By KAREN BARROW

Published: January 30, 2012

Hemophilia, which affects 20,000 Americans, is a genetic disorder in which the body lacks a clotting factor needed for blood to coagulate. Depending on severity, it can lead to life-threatening, seemingly unprovoked bleeding episodes — or it can go undetected for years, until a serious injury or a routine medical procedure suddenly touches off uncontrolled bleeding.

Hemophilia is more common in boys and men. But some girls and women have the disorder, and it can cause complications during menstruation and childbirth.Hemophilia (actually a group of disorders, marked by different missing blood factors) has no cure. But treatment has improved substantially in recent years. Today many bleeding episodes are prevented by prophylactic injections of the missing factor; until this treatment was developed, patients had to rely on transfusions after a bleeding episode, leaving them vulnerable to infectious diseases and chronic joint damage.

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Go to this link to view Patient Voices: Hemophilia a multimedia feature in the New York Times Health section online.

N Engl J Med 2012; 366:281-283January 19, 2012

ArticleTo the Editor:
Patients with hemophilia A (a deficiency of factor VIII [FVIII]) have spontaneous bleeding because of abnormal thrombin generation, which results in the formation of weak, unstable clots.1 The formation of these weak clots is also the result of delayed and reduced activation of coagulation factor XIII (FXIII).2 The standard treatment is based on FVIII substitution to control and prevent bleeding, but this process is expensive and time-consuming. We hypothesized that supraphysiologic levels of FXIII would normalize clot stability at low levels of FVIII.

Clot stability was recorded by means of the changes in plasma turbidity after the simultaneous addition of tissue factor (dilution, 1:40,000) and tissue plasminogen activator (0.75 nM). FVIII-deficient, platelet-poor plasma, spiked with increasing concentrations of recombinant FVIII plus plasma-derived FXIII or buffer, was compared with normal control plasma. The primary end point was the area under the curve (AUC) for turbidity.

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Study of gene therapy developed at UCL and St. Jude Children’s Research Hospital offers first proof adults with haemophilia B benefit from treatment, reducing need for injections with clotting factor to prevent bleeds.

Symptoms improved significantly in adults with the bleeding disorder haemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children’s Research Hospital in Memphis, US and demonstrated to be safe in a clinical trial conducted by UCL.

The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear online ahead of print today in the New England Journal of Medicine. The research is also scheduled to be presented on 11 December at the 53rd annual meeting of the American Society of Hematology in San Diego, US.

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November 16, 2011

Baxter Healthcare Corporation has sent a notice of a labeling error affecting the expiration date of the Sterile Water for Injection that is packaged as a diluent with RECOMBINATE [Antihemophilic Factor (Recombinant)]. The Sterile Water for Injection label indicates that the shelf life is longer than what was actually approved by the FDA.

Please continue to use the RECOMBINATE product as labeled on the kit. There is no impact to the safety or efficacy of Recombinate if the Sterile Water for Injection diluent is used before the expiration date of the RECOMBINATE.

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Practical advice to help plan airplane trips

By Elizabeth Thompson Beckley | 06.24.2010
Originally Published January 2007 | Updated June 2010

Hopping a plane can be stressful nowadays, as you deal with long check-in lines, baggage fees and enhanced security. But for people with hemophilia and other bleeding disorders, the biggest concern is often how to transport their factor products and supplies safely to their destination. Here’s what you need to know about traveling with medication.

After the discovery of a 2006 London terrorist plot involving liquid explosives, airlines limited the amount of liquids, aerosols and gels travelers can carry on board airplanes. This regulation particularly affects passengers who need to keep medical supplies with them. This includes clotting factor products, needles, syringes and other supplies required for treatment of bleeding disorders. Being prepared with complete information about your medications, and packing them properly, will go a long way toward smoothing the security screening process.

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Medical Advisory #414
October 26, 2011

WARNING ABOUT USE OF NON-STERILE ALCOHOL WIPES

In January 2011, NHF issued a Medical Advisory (#412) about a recall of non-sterile alcohol wipes produced by one company, Triad. In April, the FDA forced this company to close. Now, additional infections have occurred in patients using non-sterile alcohol wipes manufactured by another company, Pacific Disposables International (PDI). This company has also been required by the FDA to recall their products. Both of these companies package their products under their own label and under other companies’ labels.

At this time, it appears prudent to recommend that all individuals who have alcohol wipes or other prep pads/swabs in their home examine the individual packages to see if they contain the word “Sterile.” This includes wipes that are packaged with factor or other IV preparation kits. If the word “sterile” does not appear on the package, there is no way to determine if they are in fact sterile or not. Therefore, individuals should discontinue use of any wipes that do not state “sterile” and request replacement with sterile wipes from their clotting factor distributor.

Nurses should also check their supply of alcohol wipes to ensure that they are only using sterile wipes. This is particularly important for patients with indwelling venous access devices (catheters or ports) and for patients with compromised immune systems, but should be standard of care for all patients.

By JoNel Aleccia
Health writer

msnbc.com
updated 10/10/2011 8:46:40 AM ET 2011-10-10T12:46:40

A massive recall of potentially contaminated alcohol prep products by a second manufacturer is raising sharp questions about an entire category of medical supplies: non-sterile pads and swabs, infection experts say.

“Is there any place for a non-sterile alcohol prep pad in a hospital setting?” said Dr. Christine Nyquist, a Colorado infection control director who blew the whistle on dangerous bacteria in alcohol wipes last fall. “We believe there isn’t.”

Nyquist and other microbiology experts say a debate is brewing about whether the non-sterile pads and wipes routinely used in hospitals, clinics and private homes to clean skin before shots and other procedures should be curtailed — or perhaps banned —
because of the potential for infection. While sterile pads have had problems, too, non-sterile products may raise alarms from the start.

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By Melanie Padgett Powers | 09.15.2011

Originally Published September 2011

Children and adults with bleeding disorders can and should receive the recommended immunizations for their age group. These vaccinations are often given by their primary care physician.

Most vaccinations can be given subcutaneously, or under the skin, instead of intramuscularly, or into the muscle, to reduce the risk of a muscle bleed. However, some healthcare providers may prefer to give intramuscular injections using a smaller needle in a larger muscle, like the thigh, according to “Caring for Your Child,” published by the National Hemophilia Foundation.

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September 5, 2011

A University of Central Florida and University of Florida collaboration has landed $5.6 million in National Institutes of Health grants for hemophilia research.

The first grant, $3.6 million over five years, is aimed at determining whether a green technique pioneered at UCF will help make treatment of hemophilia A more effective. The second grant, $2 million for four years, covers similar research for hemophilia B. The grants were awarded to UCF and UF, which applied for funding jointly thanks to a long-time collaboration between scientists at both institutions. Duke University also is a partner for the research on hemophilia A.

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