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Hope for the Whole Spectrum of Life
CHORI Scientist Determines Haematopoietic Cell Transplantation for Sickle Cell Disease Carries No Risk of Impaired Growth

In the January 11th issue of the British Journal of Haematology, Mark Walters, MD, CHORI scientist and the director of the Blood and Marrow Transplant Center at Children's Hospital and Research Center Oakland (CHRCO), reports for the first time on the effects of haematopoietic cell transplantation (HCT) on growth and development in patients with sickle cell disease (SCD).

Questions surrounded the impact of HCT on two counts. The first was that researchers like Dr. Walters had hoped that providing a cure through HCT could alleviate the slower growth rates observed in children with SCD.

"A lot of energy has to go into making the blood in patients with SCD, which diverts energy from their growth," explains Dr. Walters. "We were mostly interested in investigating whether the transplant itself would make a difference."

The second was whether or not HCT could result in decreased growth due to the high doses of chemotherapy required - which can damage the ovaries and testes. That, in turn, could cause a reduction in the development of hormones essential for stimulating growth throughout puberty.

While the results of the study did not indicate a significant increase in growth rates across the board, they did resolve concerns surrounding the negative potential for decreased growth. When the transplant was done well in advance of the adolescent growth spurt, growth was entirely normal, yet when the transplant was done right at, or just before the spurt, there was some decrease in growth, particularly in boys.

"The study has two messages," says Dr. Walters. "The first is that transplantation doesn't adversely effect growth in sickle cell disease. The second is that to avoid any potential for a negative effect, do it early in life. Don't wait until kids are older and more likely to have problems."

Part of a 10 year, multi-center study designed to assess the risks and benefits of HCT in sickle cell patients, Dr. Walter's latest publication adds to the growing evidence of the benefits of HCT, the only curative treatment currently available for SCD, which affects hundreds of thousands, if not millions, of individuals world-wide.

First discovered as a potential treatment in 1984, HCT has a success rate of 85 to 90 percent, offering hope to those children suffering from the painful complications of SCD and to their families. No procedure comes without risks, however, and HCT is no different.

"We do think that there is about a 5 percent chance the person might die as a result of the procedure," acknowledges Dr. Walters. "No one can guarantee that it will be successful and that's a real hurdle that families and doctors struggle with."

In making such decisions, though, Dr. Walters emphasizes the importance of looking at the whole spectrum, including not only quality of life, but life span - the median life span for sickle cell patients in this country is still only 45 to 50 years, compared to the median lifespan of 70 - 80 years in African Americans unaffected by sickle cell anemia.

"Our feeling has been that this is really a terrible disease to have and most individuals who inherit it don't really live healthy lives or long lives," says Dr. Walters. "The way to balance the risk is to look at the other end, the whole life spectrum."

With his continued research, Dr. Walters hopes to be able to bring more families the opportunity to choose HCT as an alternative, not only through continuing to assess the risks and benefits, but by ultimately reducing the overall risk as well.

"There may be ways to make transplantation even safer, and we're working on that," says Dr. Walters. "The place we'd like to be is offering a family with a two- or three-year old a curative approach with results that are good enough to be convincing that it really is the right thing to do. That's really where we'd like to be one day."

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Monday, May 16, 2011 11:33 PM

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