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Revolutionizing the Treatment of Iron Overload
CHORI Spearheads Development and Use of Novel Iron Chelators

"In transfused patients, iron overload is a very serious problem that can be fatal. Thalassemia patients are transfused by birth, and by the age of 24, 90 percent of all sickle cell patients have received multiple transfusions."

CHORI’s Center for Sickle Cell and Thalassemia has been pioneering novel treatments and defining excellence in the care of hematological disorders for decades, earning national and international recognition for its cutting-edge research and treatment. A perfect example of such excellence is the spearheading of the development and use of novel iron chelators by CHORI’s Elliott Vichinsky, MD, chief of the division of Hematology/Oncology at CHRCO. The fruits of Dr. Vichinsky’s latest labors were reported in the April issue of Acta Haematologica as well as in a June commentary by Dr. Vichinsky in Pediatrics.

Although iron chelation has long be available to treat the iron overload that results from regular transfusions, the traditional chelator drug of choice, desferoxamine (DFO), has significant drawbacks, from how it's administered to the length of administration.

As Dr. Vichinsky explains: "You take a needle, put it under your skin, push it in and tape it there. Then you hook it to pump and let it run 8 to 12 hours a night, every night, night after night. It hurts, it causes reactions, people don't like it, and don't want to do it."

In addition to the patient discomfort and hassle, there is the cost of the pump and software required to administer the drug, all of which result in the potential for a dangerous lack of patient compliance. As a result, Dr. Vichinsky and his colleagues have been searching out alternative chelation options that increase patient compliance.

One, a drug developed by CHRCO's Paul Harmatz, MD and Dr. Vichinksy, is a starch-conjugated version of DFO that can be administered once a week instead of every night (British Journal of Haematology, 138, 374-381).

"With one injection, this new agent may last up to one week. In addition, it can be used with other chelators as well, and may actually be even more effective when augmented by combined therapies," says Dr. Vichinsky.

Another alternative, which Dr. Vichinsky advocates in his Pediatric commentary and has pioneered in his own research, is deferasirox, or Exjade, which, like DFO must be taken every day. In sharp contrast to DFO, however, Exjade can be popped in the mouth like any other daily vitamin.
"With the oral administration Exjade provides, patients re-ported a signif-icant increase in quality of life. They miss less time from work, they spend less time hook-ing themselves up."
The results reported in the April issue of Acta Haematologica bear out that increased satisfaction. Eighty-four percent of patients using Exjade reported that they would be willing to continue treatment - compared with only 11 percent of patients on DFO. Only 1 percent of patients taking Exjade said they would never want to take it again, while 53 percent of patients on DFO said they wouldn't want to take it again.

"These are critical results because they are surrogate markers of compliance, and the most important predictor of survival is compliance," says Dr. Vichinsky.

"With no treatment for iron overload, the median lifespan for patients receiving chronic transfusions is only 20 to 25 years. With chelation? You could live into your sixties."
Now, living into their sixties may be a much more accessible option for many more patients who face life-long transfusions and the potentially fatal effects of iron overload that result.

With the burgeoning options being discovered and pursued by CHORI researchers like Dr. Vichinsky, from having an injection once a week to taking a simple pill, patient compliance can only increase, reducing fatal complications and increasing quality of life and longevity at the same time - just part and parcel of what CHORI's Center for Sickle Cell and Thalassemia strives for every day, improving people's lives, one patient at a time.


Tuesday, May 17, 2011 8:19 AM

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