Barr Ronald D, Saleh Mahassen, Furlong William, Horsman John, Sek Julia, Pai Mohan, Walker Irwin
McMaster University Medical Center, Hamilton Health Sciences Corporation, 1200 Main Street West, Hamilton, Ontario L85 4J9, Canada.
Am J Hematol. 2002 Nov;71(3):152-60. doi: 10.1002/ajh.10191.
The hemophilias are a group of disorders associated with a chronic burden of morbidity and early mortality. Improvements in these adverse features have been achieved by the use of clotting factor concentrates within comprehensive centers of specialized care providing home infusion programs. Offsetting effects from transfusion-transmitted hepatitis and HIV infection are in recent decline. The net impact of these changes merits assessment. To test the a priori hypotheses that increasing severity of factor VIII deficiency would be associated with an increasing burden or morbidity and that hepatitis and HIV positivity would impair health status further, a cross-sectional study of a population-based cohort was undertaken in a regional hemophilia program in Ontario, Canada. A survey was made of mild, moderate, and severe hemophiliacs over 13 years of age who self-reported their health status using a standard 15-item questionnaire. The responses were converted to levels in the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3) health status classification systems to form multi-element vectors from which single-attribute morbidity and overall health-related quality of life utility scores were determined. The burden of morbidity was greater in hemophiliacs than in the general population and correlated with the category of disease (mild < moderate < severe). Hepatitis and HIV positivity conferred additional burdens of morbidity, which were mainly in the attributes of mobility (HUI2), ambulation (HUI3), and pain (HUI2/3), all of these differences reaching levels of statistical significance. Despite demonstrable improvements in the safety, effectiveness, and utilization of clotting factor concentrates, hemophiliacs continue to experience an important burden of morbidity. Measurement of this burden, as reported here, provides a basis for future economic evaluation of the costs and consequences of health care interventions provided to this population.
血友病是一组与慢性发病负担和早期死亡率相关的疾病。通过在提供家庭输液计划的专业综合护理中心使用凝血因子浓缩物，这些不良特征已有所改善。输血传播的肝炎和艾滋病毒感染的抵消作用最近有所下降。这些变化的净影响值得评估。为了检验先验假设，即因子VIII缺乏症严重程度的增加会与发病负担的增加相关，以及肝炎和艾滋病毒阳性会进一步损害健康状况，在加拿大安大略省的一个地区血友病项目中，对一个基于人群的队列进行了横断面研究。对13岁以上的轻度、中度和重度血友病患者进行了调查，他们使用标准的15项问卷自我报告健康状况。将回答转换为健康效用指数Mark 2（HUI2）和Mark 3（HUI3）健康状况分类系统中的水平，以形成多元素向量，从中确定单属性发病率和与总体健康相关的生活质量效用得分。血友病患者的发病负担高于一般人群，且与疾病类别相关（轻度<中度<重度）。肝炎和艾滋病毒阳性带来了额外的发病负担，主要体现在活动能力（HUI2）、行走能力（HUI3）和疼痛（HUI2/3）方面，所有这些差异均达到统计学显著水平。尽管凝血因子浓缩物在安全性、有效性和利用率方面有明显改善，但血友病患者仍继续承受着重要的发病负担。如本文所述，对这一负担的测量为未来对该人群提供的医疗保健干预措施的成本和后果进行经济评估提供了依据。
