| Background and purpose:Hemophilia A is rare heritage FⅧlack of hemorrhagic diseases. It is spontaneous or traumatic the joints, muscles, soft tissue bleeding for main features. Long-term repeated hemorrhage causing joint deformities, muscle atrophy and lifelong disability associated complications, Affect survival and quality of the patient and bring the great physical and emotional pain.On current, there is no a Radical method for cure Hemophilia A The clinical treatment mainly rely on blood coagulation factor lifelong continuous replacement therapy, that the main purpose is to reduce hemorrhage occurs, prevent joints and muscles damage and function impaired accumulation, so as to maintain a healthy life quality. Treatment includes on-demand treatment and prevention and treatment of two ways.Nearly 20 years of study has confirmed that prevention and treatment significantly better than on-demand treatment in reducing the risk of bleeding, joint damage and maintain normal life quality.But the international community has not yet been on the prevention programs, such as the dose of the treatment issues consensus, because the developed western countries recommended standard doses (25～40u/kg/time, three times a week) huge cost and frequent problems in injection, some developing countries have begun to explore a low doses of scholars to prevent treatments. Because of China's economic level and lack of medical system hemophilia, especially the diagnosis, treatment and prognosis of children hemophilia optimistic, in high incidence of hemophilia joint damage (13) than 68% to 92%(17-18), and most can study and life quality is poor, survival time is short, to point out the emphasis of improving of the treatment situation is first in pediatric patientsBased on the current economic conditions and hemophilia children of the urgent situation,Discusses the situation of low-dose prevention and treatment,which is the urgent need, thereby fundamentally change our hemophiliac high disability rate and improve quality of lifeBut foreign literature shows that, because of teenagers in bone joint rapidly developing vulnerable, prevention and treatment of the early age, joint function better protection, prevention and treatment of children in the future life is twice the result with half the effort effect.But at present domestic has yet to prevention and treatment of children's study hemophilia.Therefore imagination, Through observation evaluation of prevention and treatment of low doses (10u/kg/times, twice weekly) in improving the clinical effect of various indexes, and quality of life index (daily family and school activities during the children and their parents, anxiety and depression from two aspects of the psychological emotional control,discusses Low doses of prevention and treatment plan's Effect, safety and economic benefits, In order to avoid expensive medical expenses, minimize bleeding frequency and joint damage degree, improve the quality of life of the patients, for to explore low doses of prevention and treatment scheme provides the evidence, And for the relevant departments for health policies and medical insurance system to provide the reference.Data and Method1,Case of patient:random selection in our hospital hemophilia treatment centers and registered long-term follow-up, living in Guangzhou and the surrounding areas have received 36 cases of the past FⅧhemophilia A treatment of children, all male, median age 7. years (2～12 years). One heavy (FⅧ:C<1%) 8 cases (22.2%), medium (FⅧ:C 1～5%) 21 cases (58.3%), light (FⅧ:C>5%) 7 cases (19.5%); 36 cases of patients into the study group FⅧnegative inhibitor; on bleeding frequency were> 1,> 2 times those of which 32 cases (88.9%),≥3 times were 25 cases (69.4%); into the group in February before the recent during the 9/36 cases of severe bleeding events occurred in 11 cases.2,Kogenate FS (Bayer HealthCare Pharmaceuticals,Berkeley, CA, USA) is a full-length rFVIII productthat is formulated with sucrose (rFVIII-FS) as stabilizer, and produced without the addition Of plasma-derived proteins during its purification and formulation, and treated with a solvent-detergent viral inactivation step. Drug lot number:27N1346; is valid until March 2,2009.3,Low-dose preventive treatment programs:10U/kg/times,2 times per week; every Tuesday, Friday afternoon for vaccination. If the prevention and treatment of patients with no acute bleeding occurred during the incident, is still at the same time to give on-demand treatment.4,Duration of study:Since December 23,2008 to March 2,2009 only a total of 70 days (10 weeks).5,OUTCOME MEASURES:Clinical observation, including the frequency of bleeding, serious bleeding events, assessment of joint function hemophilia (hemophilia joint function of self-assessment system, FISH score) and the observation of safety (adverse reactions, FⅧinhibitor incidence); quality of life assessment include:①the level of day-to-day family activities,②the level of in-school activities,③self-rating anxiety scale,④self-rating depression scale.6,Statistial analysis:statistics SPSS13.0 package. Count data using Wilcoxon rank sum test for statistical analysis; measurement data using paired T-test statistical analysis. To P<0.05 for the difference was significant.Result1,36 cases of hemophilia A patients,23 patients received≥8 weeks continued prevention and treatment, and the other 13 cases of sustained prevention and treatment 3-6 weeks. Observation showed that 36 cases of hemophilia A treatment of children to prevent bleeding frequency than on the prevention of pre-treatment significantly reduced 77%(3.69±1.47 VS 0.72±0.84, P=0.00), and the two-month period the total cases of severe bleeding events at 11 cases reduced to 1 second (P=00).2,Continued prevention and treatment≥8 weeks in patients with 23 cases (prevention and treatment in February before the treatment and prevention during February) observed that the self-control, the prevention of bleeding during treatment on the prevention of pre-treatment than the frequency dropped 73%(4.43±2.35 VS 1.17±0.98, P=0.00); to prevent serious bleeding events during treatment than before treatment to prevent decreased 77%(P=0.02); prevention FISH score after treatment than before treatment to prevent a marked increase (15.52±5.15 VS 24.96±4.13, P=0.00) on the prevention of joint function after treatment of children and improved self-care ability; At the same time, low-dose preventive treatment doses of FⅧmonth (115.1U/Kg) is obviously higher than that of children of the group actually used dose (52 U/Kg) increased 121.3%, we suggest that the low dose needed for prevention and treatment programs instead of FⅧslightly smaller dose, and give children the clinical indicators of hemophilia (bleeding frequency of severe bleeding cases, FISH joint function score) and quality of life indicators (home and at school activity level, psychological anxiety and depression) is significantly improved, and its short-term economic benefits are obvious. More likely long-term prevention and treatment in the future to reduce the late complications of treatment costs and social burdens and bring more benefits costs.3,36 cases of children 3 to 10 weeks, a total of 568 times injection el kochi total 118300U, in each case on an average of 15.8 days exposure. All children in the use of the process of worship kochi not have any adverse reactions occur. All patients in the study after the end of the week to review FⅧinhibitor were negative. The reorganization of FⅧagents (el kochi) The use of children in our country hemophilia prevention of low-dose treatment with better security.4,Sustained prevention≥8 weeks treatment of 23 cases of hemophilia children's participation in activities of daily living observed that the self-control, after a short-term preventive therapy to improve joint function of children, the children can not engage in normal family activities (excluding activities, walking slowly), the ratio decreased (47.8% vs 82.6%, P<0.05), can not be in school during the normal activities (excluding activities, recess activities only), the ratio also decreased significantly (47.8% vs 60.9%, P<0.05), especially the "inactive" zero children. That appropriate treatment modalities, such as short-term low-dose preventive treatment to bring life to change the hemophiliacs, and help patients improve the quality of life.5,The first time that we assess the children to observe the domestic hemophilia emotional state of anxiety and depression,23 cases of hemophilia A in the prevention of children's pre-treatment incidence of anxiety and depression 82.6% and 30.4%. After more than eight weeks of short-term preventive treatment significantly reduced the frequency of bleeding and to improve home-school children's activity levels, the incidence of children's anxiety as much as 78.2 percent and 13.0 percent incidence of depression was significantly lower (P all<0.05), anxiety and depression both psychological certain short-term sentiment improved. And we also observed the child's parents during this period of anxiety and depression in contrast to changes in their own. It was found that the prevention of pre-treatment of hemophilia patients with the incidence of parental anxiety and depression as high as 100% incidence of 69.5 percent, significantly higher than that of children, indicate the existence of hemophiliacs and their parents more serious mental health problems, the conclusions and a number of foreign similar results. However, there is no current prevention and treatment of hemophilia study of children reported. After short-term prevention and treatment of children, their parents, the incidence of anxiety and depression as high as 65.2% and 26.0% significantly reduced the incidence (P all<0.05), indicating that parents of psychological anxiety and depression has been significantly improved。Conclusions1. Short-term low-dose preventive treatment for the hemophilia children who had a insufficient treatment in the past,can significantly reduce bleeding frequency and serious bleeding events; and it can significantly improve the joint function and the self-care ability in daily life;2. Short-term low-dose preventive treatment can improve the hemophilia children's ability in activities at school or family. At the same time, it is helpful for the hemophilia children and their parents to reduce the anxiety and depression feelings;3. Recombinant agents of FⅧused in low-dose preventive treatment of children has a better security;4. The cost and effectiveness of low-dose preventive treatment is in line with the national conditions, it should be advocated at this stage and can provide some references for relevant departments to make health policies and medical insurance system.
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