Prophylaxis versus on-demand regimens to prevent arthropathy in real-life patients with hemophilia A

論文翻譯標題: A型血友病患者接受預防性治療與需要性治療保護關節病變之比較效果研究
  • 賴 仕杰

學生論文: Master's Thesis


Introduction Hemophilia is an inherited bleeding disorder resulting from the absence or deficiency of coagulation factors There are two major types of hemophilia hemophilia A and hemophilia B Since the introduction of clotting factor concentrates regular prophylaxis and more comprehensive care have led to an increased life expectancy of persons with hemophilia (PWH) The National Health Insurance Program has updated the reimbursement guideline for the use of prophylaxis therapy inpatient with hemophilia in 2014 However there is no sufficient evidence to support what the implement status nowadays and how treatment behavior changed after new national guideline being introduced Nonetheless the effectiveness of prophylaxis regimen of hemophilic arthropathy in Taiwan remains unknown Objective The objectives of the study are as the following: (1) To investigate population-based epidemiologic data of hemophilia in Taiwan including prevalence age distribution incidence rate and mortality rate of hemophilia patients in Taiwan (2) To analyze utilization patterns of coagulation factor for hemophilia patients including the dosage cost and utilization of healthcare resource including the number of visits and related medical costs (3) To study health outcomes of patients with hemophilia including the annual bleeding rate of patients with hemophilia and the time of joint outcomes (e g arthropathy and joint replacement) for hemophilia patients from their birth date (4) To explore the comparative risk of arthropathy between prophylaxis vs on-demand regimens Materials and methods We used the Taiwan National health Insurance Research databases for the following studies Firstly we constructed cohorts from 2003 to 2015 of diagnosed hemophilia patients to explore the epidemiologic data Secondly we used data of male patients with hemophilia to conduct the research of utilization pattern of health care resource and factor concentrate Thirdly we estimated the health outcome of male patients with hemophilia A Lastly we used conditional logistic regression to compare the risk of arthropathy between patients with prophylaxis and patients with on-demand regimen In this study we used age and the different dosage group to match case and control Results Firstly we identified 1184 patients with hemophilia The number of patients were 1040 in 2003 and increased to 1092 in 2015 The prevalence of patients with hemophilia was estimated 4 6 per 100 000 people while male patients with hemophilia was estimated 7 3 per 100 000 male in 2015 The newborn incidence ranged from 1 9 to 10 3 per 100 000 newborn while the male newborn incidence ranged from 3 6 to 19 5 per 100 000 male newborn The number of deaths ranged from 5 to 12 and the standardized mortality ratio was1 8 in patients with hemophilia A and hemophilia B In the second part the total medical cost of male patients with hemophilia was 1 0 billion NTD to 3 3 billion NTD from 2003 to 2015 The median number of visits using factor concentrate in 2015 was 6 in PWHA and 7 in PWHB The cost of factor concentrate and bypassing agents (Factor VII and aPCC) accounted for 96% to 98% of total medical cost In addition the mean bleeding rate of patients with hemophilia A was 4 5 (SD 12 5)in 2015 The mean age of first diagnosis of arthropathy in patients with hemophilia A was 30 9 (SD 17 9)years old In the nested case-control analysis we included 512 cases and 512 controls with male gender and hemophilia A We then divided these patients of both cases and controls into prophylaxis groups and on-demand groups There were 79 and 433 cases in prophylaxis and on-demand groups We used conditional logistic regression model to compare the odds between cases and controls We found that the point estimate was 4 44(95% CI:=0 26-7 63) We further analyzed the subgroup study population whose were between 2 and 12 years old and we found that the point estimate was 0 7 (95% CI:= 0 27-1 84) Conclusions The results of the epidemiologic data utilization pattern and health outcome were consistent with previous studies The findings indicated the average ages of patients were older by years which implicated the increased life expectancy of persons with hemophilia Therefore age-related disorders will be the possible issue in the future We found the use of by passing agents was increase by years which warrants attention on the effectiveness and related issues As time passed by we found the use of factor concentrate and the medical cost increased by years which highlights the importance for assessing the effectiveness or cost-effectiveness for the replacement therapy Although there was no statistical significance we found prophylaxis regimen tended to have better effectiveness than on-demand regimen in patients aged 2 to 12 years which may imply that the early use of the prophylaxis regimen may pose some benefits in patients with hemophilia Key words: Hemophilia Coagulant factor Arthropathy Prophylaxis regimen Nested case control study
獎項日期2018 八月 21
監督員Edward Lai (Supervisor)