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NEW YORK, Mar 22 (Reuters Health) - A new transplant procedure is still risky, but appears to offer hope for patients with the most severe forms of chronic granulomatous disease (CGD), a rare, inherited immune disorder that results in repeat, often life-threatening infections.
Transplanting immature immune cells from unaffected relatives (a procedure known as stem cell transplantation) can help CGD patients, but earlier techniques required destruction of the recipient's own bone marrow in order for the new cells to take root.
As a result, high rates of complications and deaths made it a last resort treatment for the sickest patients, according to Dr. Mitchell Horwitz and colleagues from the National Institutes of Health in Bethesda, Maryland.
Newer approaches to stem cell transplantation do not require total destruction of the patient's bone marrow, so the investigators tried this safer bone marrow-sparing technique to treat 10 patients with severe CGD.
Eight of the 10 patients saw their abnormal immune cells replaced by the normal immune cells of the donor, according to the report in the March 22nd issue of The New England Journal of Medicine. In the 18 months after the transplant, seven patients are alive and only one has experienced a CGD-related infection.
However, the procedure also carries risks, the report indicates. Three of the patients subsequently died--all adults--one from pneumonia, one from complications of a second transplant after the first was rejected, and one from graft-versus-host disease--a transplant complication in which donor cells attack the cells of the recipient.
Complications were common, the researchers note, with four patients developing graft-versus-host disease and four patients experiencing transplant-related infections.
"We believe that the best candidates for this treatment are younger patients with the severe forms of CGD," Horwitz told Reuters Health. "Our data would suggest that these patients have fewer complications and lower risk of death from the treatment."
This "approach is particularly desirable for treatment of children because it does not involve the use of radiation," Horwitz explained in an interview. "It is expected that patients conditioned in this way will still be able to have children...and will have normal growth and development." The immune system-destroying step used in earlier techniques would often damage a patient's future fertility.
Dr. R. Alan B. Ezekowitz from MassGeneral Hospital for Children in Boston, Massachusetts, expresses a somewhat different view in his editorial.
"Until we have better (transplant methods)," he writes, "I would reserve this approach for older patients, in whom the (death) rate associated with chronic granulomatous disease is highest."
Regardless of who gets the transplant, Horwitz cautioned, "the procedure is not without significant risks, and the patient or the patient's family must be carefully informed of these risks."
About 1,000 people in the US have CGD, and the disease affects 25,000 people worldwide.
SOURCE: The New England Journal of Medicine 2001;344:881-888, 926-927.
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