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Blood Transfusions Reduce Risk for Children with Sickle Cell Anemia
- Updated: August 20, 2014
According to a new study published in the New England Journal of Medicine, there could be a link between a reduction in stroke incidence for children with Sickle Cell Anemia and monthly blood transfusions. To date, information gathered from several clinical trials seem to support that belief.
As quoted by Dr. James Casella, Vice Chair of the trial but also Director of Pediatric Hematology at Johns Hopkins Children’s Center in Baltimore, “Now we have a treatment to offer”. Prior to this discovery, children with this condition had no treatment and therefore, screening was seldom offered to identify silent strokes. As a caveat, Casella also states, “This study is a first step, not the last one”.
Even though early findings are extremely promising, one aspect that remains unknown is how long children with Sickle Cell Anemia would need to continue getting transfusions. In other words, are blood transfusions needed once a month for a year, five years, ten years, or perhaps life? Obviously, this is a huge question and one that must be answered before a treatment such as this could be made available for everyone.
Unlike many other serious diseases, Sickle Cell Anemia has a very select target to include people primarily of African, Mediterranean, and South or Central American descent. According to the United States National Institutes of Health, it is also a very serious disease that affects one in every 500 African American children born in the US.
With this disease, crescent-shaped red blood cells are produced opposed to the normal disc-shape. The problem, these abnormal cells are sticky, thereby compromising blood flow. Because of this, secondary problems are common to include silent strokes and strokes, which cause damage to brain tissue. Unfortunately, there are no obvious symptoms of an impending disaster.
In this new study involving Dr. Casella, he and his colleagues scanned more than 1,000 patients between the age of 5 and 15 who were diagnosed with Sickle Cell Anemia. Using MRI technology, the goal was to identify any signs of the children having suffered a silent stroke in the past. At the conclusion of the study, 196 shown to have a past stroke were assigned randomly between groups. One group was given monthly blood transfusions while the other group received only conventional care.
After being monitored over a three-year period, of the children in the group receiving monthly blood transfusions, 6% had experienced a new silent stroke or actual stroke compared to 14% for those in the second group who did not receive transfusions. Although a correlation between reduced risk of silent stroke and blood transfusions existed, a direct cause and effect link did not.
Preventing Silent Strokes – What are the Key Benefits?
As mentioned, silent strokes lead to damaged tissue in the brain. To ensure no impairment of IQ and executive function, which includes planning, focusing, and organizing, prevention is imperative. However, while it appears blood transfusions prevented silent strokes and thereby damage to brain tissue, there was no evidence that these children had higher IQ levels or better mental function.
In looking at the results of this study, Dr. Martin Steinberg, Director of the Center of Excellence in Sickle Cell Disease at Boston University School of Medicine agreed that trial results were solid. However, two concerns were raised. The first was the way this new treatment would transition from a clinical study to teaching hospitals and then to the real world of medicine and the second was potential limitations to blood transfusions due to some medical centers not having adequate resources.
Summing it Up
The bottom line – in order for monthly blood transfusions to be turned into a viable treatment used to reduce stroke risk in children with this disease, a tremendous amount of monitoring would be required. After all, there could be specific risks involved to include iron overload, something quite common when levels of iron in the blood become too high and therefore dangerous.
Dr. Casella agrees that further research and studies are needed to fully understand the benefits but also execution of this treatment. Based on his findings, Casella feels it is reasonable for children with Sickle Cell Anemia to undergo MRI testing prior to starting school. If at that time any signs of a silent stroke were found, monthly transfusions might be considered as a treatment option.