本文選題：血友病　切入點：靶關(guān)節(jié)　出處：《昆明醫(yī)科大學》2017年碩士論文

【摘要】：[目的]明確云南省血友病患者預防治療對關(guān)節(jié)畸形的影響情況,明確血友病患者的最佳治療方案,減少或避免患者關(guān)節(jié)不必要的出血風險,改善預后,提高血友病患者的生活質(zhì)量。同時也為云南省各個血友病診療醫(yī)院提供一份關(guān)于血友病患者預防治療的參照方案。[方法]采用PedsQL生活質(zhì)量量表評估患者生活質(zhì)量,關(guān)節(jié)出血次數(shù)評估療效,關(guān)節(jié)超聲評估關(guān)節(jié)病變,對2015年7月1日至2017年3月30日到昆明醫(yī)科大學第一附屬醫(yī)院血友病信息管理中心及診療中心登記管理并進行治療的31例云南省血友病病人進行篩選、分組,分為預防治療組及按需治療組,血友病A的小劑量預防治療的劑量:每次使用重組凝血因子Ⅷ10-15U/kg.d,每周2次,持續(xù)至少6個月。血友病B的小劑量預防治療劑量:每次使用重組凝血因子Ⅸ10-20U/kg.d,每周2次,持續(xù)至少6個月。按需治療組:患者在出現(xiàn)出血時才給予補充凝血因子:血友病A的按需治療的劑(量重組凝血因子Ⅷ):體重(kg)X所需要提高的因子活性水平(%)÷2,血友病B的按需治療的劑量(重組凝血因子Ⅸ):體重(kg)X所需要提高的因子活性水平(%)。分別通過問卷調(diào)查、電話追蹤及定期復診、隨訪,同時給血友病病人進行定期的關(guān)節(jié)超聲檢查,兩組進行對比,將調(diào)查表所收集的患者臨床信息資料及超聲檢查結(jié)果運用SPSS22.0統(tǒng)計軟件進行數(shù)據(jù)統(tǒng)計分析,得出云南省血友病患者關(guān)節(jié)病采取預防治療的預后。[結(jié)果]1、關(guān)節(jié)的結(jié)構(gòu)狀態(tài):通過觀察11例小劑量預防治療的血友病患者關(guān)節(jié)超聲檢查情況及9例按需治療的血友病患者的關(guān)節(jié)病變情況來分析預防治療與按需治療的區(qū)別,通過關(guān)節(jié)超聲(HA)評估方法來評估,其評估兩組患者以下幾個方面的區(qū)別:滲出:關(guān)節(jié)積液或積血、纖維隔膜、滑膜增生、滑膜增厚伴滑膜血管增生、含鐵血黃素沉積。結(jié)果顯示預防治療組患者的關(guān)節(jié)損害較按需治療組輕。2、關(guān)節(jié)年出血次數(shù)情況:預防治療組的年關(guān)節(jié)出血次數(shù)的均數(shù)為4.29(次),按需治療組的年關(guān)節(jié)出血的均數(shù)為15.14(次),兩獨立樣本t檢驗P=0.0010.05,說明二者存在統(tǒng)計學意義,血友病患者預防治療能明顯減少患者關(guān)節(jié)出血次數(shù)。3、年靶關(guān)節(jié)出血數(shù)目:通過統(tǒng)計11例預防治療血友病患者及9例按需治療血友病患者在半年研究期間靶關(guān)節(jié)出血關(guān)節(jié)數(shù)目,再乘于2得出年總出血關(guān)節(jié)數(shù)目,具體檢查結(jié)果為:預防治療組的年關(guān)節(jié)出血數(shù)目的均數(shù)為1.5(個),按需治療組的年關(guān)節(jié)出血數(shù)目的均數(shù)為4.0(個),隨機設(shè)計兩獨立樣本t檢驗P=0.0030.05,說明預防治療可以減少把關(guān)節(jié)年出血數(shù)目。4、因子使用情況:計算每年凝血因子使用量(IU/kg年),結(jié)果顯示:預防治療組年凝血因子使用量均數(shù)為682.7(IU/kg年),按需治療組年凝血因子使用量均數(shù)為886.5(IU/kg年),隨機設(shè)計兩獨立樣本t檢驗P=0.1520.05,二者無統(tǒng)計學意義。但預防治療組的年因子平均使用量較按需治療組的年因子使用量少,說明預防治療組年使用因子量還是有減少的趨勢。5、患者的生活質(zhì)量:通過PeDsQL調(diào)查表和病假時間(d/半年)來評價:5.1、使用PeDsQL調(diào)查表,采集了 11例(年齡小于14歲兒童)血友病患者的生活情況,其中5例使用預防治療,6例使用按需治療的方案,結(jié)果使用四格表確切概率法檢驗,統(tǒng)計結(jié)果顯示:P=0.2420.01,無統(tǒng)計學意義,雖無統(tǒng)計學意義,但預防治療組的有效率(60%)較按需治療組有效率(15%)有增加趨勢,說明預防治療組有改善生活質(zhì)量的趨勢。5.2、年病假時間(d/年)。統(tǒng)計了 31例患者(兒童、成人均有),其中小劑量預防治療組有16例,按需治療組有15例,統(tǒng)計出其半年的病假日,再乘于2得出一年的病假日,結(jié)果顯示:小劑量預防治療組的病假日均數(shù)是12.1(天),按需治療組的病假日均數(shù)是39.6天,隨機設(shè)計的兩獨立樣本t檢驗,P=0.015,其P值小于0.05,統(tǒng)計學有意義,小劑量預防治療可以減少血友病的病假時間。6、血漿Ⅷ/Ⅸ因子抗體(抑制物)產(chǎn)生率:統(tǒng)計分析了 16例預防治療組與15例按需治療組治療中半年時間復查的抑制物產(chǎn)生情況,分析預防治療的安全性,統(tǒng)計結(jié)果顯示:兩組均未出現(xiàn)抑制物陽性患者病例。[結(jié)論]本實驗通過對照研究得出以下結(jié)論:血友病患者中使用預防治療可以減少血友病患者關(guān)節(jié)結(jié)構(gòu)和功能的損傷、減少關(guān)節(jié)年出血次數(shù)、減少年靶關(guān)節(jié)出血數(shù)目、減少病假日;預防治療及按需治療相比,預防治療組有可以減少年凝血因子使用量的趨勢;預防治療組的生活質(zhì)量有改善的趨勢;兩組中均未見血漿Ⅷ/Ⅸ因子抗體(抑制物)產(chǎn)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).

【學位授予單位】：昆明醫(yī)科大學
【學位級別】：碩士
【學位授予年份】：2017
【分類號】：R554.1;R684

【參考文獻】

相關(guān)期刊論文 前10條

1 謝曉艷;孫梅;顧健;;重組凝血因子Ⅷ預防治療血友病A模式的現(xiàn)狀及展望[J];臨床血液學雜志;2016年05期

2 應益昕;;中國工程院院士、傳染病學專家李蘭娟:應盡快實現(xiàn)血友病預防治療[J];科學新聞;2016年04期

3 嚴紅;曾凡一;;血友病B替代治療藥物研究現(xiàn)狀及進展[J];生物工程學報;2016年02期

4 胡群;;再談兒童血友病預防治療[J];中國小兒血液與腫瘤雜志;2016年01期

5 張靜;王淑紅;劉軍;;48例兒童血友病A低劑量預防治療的效果評價[J];血栓與止血學;2015年05期

6 張慶;舒慧英;周敏;徐鳴;李曉靜;;四川省兒童血友病臨床資料及生活質(zhì)量分析[J];華西醫(yī)學;2015年09期

7 張鵬;;兒童關(guān)節(jié)滑膜增生病變的MRI影像表現(xiàn)[J];山東醫(yī)藥;2015年15期

8 李軍;丁小玲;呂冰梅;薛峰;孫慶利;郭新娟;李東爽;張文鳳;張文君;徐雨霞;楊仁池;;血友病性膝關(guān)節(jié)病的超聲特征[J];中華血液學雜志;2014年05期

9 唐凌;張紀水;吳潤暉;;中、重度合并關(guān)節(jié)病變血友病患兒主觀生活質(zhì)量與其家庭負擔評價[J];血栓與止血學;2014年01期

10 吳潤暉;;兒童血友病診斷與治療[J];中國實用兒科雜志;2013年09期

相關(guān)會議論文 前2條

1 李軍;呂冰梅;丁小玲;;高頻超聲評估血友病性肘關(guān)節(jié)改變[A];中國超聲醫(yī)學工程學會成立30周年暨第十二屆全國超聲醫(yī)學學術(shù)大會論文匯編[C];2014年

2 李軍;丁小玲;;血友病關(guān)節(jié)病膝關(guān)節(jié)損傷的超聲評價[A];中國超聲醫(yī)學工程學會第十一屆全國超聲醫(yī)學學術(shù)大會論文匯編[C];2012年

相關(guān)碩士學位論文 前4條

1 楊春晨;血友病患者的基因診斷及發(fā)病機制的研究[D];蘇州大學;2015年

2 宋玉;云南省97例血友病人生存質(zhì)量調(diào)查結(jié)果分析[D];昆明醫(yī)科大學;2015年

3 黃順樺;重型血友病A成人患者短期低中劑量三級預防治療的比較研究[D];南方醫(yī)科大學;2015年

4 王寧辰;中文版兒童生活質(zhì)量測定量表（PedsQL~(TM)3.0）風濕模塊（8-12歲）在我國風濕性疾病患兒中的適用性研究[D];北京協(xié)和醫(yī)學院;2014年

，

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