初步探讨31例云南省血友病患者关节病与预防治疗的关系

发布时间：2018-04-05 00:24

本文选题：血友病　切入点：靶关节　出处：《昆明医科大学》2017年硕士论文

【摘要】：[目的]明确云南省血友病患者预防治疗对关节畸形的影响情况,明确血友病患者的最佳治疗方案,减少或避免患者关节不必要的出血风险,改善预后,提高血友病患者的生活质量。同时也为云南省各个血友病诊疗医院提供一份关于血友病患者预防治疗的参照方案。[方法]采用PedsQL生活质量量表评估患者生活质量,关节出血次数评估疗效,关节超声评估关节病变,对2015年7月1日至2017年3月30日到昆明医科大学第一附属医院血友病信息管理中心及诊疗中心登记管理并进行治疗的31例云南省血友病病人进行筛选、分组,分为预防治疗组及按需治疗组,血友病A的小剂量预防治疗的剂量:每次使用重组凝血因子Ⅷ10-15U/kg.d,每周2次,持续至少6个月。血友病B的小剂量预防治疗剂量:每次使用重组凝血因子Ⅸ10-20U/kg.d,每周2次,持续至少6个月。按需治疗组:患者在出现出血时才给予补充凝血因子:血友病A的按需治疗的剂(量重组凝血因子Ⅷ):体重(kg)X所需要提高的因子活性水平(%)÷2,血友病B的按需治疗的剂量(重组凝血因子Ⅸ):体重(kg)X所需要提高的因子活性水平(%)。分别通过问卷调查、电话追踪及定期复诊、随访,同时给血友病病人进行定期的关节超声检查,两组进行对比,将调查表所收集的患者临床信息资料及超声检查结果运用SPSS22.0统计软件进行数据统计分析,得出云南省血友病患者关节病采取预防治疗的预后。[结果]1、关节的结构状态:通过观察11例小剂量预防治疗的血友病患者关节超声检查情况及9例按需治疗的血友病患者的关节病变情况来分析预防治疗与按需治疗的区别,通过关节超声(HA)评估方法来评估,其评估两组患者以下几个方面的区别:渗出:关节积液或积血、纤维隔膜、滑膜增生、滑膜增厚伴滑膜血管增生、含铁血黄素沉积。结果显示预防治疗组患者的关节损害较按需治疗组轻。2、关节年出血次数情况:预防治疗组的年关节出血次数的均数为4.29(次),按需治疗组的年关节出血的均数为15.14(次),两独立样本t检验P=0.0010.05,说明二者存在统计学意义,血友病患者预防治疗能明显减少患者关节出血次数。3、年靶关节出血数目:通过统计11例预防治疗血友病患者及9例按需治疗血友病患者在半年研究期间靶关节出血关节数目,再乘于2得出年总出血关节数目,具体检查结果为:预防治疗组的年关节出血数目的均数为1.5(个),按需治疗组的年关节出血数目的均数为4.0(个),随机设计两独立样本t检验P=0.0030.05,说明预防治疗可以减少把关节年出血数目。4、因子使用情况:计算每年凝血因子使用量(IU/kg年),结果显示:预防治疗组年凝血因子使用量均数为682.7(IU/kg年),按需治疗组年凝血因子使用量均数为886.5(IU/kg年),随机设计两独立样本t检验P=0.1520.05,二者无统计学意义。但预防治疗组的年因子平均使用量较按需治疗组的年因子使用量少,说明预防治疗组年使用因子量还是有减少的趋势。5、患者的生活质量:通过PeDsQL调查表和病假时间(d/半年)来评价:5.1、使用PeDsQL调查表,采集了 11例(年龄小于14岁儿童)血友病患者的生活情况,其中5例使用预防治疗,6例使用按需治疗的方案,结果使用四格表确切概率法检验,统计结果显示:P=0.2420.01,无统计学意义,虽无统计学意义,但预防治疗组的有效率(60%)较按需治疗组有效率(15%)有增加趋势,说明预防治疗组有改善生活质量的趋势。5.2、年病假时间(d/年)。统计了 31例患者(儿童、成人均有),其中小剂量预防治疗组有16例,按需治疗组有15例,统计出其半年的病假日,再乘于2得出一年的病假日,结果显示:小剂量预防治疗组的病假日均数是12.1(天),按需治疗组的病假日均数是39.6天,随机设计的两独立样本t检验,P=0.015,其P值小于0.05,统计学有意义,小剂量预防治疗可以减少血友病的病假时间。6、血浆Ⅷ/Ⅸ因子抗体(抑制物)产生率:统计分析了 16例预防治疗组与15例按需治疗组治疗中半年时间复查的抑制物产生情况,分析预防治疗的安全性,统计结果显示:两组均未出现抑制物阳性患者病例。[结论]本实验通过对照研究得出以下结论:血友病患者中使用预防治疗可以减少血友病患者关节结构和功能的损伤、减少关节年出血次数、减少年靶关节出血数目、减少病假日;预防治疗及按需治疗相比,预防治疗组有可以减少年凝血因子使用量的趋势;预防治疗组的生活质量有改善的趋势;两组中均未见血浆Ⅷ/Ⅸ因子抗体(抑制物)产生。
[Abstract]:[Objective] clear Yunnan province hemophilia patients for the prevention and treatment effect of joint deformity, clear the best treatment for patients with hemophilia, or reduce risk, avoid unnecessary joint bleeding in patients with hemophilia improve prognosis, improve the quality of life of patients. At the same time for various haemophilia care hospitals in Yunnan province to provide a report on the prevention and treatment of hemophilia patients according to the scheme. By using the PedsQL quality of life scale to assess the quality of life of patients with joint bleeding times to assess the outcome of joint ultrasound assessment of joint disease, from July 1, 2015 to March 30, 2017 to the First Affiliated Hospital of Kunming Medical University medical center and hemophilia information management center registration management and screening, 31 cases of patients with hemophilia treatment group in Yunnan Province, divided into prevention treatment group and on-demand treatment group, small dose of hemophilia A prophylaxis dose: every time The use of recombinant factor VIII 10-15U/kg.d, 2 times a week for at least 6 months. The prevention and treatment of hemophilia B small dose dose: every time the use of recombinant clotting factor 10-20U/kg.d, 2 times a week for at least 6 months. In the treatment group: patients was given the supplement of blood coagulation factor in hemorrhage: Hemophilia A by treatment agent (the amount of recombinant clotting factor VIII) weight: (kg) X to improve the level of activity factor (%), 2 dose on-demand treatment of hemophilia B (recombinant factor IX) weight: X (kg) to improve the level of water factor activity (%) respectively by. The questionnaire survey, telephone follow-up and regular follow-up visits, follow-up, and for patients with hemophilia were joint regular ultrasound examination, two groups were compared, the survey collected the clinical information and ultrasound examination results using SPSS22.0 statistical software for statistical analysis of data obtained in Yunnan Province Hemophilia with joint disease preventive treatment. The prognosis of]1, the structure state of joint: through the observation of 11 cases of small dose of prevention and treatment of patients with hemophilia joint ultrasound and 9 cases by joint disease requiring treatment of patients with hemophilia to analyze differences in prevention and treatment with on-demand treatment, through joint ultrasound (HA) assessment the evaluation method to evaluate the difference between the two groups were as follows: exudate: joint fluid or blood, fiber membrane, synovial hyperplasia, synovial thickening and vascular synovial hyperplasia, hemosiderin deposition. The results showed that the prevention of joint damage of patients in the treatment group than the treatment group according to.2 light, the number of years: joint bleeding preventive treatment group annual joint bleeding times increased to 4.29 (Times), on-demand treatment group annual joint bleeding are the number 15.14 (Times), two independent samples t test P=0.0010.05, indicating the existence of two statistics Significance of prevention and treatment, can significantly reduce the number of patients with hemophilia.3 patients with joint bleeding, bleeding years target joint number: hemophilia patients and 9 cases according to the treatment of hemophilia patients during the study period in the first half of the year target joint joint number through statistical analysis of 11 cases of hemorrhage prevention and treatment, then take 2 years to get the total number of joint bleeding specific test results, as follows: preventive treatment group annual joint bleeding number increased to 1.5 (a), according to the number of years of joint bleeding treatment group are the number 4 (a), randomly designed two independent samples t test shows that P=0.0030.05 can reduce the prevention and treatment of joint bleeding years number.4, the use of factor: Calculation of each factor use volume (IU/kg), the results showed: prevention and treatment group in blood coagulation factor usage increased to 682.7 (IU/kg), on-demand treatment in coagulation factor usage increased to 886.5 (IU/kg), randomized design for two independent samples t P=0.1520.05 test, two was not statistically significant. But the preventive treatment group annual average usage factor than on-demand treatment group annual factor use less, that preventive treatment group use factor or decreased.5, the quality of life of patients by PeDsQL questionnaire and sick leave time (d/ year) to evaluate 5.1, using the PeDsQL questionnaire, 11 cases were collected (less than 14 years of age children) life of hemophilia patients, including 5 cases of the use of preventive treatment, 6 cases of the use of on-demand treatment scheme, results using four exact test, the results showed that P=0.2420.01 had no statistical significance, although there was no statistically significant but, the prevention of the efficiency of the treatment group (60%) than to the efficiency of the treatment group (15%) increased, the prevention of treatment group improved the quality of life of the trend of.5.2 years, the duration of sick leave (d/). The statistics of 31 patients (children and adults are There is one small dose), preventive treatment group 16 cases, according to the 15 cases in the treatment group, the statistics of the first half of the disease would take a holiday, in 2 years that a disease of holiday, results show: small dose treatment group the average daily number of sick leave is 12.1 (days), according to the daily number of sick leave treatment the group is 39.6 days, two independent samples t test, randomized design of P=0.015, the P value is less than 0.05, statistically meaningful, small dose of prevention and treatment can reduce the duration of sick leave of haemophilia.6, von Willebrand factor (inhibitor) / IX antibody production rate: statistical analysis of 16 cases of preventive treatment group and 15 cases as needed the treatment group was six months in the treatment of inhibitor production, safety analysis of prevention and treatment, statistical results showed that the two groups had no cases of positive patients. Conclusion the inhibitor] this experiment through the control study to draw the following conclusion: in the prevention and treatment of hemophilia patients can reduce the blood Friends of patients with joint function and structure damage, reduce joint annual bleeding times, reducing annual target joint number of bleeding, reduce the disease prevention and treatment and holidays; on-demand treatment compared with preventive treatment group can reduce juvenile coagulation factor usage trends; prevention of the quality of life of the treatment group improved trend; in the two group there were no von Willebrand factor IX / antibody (inhibitor).

【学位授予单位】：昆明医科大学
【学位级别】：硕士
【学位授予年份】：2017
【分类号】：R554.1;R684

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