March is血友病宣传月, as designated by President Reagan in 1986.Dr. Barbara Konkleis interim director of ourHemophilia Care Programand describes Puget Sound Blood Center’s work in treating hemophilia and other bleeding disorders.
Hemophilia is a bleeding disorder that results from either not having a protein that’s needed for clotting or having an abnormal clotting protein.
一件事与hemophilia, and it relates to other bleeding disorders, is the perception that women don’t have bleeding disorders. Hemophilia is an x-linked disorder, so men are more severely affected, but women can also be affected with hemophilia, and they can have other bleeding disorders.
The other misconception is the is expectation that someone with hemophilia will look abnormal and they don’t. With current therapy, persons with hemophilia can lead normal lives – they can do basically what anyone else can do.
The clinical program provides comprehensive care to patients with bleeding disorders, including hemophilia; we have physicians and nurses, a social worker, physical therapy, and support staff that allows us to assess the needs of the patients and support them in their treatment. Many patients treat themselves at home with factor replacement medicine that they have to put in their vein, so the patients and families have to learn how to do this. The comprehensive care model allows us to support them in this treatment and other needs.
有一段期前，这一模型允许患者更加独立，也允许他们活得更长时间。PSBC的研究与临床计划非常密切相关。我们正在测试更好的方法来诊断和监测血友病，von Willebrand疾病和其他出血障碍，以及与行业合作，正在评估治疗出血障碍的新产品，包括血友病B的基因治疗。我们领导国家学习评估血友病老年人的心脏病和血液凝血问题。我们与南加州大学和其他机构的研究人员合作，帮助了解血友病患者的挑战和费用。我们的物理治疗师Lorraine Flaherty正在研究如何减少血友病和改善肘部问题的物理治疗的方法。
We are the central laboratory and a Steering Committee partner for a national project, called theMyLifeOurFuture program。该项目旨在确定美国所有患者中血友病的遗传原因，最终为可能是血友病载体的雌性进行测试。通过知情同意，我们正在收集和存储在储存库中的血液样本和数据，该储存库将在未来进行全国范围内用于研究血友病。
I got interested in bleeding disorders when I was a resident in internal medicine. I found the medical problems the patients face and the close link to findings in laboratory testing fascinating. Hemophilia is a chronic disease where our goal is to work with the patient and family to minimize symptoms, so they can lead a normal life. This is very rewarding. I started in a hemophilia clinic as a fellow 30 years so, so this is my 30thyear in hemophilia.
The most significant changes – because I treat adults with hemophilia – we now have young men who are coming to our clinic and they have normal joints and no other signs of past bleeding. They’re just doing phenomenally well. In our young adult patients we are now seeing the benefits of the widespread use of prophylaxis therapy from a young age.
I entered hemophilia the year before the HIV virus was defined, and that was a terrible, terrible time in hemophilia with many patients dying of AIDS. It was before we had effective treatment for HIV infection, and we lost a lot of patients. No clotting factor products have transmitted the HIV virus since 1985 and none have transmitted hepatitis since around 1990. Many older patients are still struggling with these infections, although treatment is much improved. Fortunately our younger patients don’t have to deal with these infections.
You child should have a normal life expectancy and should be able to do what other children are doing.
When I see an 18, 19-year-old, we have to expect that he will live to 80 or longer, so I have to be sure that we do all the right things so that he can get down on the floor and play with his great-grandchildren. That’s our goal.
我们对血友病的大部分治疗不再来自血液产品。这在过去至关重要，这就是为什么血友病计划通常与血液中心联系起来。虽然现在通过重组DNA技术生产的许多治疗产品，但具有出血障碍的儿童和成人仍然有出血和需要血液的风险，因此即使我们使用它们较少，捐赠仍然对血友病仍然非常重要。此外，我们的一些患者含有罕见的出血障碍仍然依赖于捐赠的血液产品进行治疗。yabo国际
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