FOR IMMEDIATE RELEASE
New Drug Poised to Radically Change the Treatment of Severe Anemias
(WASHINGTON, April 21, 2006) – Those with severe chronic
anemias need frequent blood transfusions to remain healthy, but such frequent
transfusions can cause a potentially deadly buildup of iron in the body, leading
to heart and liver failure. The traditional treatment to remove excess iron
is so onerous that many patients choose to forgo it, putting their own lives
at risk. The results of an international study on deferasirox, a new drug that
may revolutionize the way chronic iron overload is treated, will be published
in the May 1, 2006, issue of Blood,
the official journal of the American Society of Hematology.
The current standard therapy to rid the body of excess iron is deferoxamine,
administered for as long as the patient continues to receive blood transfusions,
which, for many patients, can be for the rest of their lives. Although its effectiveness
and safety are well-established, the necessity for the drug to be delivered
by slow subcutaneous or intravenous infusion for eight to 12 hours a night over
a period of five to seven days makes it an inconvenient and painful choice for
patients. Unlike deferoxamine, deferasirox is available in a once-daily, drinkable
format, providing a promising alternative.
“The ease and convenience of deferasirox means that more patients needing
frequent blood transfusions, especially young children, will be able to be successfully
treated and lead normal, healthy lives,” said Maria Domenica Cappellini,
MD, of the University of Milan, Italy, and lead study author.
To compare the efficacy and safety of the two drugs, a multicenter trial of
both children (some as young as two years old) and adults diagnosed with chronic
iron overload was conducted in a dozen countries worldwide. People with beta-thalassemia,
an inherited blood disorder, were selected for this study because complications
of chronic iron overload have been best studied in those with this disease.
All participants continued receiving regular blood transfusions to treat beta-thalassemia
throughout the year-long study.
Participants were randomized into two groups: 290 received deferoxamine infusions
five days a week; 296 drank deferasirox dissolved in water each day before breakfast.
Drug dosages were determined by each patient’s liver iron concentration
(LIC) level; those with higher levels received increased doses. Since LIC values
above 7 mg Fe/g dw are associated with increased morbidity and mortality, the
primary goal of the trial was to reduce LIC levels in those with high values
and maintain LIC levels in those with low values. At the beginning of the study,
more than two-thirds of the participants had at-risk LIC levels.
Deferasirox proved to be equally as effective as deferoxamine in patients receiving
the highest doses of the drug. A majority of these patients (nearly 60 percent),
demonstrated sustained or reduced LIC levels during the study. For those receiving
the lowest drug doses, the amount of deferasirox was found to be insufficient
in these regularly transfused thalassemia patients.
Deferasirox was generally well-tolerated. The most common side effects were
skin rash (affecting approximately 11 percent of patients) and gastrointestional
problems, which occurred in 15 percent of the group and included abdominal pain,
vomiting, diarrhea, and constipation. Most patients (92 percent) were able to
complete the study, though a small number in each treatment arm discontinued
because of safety reasons. Four deaths, determined to be unrelated to the study
drugs, also occurred during the trial.
This work was supported in part by research funding from Novartis Pharma.
To receive a copy of the study or to arrange an interview with Maria
Domenica Cappellini, MD, lead author of the study, please contact Laura Stark
by e-mail or by phone at 202-776-0544.
The American Society of Hematology is the world's largest professional society
concerned with the causes and treatment of blood disorders. Its mission is to
further the understanding, diagnosis, treatment, and prevention of disorders
affecting blood, bone marrow, and the immunologic, hemostatic, and vascular
systems, by promoting research, clinical care, education, training, and advocacy
in hematology.
Blood, the official journal of the American Society of Hematology,
is the most cited peer-reviewed publication in the field. Blood is
issued to Society members and other subscribers twice per month, available in
print and online at www.bloodjournal.org.
Back to Press Release Listing
|
