Fall 1998 - Factor Nine News

On August 27, Surgeon General David Satcher, MD announced that the U.S. Public Health Service Blood Safety Advisory Committee had reached a unanimous conclusion that blood and blood products from donors who subsequently developed Creutzfeldt-Jakob Disease (CJD) would no longer need to be quarantined and withdrawn from the market except in a few special circumstances. The advisory committee concluded that there is no evidence that CJD can be transmitted by blood or blood products and that the large number of withdrawals over the past few years have caused serious shortages in the supply of plasma products. For instance, shortages of immune globulins that have actually led to illness and death according to the Immune Deficiency Foundation.

However, the National Hemophilia Foundation (NHF) immediately stated its opposition to this new policy as did some other patient advocacy groups. The NHF believes that there is insufficient evidence that CJD is not transmitted by blood products. Because of this and other disease-transmission issues they recommend that patients switch to recombinant clotting factors whenever possible. However, there is currently a severe shortage of recombinant product and prices are high.

What is the hemophilia patient to think and do about this? First of all, DON’T PANIC! Unlike AIDS, CJD is an ancient, extremely rare disease. The nation’s blood supply has probably always contained blood from donors who later developed CJD, and yet as hard as researchers have looked they have never been able to identify even one case of transmission of CJD from blood or blood products. In spite of the large amount of blood products they use, no person with hemophilia has ever been diagnosed with CJD.

CJD causes a very rare form of dementia due to damage to the brain. It affects about one person in a million. It is 100% fatal, and its victims only live an average of eight months from the onset of symptoms. There is no effective treatment and no test for the disease. The initial diagnosis is made based on the patient’s symptoms which are similar to those of Alzheimer’s disease, but the patient’s condition degrades much more rapidly. The diagnosis can only be confirmed after death by an autopsy on the brain. The actual cause is unknown. One popular theory is that it is caused by a prion, an infectious protein that is neither virus or bacteria. Although 5 - 10% of cases are inherited, the other 90% seem to appear spontaneously with no apparent reason for contracting it. CJD primarily appears in people over 60 years old.

CJD belongs to a class of diseases called Transmissible Spongiform Encephalopathies (TSE’s) which includes scrapie in sheep and Bovine Spongiform Encephalopathy (BSE), also known as “Mad Cow Disease”, in cattle. Although scientists have been studying CJD since the 1920’s, it and other TSE’s have received much attention in the past few years partly due to the concern in Britain about eating beef that might be infected with BSE. In fact, several cases of a disease called new variant CJD (nvCJD) which appears to be related to BSE have been diagnosed in Britain. nvCJD occurs in much younger people and is probably quite different from classical CJD. Because so little is known about nvCJD, FDA will still require withdrawal of products made from donations from people who develop nvCJD. There are currently no known cases of nvCJD in the U.S.

Research on the potential for CJD being transmitted by blood products has taken two major forms, surveys of donors and recipients and animal studies. The studies of donors and recipients are probably the most satisfying right now. Researchers in the U.S. and Europe have tracked down people who received blood or blood products from donors who later died of CJD and have seen no evidence of transmission. A recent study by the Centers for Disease Control along with hemophilia treatment centers across the U.S. looked specifically at hemophilia patients who had died of neurological disorders to see if any might have had CJD. This study also found no evidence of CJD.

Experiments on CJD are extremely difficult to perform, but researchers are starting to chip away at the problems bit by bit. One major obstacle is that there is no good test to detect CJD. The only way to see if a sample is infectious is to inject it into the brains of mice and wait several months to see if they develop the disease. In one study, human blood known to contain CJD infectivity was transfused into chimpanzees with no evidence of disease transmission. Another recent study in mice and hamsters showed that infectivity is carried in blood, mainly associated with the white blood cells, but some is also present in the plasma. The plasma­associated infectivity has been shown to be reduced markedly during some of the manufacturing processes used to produce plasma products. Many more studies need to be done to understand whether CJD can be transmitted by intravenous injection of plasma products. The historical evidence suggests that it is not transmitted, but scientists need to determine why not and if there are any conditions under which it could be transmitted.

In summary, a great deal of research is going on to learn more about CJD and how it is transmitted. Studies on blood and blood product recipients have shown no evidence of CJD transmission. There is much still unknown, but it does not appear that there is a significant risk from CJD for plasma products. FDA has agreed to meet with NHF in October to review all of the available scientific data. The individual patient should probably feel confident continuing his current treatment, and if he has any questions or concerns, he should consult his doctor.

Join the lovable cartoon character Dom the Domino as he teaches you all about hemophilia in this fund, easy-to-use computer tutorial. Learn about genetics what happens inside the body during a bleeding episode, how clotting factor is made, guidelines for physical activity and much, much more. “Learn about Hemophilia” is a great way for all family members and friends to learn about bleeding disorders. This learning tool is a collaborative effort between Roberta Poulton, RN, of St. Agnes Hospital Hemophilia Program in Baltimore, Maryland, and Hemophilia Resources of America. The CD-ROM is Windows and MAC compatible. To received a free disk, call Hemophilia Resources of America, 387 Passaic Avenue, Fairfield, NJ 07004, Telephone (800) 549-2654.

“Learn about Hemophilia” will be on display at the Hemophilia Resources of American booth at the 50th Annual National Hemophilia Foundation Meeting in Orlando.

A father of a 16 month old child with severe hemophilia and Vwd is looking to purchase a Toppen 77 protective helmut. The helmut was manufactured by a Swedish company. The United States distributor is Am-AIDIS Inc.

Any information or leads would be most appreciated. Please contact Mike via internet at mcimato@RSA-NET.COM or call Kim Phelan at (212) 554-6823.

We look forward to seeing you at the 50th Annual National Hemophilia Foundation meeting this year in Orlando, Florida October 22 -24, Please make sure you stop by our booth to say HELLO!