Franchini Massimo, Salvagno Gian Luca, Lippi Giuseppe
Servizio di Immunoematologia e Trasfusione - Centro Emofilia, Ospedale Policlinico, Piazzale Ludovico Scuro, 37134 Verona, Italy.
Thromb Haemost. 2006 Aug;96(2):113-8.
The development of inhibitors in patients with mild/moderate hemophilia A is an increasingly recognized occurrence and is manifested by the patients' bleeding pattern becoming more severe. Inherited (hemophilia genetic mutations) and acquired (type and delivery of factor VIII replacement therapy) factors have been associated with an increased likelihood of developing factor VIII inhibitors. Although the use of bypassing agents (i.e. activated prothrombin complex concentrates and recombinant factor VII activated) has been demonstrated to be effective in controlling bleeding episodes in patients who develop factor VIII inhibitors, the limited data available in the literature are insufficient to determine the optimal approach to the eradication of inhibitors (i.e. immune tolerance induction, immunosuppression or both) for this group. Particular attention should be directed to the prevention of this complication in those patients with mild/moderate hemophilia recognized to be at increased risk of developing a factor VIII inhibitor. In conclusion, large prospective trials are warranted in order to elucidate the many still unclear pathogenic and therapeutic aspects of the development of inhibitors in patients with mild/moderate hemophilia A.
在轻度/中度甲型血友病患者中，抑制剂的产生是一种越来越被认识到的现象，其表现为患者的出血模式变得更加严重。遗传因素（血友病基因突变）和后天因素（凝血因子VIII替代疗法的类型和给药方式）都与产生凝血因子VIII抑制剂的可能性增加有关。尽管已证明使用旁路制剂（即活化凝血酶原复合物浓缩物和重组活化凝血因子VII）在控制产生凝血因子VIII抑制剂的患者的出血发作方面是有效的，但文献中可用的数据有限，不足以确定针对该组患者根除抑制剂的最佳方法（即免疫耐受诱导、免疫抑制或两者兼用）。对于那些被认为有增加产生凝血因子VIII抑制剂风险的轻度/中度血友病患者，应特别注意预防这种并发症。总之，有必要进行大型前瞻性试验，以阐明轻度/中度甲型血友病患者中抑制剂产生的许多仍不清楚的发病机制和治疗方面的问题。
