【摘要】 目的 探討重組人凝血因子Ⅷ制劑小劑量短程預防性輸注能否有效減少中重度血友病A患兒關節出血問題。 方法 對2008年11月-2009年4月期間就診的13例年齡3~11歲的中重度血友病A患兒，均在為期2個月內接受重組人凝血因子Ⅷ 2次/周、間隔3 d、每次7.5~10.0 U/kg的靜脈預防性輸注，記錄治療前2個月與治療2個月時關節出血次數，以及同一關節反復發生出血的情況。 結果 治療前關節出血的發生次數為（3.77±2.13）次，治療后關節出血的發生次數為（0.46±0.87）次，治療前后比較，差異有統計學意義（Plt;0.01）；治療前靶關節出血的發生率為35.7％，治療后靶關節出血的發生率為0.0%，治療前后比較，差異有統計學意義（Plt;0.01）。患兒治療成本約510~680元/（kg?2個月）。 結論 重組人凝血因子Ⅷ制劑小劑量短療程預防性輸注能有效減少中重度血友病A患兒關節出血次數，同時可有效減少靶關節出血的發生率，從而在一定程度上保護關節的功能。治療費用相對可接受。【Abstract】 Objective To evaluate the efficacy of low-dose short-course infusion of recombinant human factor Ⅷ concentration in treating joint bleeding in children with severe and moderate hemophilia A. Methods Thirteen children aged 3 to 11 years old with severe or moderate hemophilia A were included in the present study from November 2008 to April 2009. For children in the treatment group, they were treated with low-dose short-course infusion of recombinant human factor Ⅷ concentration with a dose of 7.5-10.0 U/kg twice weekly as secondary prophylaxis for two months. The incidence of joint bleeding 2 months before treatment (control group) and during the 2 months of treatment (treatment group) was observed. Moreover, the incidence of their target joint bleeding was measured in both groups. Results Children in the control group had (3.77±2.13) joint bleedings while children in the treatment group had (0.46±0.87) joint bleedings, which was obviously lower than those in the control group (Plt;0.01). Meanwhile, the incidence of target joint bleeding in the treatment group was 0%, which was obviously lower than that in the control group (35.7%) (Plt;0.01). In the treatment group, the costs of treatment were about RMB 510-680 yuan/kg every 2 months. Conclusions Treatment with low-dose short-course infusion of recombinant human factor Ⅷ concentration can effectively decrease joint bleeding in children with severe and moderate hemophilia A, and can effectively decrease the incidence of target joint bleeding. Therefore, this method may play an important role in protection of the joint function in those patients at an acceptable cost.
獲得性血友病A是一種發病率極低但病死率較高的自身免疫性疾病。多種原因導致患者體內產生了特異結合、滅活凝血因子Ⅷ的凝血因子抑制物而發病。患者在其體內凝血因子抑制物完全清除之前都處于嚴重出血甚至致死的風險之中。因此，如何正確合理地選擇免疫抑制劑來進行抑制物清除治療是消除患者出血風險，達到長期穩定完全緩解以及最終治愈該病的重要環節。
目的系統評價凝血因子Ⅷ治療血友病A的藥物經濟學研究。方法計算機檢索PubMed、EMbase、Web of Science、The Cochrane Library、CNKI、VIP和WanFang Data數據庫，搜集凝血因子Ⅷ治療血友病A的藥物經濟學研究，檢索時限均為建庫至2022年2月。由2名研究者獨立篩選文獻、提取資料并評價納入研究的偏倚風險后，從研究模型、研究參數和不確定性分析等方面進行定性系統評價。結果最終納入17個藥物經濟學研究。納入研究總體質量較高，絕大多數符合藥物經濟學研究的基本框架，但仍存在模型設定、參數選擇等方面的不足。大部分研究結果顯示凝血因子Ⅷ對血友病A的預防治療與按需治療相比具有成本-效果優勢。結論當前證據顯示，凝血因子Ⅷ對血友病A的預防治療相較于按需治療可能具有一定的成本-效果優勢，但仍需在中國環境下驗證。