A novel bone marrow transplantation procedure that removes only some of the patient’s own bone marrow has shown promise in patients with chronic granulomatous disease, an inherited immune disorder.
Investigators at the National Institute of Allergy and Infectious Diseases (NIAID) reported their findings on March 22 in The New England Journal of Medicine (2001;344:881-888). Five children and five adults with chronic granulomatous disease were treated with peripheral blood stem cell transplantation from HLA-identical siblings. The low-intensity stem cell transplantation procedure uses no radiation and is less risky than conventional stem cell transplantation because not all of the patient’s bone marrow cells are destroyed before transplantation, the NIAID said in a statement.
Although claims of cure would be premature, the data "suggest that for patients in whom the transplant was successful, the immune system is functioning significantly better than before treatment," the NIAID said.
According to NIAID director Anthony S. Fauci, MD, "Nonmyeloablative stem cell transplantation has previously shown promise in leukemia and kidney cancer patients, but this is the first clinical trial of this strategy in a series of patients who have an inherited immune disorder."
Patients in the study ranged in age from 5 to 36. Overall, the procedure worked best in the five children who were younger than 13; the only case of graft-versus-host disease in the children was mild. Three of the five adults in the study died.
In an editorial in The New England Journal of Medicine, R. Alan B. Ezekowitz, MB, of MassGeneral Hospital for Children in Boston described the new approach as "a feasible option for patients with an HLA-matched related donor." However, because of the mortality and complication rates, he urged that the approach be reserved for older patients, in whom the mortality rate associated with chronic granulomatous disease is highest.
For Your Information:
- Read the abstract at www.nejm.org/content/2001/0344/0012/0881.asp
- and the full text of the editorial at www.nejm.org/content/2001/0344/0012/0926.asp.
- The NIAID posts its statement at www.niaid.nih.gov/newsroom/CGD-stemcell.htm.
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