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目的评估第三代全长链重组凝血因子Ⅷ(f LFⅧ)预防治疗中间型、重型血友病A患儿的年出血次数、关节年出血次数和安全性。方法 2014-11-01—2016-11-30采用单中心、前瞻、非随机、自身对照比较南京大学医学院附属鼓楼医院血液科应用f LFⅧ进行大剂量预防治疗1年后的中间型、重型血友病A患儿年出血次数、关节年出血次数、凝血因子Ⅷ(FⅧ)抑制物的改变及药物副反应。结果 15例患儿均为男性,年龄中位数15岁(5~18岁),所有患儿FⅧ活性(FⅧ∶C)<2.2%,其中,重型血友病A 8例。应用f LFⅧ20~40 U/kg、每周2~3次进行大剂量预防治疗1年,年出血次数由(27.60±13.67)次降为(2.57±1.36)次(P<0.001),减少了90.7%;关节年出血次数由(11.13±8.81)次降为(0.93±1.10)次(P<0.001),减少了91.6%。7例经大剂量预防治疗达到关节年出血0次。不同程度的年出血次数均下降,其中重度年出血次数下降为0次。所有患儿在进行大剂量预防治疗1年间未发生药物相关副反应或严重不良反应,FⅧ抑制物滴度检测有3例0.1 BU/m L,其余未检出。结论应用f LFⅧ进行大剂量预防治疗可有效降低中间型、重型血友病A患儿年出血次数和关节年出血次数,安全、无副反应,无抑制物产生。
Objective To evaluate the annual bleeding, the number of joint bleeding and the safety of the third generation full length recombinant factor Ⅷ (f LFⅧ) in the prevention and treatment of middle and severe hemophilia A children. Methods 2014-11-01-2016-11-30 Single center, prospective, non-random, self-control comparison Gulou Hospital, Nanjing University Medical College Department of Hematology f LF â ... ¢ high-dose prevention and treatment of 1 year after the intermediate type, heavy blood Friendship disease A number of children with hemorrhage, the number of joint bleeding, coagulation factor Ⅷ (F Ⅷ) inhibitors and drug side effects. Results All 15 children were male, with a median age of 15 years (range, 5-18 years). All patients had FⅧ: C <2.2%, of which 8 were severe hemophilia A. The number of annual bleeding was reduced from (27.60 ± 13.67) to (2.57 ± 1.36) times (P <0.001), and decreased by 90.7% with the application of LF8020 to 40 U / kg for two to three times a week for one year. %; The number of bleeding in articular joint decreased from (11.13 ± 8.81) to (0.93 ± 1.10) times (P <0.001), a decrease of 91.6%. 7 cases of high-dose prophylaxis and treatment of joint bleeding reached 0 times. Different degrees of annual bleeding were decreased, of which the number of severe bleeding decreased to 0 times. All children in the high-dose prophylaxis therapy 1 year without drug-related side effects or serious adverse reactions, FⅧ inhibitor titers detected in 3 cases 0.1 BU / m L, the rest were not detected. Conclusions The high dose of f LF Ⅷ can effectively reduce the number of annual bleeding and joint bleeding in middle and heavy hemophilia A patients. It is safe, without side effects and without inhibitors.