重组人凝血因子Ⅷ小剂量短疗程次级预防性治疗中重度血友病A患儿关节出血

【】　目的　探讨重组人凝血因子Ⅷ制剂小剂量短程预防性输注能否有效减少中重度血友病A患儿关节出血问题。　方法　对2008年11月-2009年4月期间就诊的13例年龄3~11岁的中重度血友病A患儿，均在为期2个月内接受重组人凝血因子Ⅷ2次/周、间隔3d、每次7.5~10.0U/kg的静脉预防性输注，记录治疗前2个月与治疗2个月时关节出血次数，以及同一关节反复发生出血的情况。　结果　治疗前关节出血的发生次数为（3.77±2.13）次，治疗后关节出血的发生次数为（0.46±0.87）次，治疗前后比较，差异有统计学意义（P<0.01）；治疗前靶关节出血的发生率为35.7％，治疗后靶关节出血的发生率为0.0%，治疗前后比较，差异有统计学意义（P<0.01）。患儿治疗成本约510~680元/（kg?2个月）。　结论　重组人凝血因子Ⅷ制剂小剂量短疗程预防性输注能有效减少中重度血友病A患儿关节出血次数，同时可有效减少靶关节出血的发生率，从而在一定程度上保护关节的功能。治疗费用相对可接受。【Abstract】　Objective　Toevaluatetheefficacyoflow-doseshort-courseinfusionofrecombinanthumanfactorⅧconcentrationintreatingjointbleedinginchildrenwithsevereandmoderatehemophiliaA.　Methods　Thirteenchildrenaged3to11yearsoldwithsevereormoderatehemophiliaAwereincludedinthepresentstudyfromNovember2008toApril2009.Forchildreninthetreatmentgroup,theyweretreatedwithlow-doseshort-courseinfusionofrecombinanthumanfactorⅧconcentrationwithadoseof7.5-10.0U/kgtwiceweeklyassecondaryprophylaxisfortwomonths.Theincidenceofjointbleeding2monthsbeforetreatment(controlgroup)andduringthe2monthsoftreatment(treatmentgroup)wasobserved.Moreover,theincidenceoftheirtargetjointbleedingwasmeasuredinbothgroups.　Results　Childreninthecontrolgrouphad(3.77±2.13)jointbleedingswhilechildreninthetreatmentgrouphad(0.46±0.87)jointbleedings,whichwasobviouslylowerthanthoseinthecontrolgroup(P<0.01).Meanwhile,theincidenceoftargetjointbleedinginthetreatmentgroupwas0%,whichwasobviouslylowerthanthatinthecontrolgroup(35.7%)(P<0.01).Inthetreatmentgroup,thecostsoftreatmentwereaboutRMB510-680yuan/kgevery2months.　Conclusions　Treatmentwithlow-doseshort-courseinfusionofrecombinanthumanfactorⅧconcentrationcaneffectivelydecreasejointbleedinginchildrenwithsevereandmoderatehemophiliaA,andcaneffectivelydecreasetheincidenceoftargetjointbleeding.Therefore,thismethodmayplayanimportantroleinprotectionofthejointfunctioninthosepatientsatanacceptablecost.