活性(FEIBA®)治疗急性出血事件的高效价抑制剂的儿童进行了一项随机、双盲、安慰剂对照研究。在每一次关节出血时，患者被随机分为泼尼松龙2 mg kg−1天−1次或安慰剂分割的tid。在肺霸的基础上加用2 天(6剂)。主要终点是所需的后续剂量的Feiba。研究药物的效果也由患者或父母通过体检和关节反复出血的方式进行主观评估。在研究期间，7名患者参加了45个可评估的事件，其中24个接受强的松龙治疗，21个接受安慰剂治疗。在接受强的松龙和安慰剂治疗的患者中，平均分别输注2.08和1.86剂量的飞霸。经Wilcoxon Rank Sum检验，服用皮质类固醇和服用安慰剂的患者在额外输注Feiba的次数或症状持续时间方面没有统计学上的显著差异。我们的结论是，对于血友病和高滴度抑制剂的患者，口服皮质类固醇作为辅助治疗的2天疗程并没有显著的益处。
Treatment of acute bleeding events is unsatisfactory in patients with haemophilia and high‐titre factor VIII (FVIII) inhibitors. In order to determine whether short‐term corticosteroid therapy enhances resolution of the signs and symptoms of acute haemarthrosis, we performed a randomized, double‐blind, placebo‐controlled study in children with FVIII deficiency and high‐titre inhibitors receiving Factor Eight Inhibitor Bypass Activity (FEIBA®) for acute haemorrhagic events. At each haemarthrosis, patients were randomized to receive either prednisolone 2 mg kg−1day−1or placebo‐divided t.i.d. for 2 days (six doses) in addition to FEIBA. The primary endpoint was the number of subsequent doses of FEIBA required. The effect of the study medication was also assessed subjectively by patients or parents, by physical examination and by repeated haemorrhages into the joint. During the study period, seven patients were enrolled with 45 evaluable events, 24 treated with prednisolone and 21 with placebo. An average of 2.08 and 1.86 doses of FEIBA were infused in the prednisolone‐ and placebo‐treated patients, respectively. By Wilcoxon Rank Sum Test, there was no statistically significant difference in number of additional infusions of FEIBA or duration of symptoms between the corticosteroid and placebo arms. We conclude that there is no significant benefit of a 2‐day course of oral corticosteroids as adjunctive therapy for haemarthrosis in patients with haemophilia and a high‐titre inhibitor.