Foundation Hosts Telephone Conference on Deferiprone Oral Iron Chelator

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Deferiprone is an alternative drug for treating thalassemia, a genetic blood disease. The treatment can be obtained under the FDA's Treatment Use Program and may provide additional cardioprotective benefits.

The Cooley’s Anemia Foundation (CAF) hosted a special telephone conference on June 21 to discuss deferiprone, an oral iron chelator that offers a possible alternative to people suffering from the fatal genetic blood disease, thalassemia. The call focused on information about the drug, including its cardioprotective aspects and its current availability to some patients under the Food and Drug Administration’s Treatment Use Program.

Individuals born with thalassemia major (a.k.a. Cooley’s anemia) require lifelong blood transfusions every two to four weeks. These transfusions result in a deadly build-up of excess iron in the body; left untreated, this excess iron results in heart and liver failure. At this point in time, the only FDA-approved treatment for this iron overload is deferoxamine, a drug that must be delivered nightly via infusion over an 8 to 12 hour period.

Because this method of administration is so burdensome, few patients can maintain the optimal treatment, resulting in an unacceptable level of mortality among the patient population. An effective oral chelator, which would be easier to administer, is desperately needed to stop premature deaths among these patients. Deferiprone, which is in use in 48 countries but is not yet approved for general use by the FDA, is one possible alternative, especially as there are indications that it may have greater cardioprotective benefits than deferoxamine.

The telephone conference was moderated by Dr. Elliott Vichinsky, Director of the Thalassemia Program at Children’s Hospital Oakland. Speakers included Dr. Alan Cohen, head of the Thalassemia Program at Children’s Hospital of Philadelphia; Dr. Antonio Piga, of the Department of Pediatric Hematology at the University of Turin, Italy; Dr. Dudley Pennell, cardiologist with Royal Brompton Hospital and Imperial College, London; and Maria Hadjidemetriou, a thalassemia patient with experience using deferiprone.

Among the information presented during the call:

  • Prospective studies have demonstrated that deferiprone is a viable treatment option for patients with iron overload, especially if they cannot obtain acceptable results using their current therapy.
  • Some patients may develop side effects ranging in severity from rare cases of agranulocytosis to joint problems and gastrointestinal disturbances; however, careful monitoring of the patients and modification of their treatment can minimize the risk of these side effects.
  • Several studies indicate that patients using deferiprone, either alone or in combination with deferoxamine, may experience a greater and more rapid reduction of cardiac iron.
  • Some thalassemia patients who appear to be well-chelated with deferoxamine may still develop cardiac problems. For that reason, it is recommended that patients avail themselves of T2* technology, a new method of measuring cardiac iron stores, to better ascertain potential risk of heart disease.
  • Although deferiprone is not currently available to the general population in the United States, doctors can utilize the FDA’s Treatment Use Program to obtain the drug for those patients whom it would benefit.

Peter Chieco, CAF Vice President for Medical Information, stated that “there is a crucial need for an alternative treatment for people with thalassemia. We believe that deferiprone is one such alternative. While we are grateful that this drug is available to some patients through the Treatment Use Program, we reiterate the need for ready access of deferiprone for the general thalassemia population.”

Founded in 1954, the Cooley’s Anemia Foundation is the nation’s leading nonprofit organization dedicated to fighting thalassemia. The Foundation provides valuable patient services, funds important medical research, educates medical professionals about the latest advances in thalassemia and spreads awareness of the disease among the general public.

For more information, contact the Cooley’s Anemia Foundation at 800-522-7222.

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Craig Butler

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