• 成都市婦女兒童中心醫(yī)院兒內(nèi)1科（成都，610091）;

【摘要】　目的　 探討重組人凝血因子Ⅷ制劑小劑量短程預(yù)防性輸注能否有效減少中重度血友病A患兒關(guān)節(jié)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(nèi)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(yù)防性輸注，記錄治療前2個月與治療2個月時關(guān)節(jié)出血次數(shù)，以及同一關(guān)節(jié)反復(fù)發(fā)生出血的情況。　結(jié)果　 治療前關(guān)節(jié)出血的發(fā)生次數(shù)為（3.77±2.13）次，治療后關(guān)節(jié)出血的發(fā)生次數(shù)為（0.46±0.87）次，治療前后比較，差異有統(tǒng)計學(xué)意義（P lt;0.01）；治療前靶關(guān)節(jié)出血的發(fā)生率為35.7％，治療后靶關(guān)節(jié)出血的發(fā)生率為0.0%，治療前后比較，差異有統(tǒng)計學(xué)意義（P lt;0.01）?；純褐委煶杀炯s510~680元/（kg?2個月）。　結(jié)論　 重組人凝血因子Ⅷ制劑小劑量短療程預(yù)防性輸注能有效減少中重度血友病A患兒關(guān)節(jié)出血次數(shù)，同時可有效減少靶關(guān)節(jié)出血的發(fā)生率，從而在一定程度上保護(hù)關(guān)節(jié)的功能。治療費(fèi)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 (P lt;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%) (P lt;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.

引用本文： 李丹,徐鳴,張嫻,蔣永梅. 重組人凝血因子Ⅷ小劑量短療程次級預(yù)防性治療中重度血友病A患兒關(guān)節(jié)出血. 華西醫(yī)學(xué), 2011, 26(8): 1136-1138. doi: 復(fù)制

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2. 　Tagliaferri A, Di Perna C, Rivolta GF. Secondary prophylaxis in adolescent and adult haemophiliacs[J]. Blood Transfus, 2008, 6(2): 17-20.
3. 　張之南, 沈悌. 血液病診斷及療效標(biāo)準(zhǔn)[M]. 3版. 北京: 科學(xué)出版社, 2007: 191-193.
4. 　全國血友病協(xié)作組. 中國血友病患病率和八個地區(qū)生存率調(diào)查[J]. 中華血液學(xué)雜志, 1992, 13(9): 461-463.
5. 　Zhang L, Li H, Zhao H, et al. Retrospective analysis of 1 312 patients with haemophilia and related disorders in a single Chinese institute[J]. Haemophilia, 2003, 9(6): 696-702.
6. 　孫淑娟,　張磊,　楊仁池, 等. 101 例血友病骨關(guān)節(jié)病的臨床與X線分析[J]. 中國綜合臨床, 2006, 22(4): 326-328.
7. 　Nilsson IM, Berntorp E, Löfqvist T, et al. Twenty-five years experience of prophylactic treatment in severe haemophilia A and B[J]. J Intern Med, 1992, 232(1): 25-32.
8. 　Berntorp E, Boulyjenkov V, Brettler D, et al. Modern treatment of haemophilia[J]. Bull World Health Organ, 1995, 73(5): 691-701.
9. 　Kreuz W, Escuriola-Ettingshausen C, Funk M, et al. When should prophylactic treatment in patients with haemophilia A and B start? The German experience[J]. Haemophilia, 1998, 4(4): 413-417.
10. 　van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia[J]. Br J Haematol, 2001, 112(3): 561-565.
11. 　Yee TT, Beeton K, Griffioen A, et al. Experience of prophylaxis treatment in children with severe haemophilia[J]. Haemophilia, 2002, 8(2): 76-82.
12. 　Coppola A, Franchini M, Tagliaferri A. Prophylaxis in people with haemophilia[J]. Thromb Haemost, 2009, 101(4): 674-681.
13. 　Alok Srivastava, WFH Treatment Guidelines Working Group. Guidelines for the management of hemophilia[EB/OL]. Montreal: the World Federation of Hemophilia, 2005[2011-07-17]. http://www.wfh.org/2/docs/Publications/Diagnosis_and_Teatment/Guidilines_mng_Heamophilia.pdf.
  1. 1. 　王兆鉞. 血友病診治的進(jìn)展與展望[J]. 臨床血液學(xué)雜志, 2004, 17(2): 63-66.
  2. 2. 　Tagliaferri A, Di Perna C, Rivolta GF. Secondary prophylaxis in adolescent and adult haemophiliacs[J]. Blood Transfus, 2008, 6(2): 17-20.
  3. 3. 　張之南, 沈悌. 血液病診斷及療效標(biāo)準(zhǔn)[M]. 3版. 北京: 科學(xué)出版社, 2007: 191-193.
  4. 4. 　全國血友病協(xié)作組. 中國血友病患病率和八個地區(qū)生存率調(diào)查[J]. 中華血液學(xué)雜志, 1992, 13(9): 461-463.
  5. 5. 　Zhang L, Li H, Zhao H, et al. Retrospective analysis of 1 312 patients with haemophilia and related disorders in a single Chinese institute[J]. Haemophilia, 2003, 9(6): 696-702.
  6. 6. 　孫淑娟,　張磊,　楊仁池, 等. 101 例血友病骨關(guān)節(jié)病的臨床與X線分析[J]. 中國綜合臨床, 2006, 22(4): 326-328.
  7. 7. 　Nilsson IM, Berntorp E, Löfqvist T, et al. Twenty-five years experience of prophylactic treatment in severe haemophilia A and B[J]. J Intern Med, 1992, 232(1): 25-32.
  8. 8. 　Berntorp E, Boulyjenkov V, Brettler D, et al. Modern treatment of haemophilia[J]. Bull World Health Organ, 1995, 73(5): 691-701.
  9. 9. 　Kreuz W, Escuriola-Ettingshausen C, Funk M, et al. When should prophylactic treatment in patients with haemophilia A and B start? The German experience[J]. Haemophilia, 1998, 4(4): 413-417.
  10. 10. 　van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia[J]. Br J Haematol, 2001, 112(3): 561-565.
  11. 11. 　Yee TT, Beeton K, Griffioen A, et al. Experience of prophylaxis treatment in children with severe haemophilia[J]. Haemophilia, 2002, 8(2): 76-82.
  12. 12. 　Coppola A, Franchini M, Tagliaferri A. Prophylaxis in people with haemophilia[J]. Thromb Haemost, 2009, 101(4): 674-681.
  13. 13. 　Alok Srivastava, WFH Treatment Guidelines Working Group. Guidelines for the management of hemophilia[EB/OL]. Montreal: the World Federation of Hemophilia, 2005[2011-07-17]. http://www.wfh.org/2/docs/Publications/Diagnosis_and_Teatment/Guidilines_mng_Heamophilia.pdf.