| Objectives: To understand the prophylactic treatment status of children with hemophilia A in Sichuan Province, and to provide the basic data for prophylactic regimen adjustment and to improve the quality of life of children with hemophilia A.Methods: Prophylactic treatment data, including outpatients’ and inpatients’ medical records of children with hemophilia A from January 1, 2008 to August 1, 2015 in Chengdu Women and Children’s Central Hospital Hemophilia Center were collected, followed up and checked up by telephone call. Clinical characteristics were then retrospectively analyzed.Results:1. General Information:There were 156 cases of hemophilia A registered in Chengdu Women and Children’s Central Hospital hemophilia center between January 1, 2008 and August 1, 2015, in which 61 cases(39.10%) were treated by prophylaxis. The median age was 10 years(range 2.16 y t0 18y). Family history was positive in 19 cases(31.15%). In 61 cases,63.93% were moderate disease and 36.07% were severe.2. prophylaxis status:(1) Prophylactic regimen :Seven cases(11.48%)received primary prophylaxis, twenty six cases(42.62%)received secondary prophylaxis, seven cases(11.48%)received tertiary prophylaxis, and twenty one cases(34.43%)received intermittent prophylaxis.(2) The median age of prophylaxis initiation was 5.75(0.67~16.58)years. The dosage of prophylaxis was 5~25 IU/kg in 86.89%, and the median dose was 14(5~33)IU/kg. The frequency of prophylaxis was 1~2 times per week in 83.61%,and the median frequency was 1(0.25~2)times per week. The median duration of prophylaxis was 1.67(0.08~7.00)years3. Bleeding event: There were 1.00(0.00~8.00)times joint bleeding per month before prophylaxis, significantly decreased to 0.30(0.00~5.50)times per month after prophylaxis(P=0.000). The severe bleeding episodes also significantly decreased by 87.50% during the prophylaxis period(P=0.000).4. Arthropathy: Before and after prophylaxis the incidences of hemophilic arthropathy were 11.48% and 26.23% respectively(P=0.037).During the secondary prophylaxis period, 9 patients(34.62%), 16 joints had developed to hemophilic arthropathy. During the Tertiary prophylaxis period, 2 patients(28.57%), 2 joints had developed to hemophilic arthropathy. Until August 31, 2015,7 patients(34.33%), 12 joints had developed to hemophilic arthropathy in the intermittent prophylaxis,. The occurrence of new hemophilic arthropathy was significantly associated with the joint bleeding before and after prophylaxis(respectively r=0.388,P=0.004;r=0.434,P=0.001).5. Joint range of motion: Joint range of motion was limited in 20 cases(32.8%) before prophylaxis. And there was no significantly improvement after prophylaxis. Before prophylaxis, the joint range of motion was significantly associated with the age of prophylaxis initiation(r=0.474,P=0.000).6. Safety screening : The rate of virus infection related to blood infusion was zero. The incidence of inhibitor was18.42%(7/38).7. Consumption: The mean consumption of FVIII was 38.06 IU/ kg per month in prophylaxis, 2.35 times than that in on-demand therapy, which was 84.71 IU/kg per month.Conclusions:1. In Sichuan province, only a small percentage of children with hemophilia A received prophylaxis therapy, mainly secondary prophylaxis and intermittent prophylaxis, with older age, small dosage, and low frequency.2. Low-dose or medium-dose prophylaxis(5~25 IU/kg,1~2 times per week) could significantly decrease the frequency of joint bleeding,the occurrence of severe bleeding event and hemophilic arthropathy, but secondary, tertiary and intermittent prophylaxis could not completely prevent the occurrence of the hemophilic arthropathy. |
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