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Frequently Asked Questions
1. What is hemophilia?
Hemophilia is a rare sex-linked, hereditary coagulation disorder that causes excessive bleeding to occur spontaneously and/or after injury. Bleeding episodes may be external or internal into joints, muscles, the abdominal cavity, the brain, and other organs. The bleeding episodes are painful, and unless treated promptly and adequately, can lead to crippling deformities of the knees, ankles, and joints.
2. What causes hemophilia?
Hemophilia is caused by a defect in the gene located on the X chromosome that contains the genetic code for one of the clotting factor proteins necessary for normal clotting. In normal blood clotting, a complex interaction of at least 14 plasma proteins (factors) results in a gelatinous, fibrillar plug that seals leaks in damaged blood vessels. One of these proteins is lacking or deficient in people with hemophilia and their blood does not form a firm clot.
3. If a person with hemophilia cuts his/her finger, will he/she bleed to death?
No. Just apply pressure and/or ice as you would anyone else. The bleeding will stop, but it will just take a little longer than with someone who does NOT have a coagulation disorder.
4. Is hemophilia a communicable disease?
No. It is a genetically sex-linked disorder. The defective gene is carried by the mother and passed on to her sons with a 50% chance of inheritance and her daughters have a 50% chance of inheriting carrier status. If a male with hemophilia has children, all of his sons will be normal and all of his daughters will be carriers.
5. Is there only one kind of hemophilia?
No. There are several different types of hemophilia. The two most prevalent are Hemophilia A, sometimes called Classical hemophilia which is a lack of Factor VIII, and Hemophilia B, sometimes called Christmas Disease and is a lack of Factor IX. It is rare for a female to present with Hemophilia A or B, but it does happen. Factor XI is sometimes called Hemophilia C and affects both men and women. Von Willenbrand Disease is yet another form of hemophilia that is prevalent in males and females. There are also some rare forms with other factors that are missing such as Factor V, X and XIII.
6. If a woman is NOT a known carrier, can she have children who have hemophilia?
Yes. More than 1/3 of the newly diagnosed cases of hemophilia have no previous family history and are considered mutations.
7. What is the incidence of hemophilia?
One in 5,000 live male births. There are approximately 20,000 diagnosed hemophiliacs in the United States today.
8. Are there advocacy organizations for persons with hemophilia?
Yes. Hemophilia Federation of America (HFA), National Hemophilia Foundation (NHF), Committee of Ten Thousand (COTT) and Coalition for Hemophilia B plus dozens of statewide and/or area organizations. For more information, see the "Links" section of this web site.
9. Is it true that more than one half of the hemophilia population got HIV through the blood supply?
Yes. Due to negligence on the part of the FDA and the manufacturers (See Institute of Medicine Report, July, 1995), HIV was allowed to be present in blood and blood products and because of the massive use of coagulation products, the persons with hemophilia were known as the "canaries in the coal mines."
10. Why do so many people with hemophilia also have hepatitis C?
Basically, the same reasons as stated in Item 9 regarding HIV. Until the last few years, the testing for Hepatitis C in blood donors and a process to remove Hepatitis C from the blood were not perfected.
11. Is treatment expensive?
YES! Average treatment can run upwards of $250,000 annually. It is even more if a person has an inhibitor.
12. What is an inhibitor?
A simple way to explain an inhibitor is that the body rejects the newly infused coagulation product. It acts in much the same way that a body rejects a donor organ. In this case, you have to circumvent the defective factor in order to cause coagulation. Sometimes physicians will use Immune Tolerance Therapy to achieve this end.
13. What is Immune Tolerance Therapy?
Massive doses of coagulation product are infused into the patient to attempt to "override" the rejection.
14. Do Lifetime Insurance Caps affect a person with hemophilia adversely?
Absolutely. Lifetime insurance caps originated in 1972 at $1 million per claimant. That was 35 years ago and these caps have not been adjusted for inflation.
15. How does a person know how much product to infuse for a bleed?
There are charts available that will give the individual an amount per kilogram of body weight as related to the type of bleed. Some lesser bleeds are treated with a 30-40% correction and they go all the way up to 100% correction for severe trauma or head bleeds.
16. Why is it necessary for patients to keep a log of bleeds and infusions?
Patients should always keep a log of bleeds and infusions. It is very helpful in predicting trends of bleeds, connecting bleeds to activities of daily living or extraordinary events, tracking use of infected factor.
17. Would there be an advantage for a patient to track bleeds and infusions electronically through use of a computer or PDA?
Yes. Records could be sent to the physician prior to a visit and help to ascertain trends in bleeds, determine whether the patient is treating each bleed efficiently, what precautions should be considered to avoid repetitive bleeds, etc.
18. Why are patients encouraged to access treatment at a Hemophilia Treatment Center?
These are usually federally funded centers with a multi-disciplinary approach to treating hemophilia. Specialists in various disciplines see the patient including pediatrics or internal medicine, orthopedics, dentistry, social work, physical therapy, etc. Reports are given to primary physician and on-going trends are followed. These people are the experts in their fields in hemophilia treatment.
19. Why do some patients choose not to access care at their HTC?
Some patients live a long way from the facility and physical access is difficult. Some patients feel they have more than adequate treatment at the local level and don’t need the HTC.
20. Why do some people (especially children) have a port implanted for venous access?
The main reasons for installation of a port or good venous access without repetitively "sticking" the vein and risking damage and ease of access for parents of small children who may not be comfortable with this process.
21. How do you decide to have a port or not?
You should always research the pros and cons. Some things to consider are convenience, infection control, patient comfort, confidence.
22. Why is it important for patients to have choice of physician, provider and product?
Hemophilia treatment is not a "one-size fits all" situation. Some people are more comfortable with one physician than another. Physical access comes into play in some instances. Choice of provider is usually a personal preference or convenience issue. Some patients respond better to one product than another. Patients should always have a choice of monoclonal (plasma derived) product or recombinant product when availability of supply is not a problem.
23. Is there a recommended age at which parents should learn to infuse their children and/or children should learn to infuse themselves?
There is no set age or time. It is a decision usually made in consultation with the physician. There are many circumstances that need to be evaluated before this decision can be made.
24. Do all hospitals stock coagulation products? If not, why not?
Fewer and fewer hospitals are stocking coagulations products in today’s market. They are very expensive inventory items and the majority of patients keep their own factor at home for ready access. Reimbursement at the hospital level is also becoming less and less and this discourages hospitals from investing in such high price inventory.
25. Is there somewhere patients who need assistance can go to for help?
Financial assistance in a crisis can be obtained, with limitations, by calling Hemophilia Federation of America Helping Hands fund. The phone number is 1-800-230-9797. Some local chapters also have charitable assistance funds.
26. If a patient is having difficulty obtaining insurance coverage, is there a place to go to for help?
Yes. Call Patient Services Incorporated at 1-800-366-7741 or visit their web site http://www.uneedpsi.org/.
27. If I need assistance in applying for SSI, where can I obtain that assistance?
Call your local chapter, HFA or the ACCESS program. HFA’s phone number is 1-800-230-9797. ACCESS program's number is 1-888-700-7010.
28. What is a home care company and how do I choose one?
Home care companies provide door-to-door delivery of coagulation products and supplies, education in hemophilia treatment and resources, nursing services and support and many other resources. There is a list of home care companies in the Resources area of our web site. Pick several and have an interview with each of them. Determine what services they offer and compare what they charge your insurance company in reimbursement. See what company best fits your needs and has the closest staff to be of assistance to you.
29. What is a 340B program and what are the benefits of these programs?
340B is a program that can purchase factor at reduced rates because that program has received a federal grant or is a hospital with a disproportionate share of lower income patients. The purpose is to provide factor at reduced rates and pass along the revenue to the hemophilia program itself and spare major hits on the patient’s lifetime caps. A danger signal to watch for is when a 340B program obtains the factor at reduced rates but does not pass the savings along to the patient which would save on a lifetime cap and/or the revenue does not go back into the funds for the hemophilia program.
30. Does my physician have the right to only provide a prescription for one product of his or her choice?
No. You are entitled to have a choice of product (brand and type) and a provider (source of your products.)
31. Where can I find support?
Local chapters, state chapters, HFA, NHF, Coalition for Hemophilia B and the Committee of Ten Thousand (COTT) all have programs of support. Attend educational sessions staged by your local chapters, by HFA and by NHF. Check content and other plusses such as child care, relevance of content of programming, convenience of access, etc.
32. What is the "settlement" I hear people talking about?
A financial settlement offered by the manufacturers to persons and/or their families who were infected by HIV through their blood products due to negligence. It paid $100,000 per infected patient or surviving family.
33. What is the Ricky Ray legislation?
Similar to the "settlement" however this was paid by the federal government due to negligence by the FDA and other governmental agencies. It also paid $100,000 per infected patient or their surviving parent, spouse or child.
34. Why are large home care companies purchasing smaller ones?
Reimbursement has become a problem. If a company has more patients they can usually purchase factor at a lower price, so many are merging in order to have a bigger patient base. Hopefully, this will benefit the patients in the long run though it does restrict choice.
35. What is BPAC?
Blood Products Advisory Committee is part of the FDA (Federal Drug Administration) and it monitors manufacturing of blood products and devices for purity and safety. It is basically a licensing agency.
36. What is BSAC?
Advisory Committee on Blood Safety and Availability is a committee of Health and Human Services and monitors the safety and availability of our nation’s blood supply.
37. What do these initials represent?
CDC – Centers for Disease Control
CMS – Centers for Medicaid and Medicare Services – formerly HCFA (Healthcare Financing Administration)
FDA – Food and Drug Administration
GAO – General Accounting Office of our nation’s government. They deal in some reimbursement matters
MCHB – Maternal and Child Health Bureau – overseers of Hemophilia Treatment Centers
HHS – Health and Human Services
HRSA - Health Resources and Services Administration – MCHB is part of this agency Printer Friendly Send to a Friend!
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