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Helping Thalassemia Patients Find Optimum Health
Transfusion in Thalassemia Patients Correlates with Increased Body Composition
A new study just published in the Journal of Pediatrics evaluates for the first time the relationship between transfusion and overall body composition in patients with thalassemia.The study was conducted through the National Institutes of Health (NIH) funded Thalassemia Clinical Research Network (TCRN), established in 1998 as a means to provide a national structure to conduct clinical studies in thalassemia. Children's Hospital Research Center Oakland has been one of only 5 original participating centers.

"Clinical practitioners have always had the sense that thalassemia patients are underweight, so we wanted to actually look at it from an evidence-based perspective," says CHORI scientist and first author, Ellen Fung, PhD.
"Clinical practitioners have always had the sense that thalassemia patients are underweight, so we wanted to actually look at it from an evidence-based perspective."

Thalassemia is a genetically-based blood disease, in which the ability of hemoglobin to transport oxygen throughout the body is impaired. Many, but not all, thalassemia patients need chronic blood transfusions as part of their regular treatment. These blood transfusions provide the critically needed red blood cells to transport oxygen to tissues, but also result in a build-up of excess body iron, which requires regular chelation therapy to remove it.

"We have limited information about nutritional status and body composition in patients with thalassemia because for so long the focus has been on iron overload and its toxic effects," says Dr. Fung. "If a patient isn't going to survive long past 30, questions about nutritional status and it's effect on overall health don't really mean much."

Now, however, with significant advances in transfusion and chelation therapy beginning in the mid 1980s, the life expectancy of thalassemia patients has increased exponentially.

"Now we have two types of chelators, so you can tailor therapies to better suit individual patients. As a result, we have transfused patients here at Children's that are living into their forties and fifties, and non-transfused patients living into their sixties," Dr. Fung says.
“Now we have the luxury of being able to look at other, less crisis-oriented questions like nutritional status, bone health and body composition.”
Body composition – the amount of fat and muscle in any given individual – is an indicator of many other things, including overall health and, in children, the ability to grow and develop to their full genetic potential.

As Dr. Fung explains, "You only have one shot at reaching your genetic potential for growth as you go through puberty. You can't make it up when you're thirty."

While patients with thalassemia face a myriad of challenges to their overall health that require a complex balance between their anemia, iron overload and chelation therapy, Dr. Fung wanted to know whether or not other issues – such as nutritional status and altered body composition – could also be playing a part.
"The main results of this study confirmed what we've seen clinically all along – that patients with thalassemia have less body fat than the average American – but what was surprising was that we found significant differences between patients that are chronically transfused and patients not receiving transfusion therapy," says Dr. Fung.

"The non-chronically transfused patients have lower total body fat, and that lower body fat was related to lower bone mass and poor growth rate. It suggests that well transfused patients may have better overall clinical outcome."

The result is surprising in particular because thalassemia patients who require regular transfusions are thought to have more severe thalassemia, and thus might be expected to have lower body fat and muscle mass. Instead, it was the non-transfused patients who were generally found to have less fat and lean mass and thus at risk for worse growth and overall health.
"We don't know whether this is a direct result of regular transfusions, or the fact that patients who are being transfused tend to be followed more closely than non-transfused patients, " says Dr. Fung.

Whatever the cause, the end result is the same: the study provides confirmation of what clinicians have long suspected, that a certain percentage of non-transfused patients with thalassemia might grow better, do better in school, and have better overall health if they started transfusion therapy.

"The decision to start transfusion is tough for the family and child to make, however, when the burden of routine transfusion and iron overload related complications are considered," says Dr. Fung.
“Now we actually have some data that confirms clinician's suspicions are right, and that there are tangible benefits to transfusion therapy in these patients.”
"Well, now we actually have some data that confirms clinician's suspicions are right, and that there are tangible benefits to transfusion therapy in these patients."

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Tuesday, May 17, 2011 8:19 AM

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