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First Time Identification of Huge Disparity in Iron Overload Monitoring between Patients with Thalassemia and Patients with Sickle Cell Disease

"While the prevalence of monitoring was not a primary outcome of this particular study, we discovered huge differ-ences in the management of iron overload in SCD and thal-assemia patients that warranted a closer look."

With a dramatic new study published in Transfusion’s May issue, CHORI investigators Ellen Fung, PhD and Elliott Vichinsky, MD, chief of the division of Hematology/Oncology at CHRCO, along with CHRCO clinical scientist Paul Harmatz, MD, hope to establish and promote the first ever standard of care guidelines for monitoring iron overload in sickle cell disease (SCD) patients.

The Multi-Center Study of Iron Overload is a 4 year, longitudinal natural history study comparing iron overload in patients with thalassemia and SCD in subjects recruited from nearly 30 different clinical hematology centers from the United States, Canada and the United Kingdom. While Dr. Fung suspects that clinicians who routinely deal with these two patient populations won't be entirely surprised by the data from the new longitudinal study, the Transfusion publication provides hard numbers for the first time to confirm a disparity that researchers and physicians have been experiencing on an anecdotal basis in the clinic for some time.

"Thalassemia patients are followed religiously to track iron-overload related issues," says Dr. Fung of the study results. "They get endocrinology work-ups every year, their hormones are checked, they get liver biopsies on a regular basis, as well as testing for cardiac failure and a whole slew of blood work. It's very regimented."

Dr. Fung wouldn't have expected to find anything less: thalassemia has had a very active patient group and significant levels of advocacy and patient support for the past 3 decades or so. The numbers on how well SCD patients were monitored, however, were alarming.

"Even though there are roughly a thousand patients in North America with thalassemia, as compared to over 70 thousand with sickle cell disease, monitoring for iron-overload and related dysfunction in sickle cell patients was entirely haphazard, even in centers that treat both types of patients," Dr. Fung says.

There are a number of potential explanations for this disparity, such as a lack of patient follow-through, lack of insurance coverage for testing, doctors who are not as well versed in the possibility of iron-overload in sickle cell populations, and a dearth of strong guidelines about how iron-overload should be addressed in this population. All of these potential causes, however, stem from the fact that the main focus of SCD treatment has been pain management, because that has been the most prominent feature of the condition.
"Blood transfusions are essential in the treatment of thalassemia from birth, so thalassemia patients are indoctrinated into the risks and realities of iron-overload from the start," explains Dr. Fung.
"Their physicians are primed to expect, and proactively treat, iron-overload in these patients, but there isn't a similar script for SCD patients. Transfusion is a relatively new therapy for use in SCD, so the focus is most often on immediate adverse events associated with transfusion or the reason transfusion begins - which is stroke risk - rather than subsequent development of iron-overload."

Dr. Fung and her colleagues believe that has to change, however, and are on a mission to establish iron overload monitoring protocols on a national basis in SCD patients.

"Until we know whether SCD patients respond differently to iron-overload, the bottom line is that the same set of recommendations and guidelines that exist for thalassemia should be followed in SCD patients," Dr. Fung says.

As a result, Drs. Fung, Vichinsky and Harmatz will be submitting the publication to the head of the National Heart Lung and Blood Institute, as well as those who head up all the major sickle cell disease centers in North America.

"We're going to push our own publicity on this as much as we can," says Dr. Fung. "We want everyone to have this data so that we can start monitoring these patients and begin to establish a much needed standard of care for iron overload in SCD patients."

With such guidelines in hand, Drs. Fung and her colleagues are committed to ultimately balancing the disparity in treatment between these two patient populations to make sure that the same rigor in proactive monitoring and care that thalassemia patients currently receive is provided to SCD patients as well.

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Tuesday, May 17, 2011 8:19 AM

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