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Toward the Future: Perfecting the Standard of Care
CHORI Publishes Results of First-Ever Comparative Study of Transfusional Iron Overload in Patients with Thalassemia and Sickle Cell Disease

The November issue of the American Journal of Hematology reports on the first in a series of studies conducted through the ground-breaking Multi-Center Study of Iron Overload. The brain child of Elliott Vichinsky, MD, a CHORI principle investigator and the chief of the division of Hematology/Oncology at CHRCO, the Multi-Center Study is a collaborative research effort crossing international boundaries.

Utilizing subjects recruited from nearly 30 different clinical hematology centers from the United States, Canada and the United Kingdom, the study is the first to conduct a comparative analysis of transfusional iron overload in patients with thalassemia and sickle cell disease.

“We know that patients with thalassemia exhibit multiple co-morbidities related to iron overload,” says Ellen Fung, PhD, the project coordinator for the Multi-Center Study.

“They are typically transfused from birth or soon thereafter in order to sustain life, and researchers have been monitoring the effects of transfusion therapy in patients with thalassemia for decades.”

While blood transfusions have been the standard of care in thalassemia, they have not been utilized with the same frequency in patients with sickle cell disease until recently.

As Dr. Fung explains, “More and more patients with sickle cell disease are being transfused, especially if they are at risk for stroke. What we’re finding is that once they start transfusion therapy, they will likely have to be transfused for life. As a result, we’re creating a similar cohort of iron-overloaded patients, but without having studied its long term effects in sickle cell patients.”

This landmark, 5-year study represents the first step in establishing a cohort of subjects to assess the impact of transfusion therapy in the sickle cell patient population. While the data analysis has only just begun and a new NIH grant is currently under review to follow these subjects for an even longer period of time, this publication does report some significant preliminary observations.

While this still needs to be confirmed over time, this morbidity didn’t appear to be related to iron overload. One plausible explanation is in patient selection; unlike younger patients with sickle cell disease, many adult patients are transfused because they already experience significant chronic ailments.

“Other previously unpublished findings that we thought were important to communicate were the relationships between employment, education and morbidity,” says Dr. Fung.

In both iron-overloaded thalassemia and sickle cell patients, the length of hospital stay was correlated with a lack of employment; patients with sickle cell disease had the highest rates of both hospitalization and unemployment.

“It makes perfect sense that if you’re in the hospital for 60 days out of the year, you’re not going to be able to keep your job, but this is the first time it’s been reported,” Dr. Fung explains.

The relationship between higher education and morbidity in transfused sickle cell patients was just as striking – with a 2.1-fold greater risk of not achieving a college education in patients who had a history of stroke. This isn’t due to a lack of intelligence in this patient population, however, but the impairment often associated with stroke.

“When you take stroke out of the equation, the educational difference disappears,” Dr. Fung says.

Such observations no doubt represent the tip of the iceberg in the first of these publications to analyze the comparative data. The Multi-Center study itself is but a beginning, but it is a much needed first step.

“We’re hoping to explore the data in even more depth, and to follow these unique patients for a much longer period of time in order to discover possible disease specific differences in how iron is handled by the body,” says Dr. Fung.

PubMed Abstract

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Monday, May 16, 2011 11:33 PM

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