New Technique Offers Hope for Kids With Immune Deficiency

WEDNESDAY, Sept. 2 (HealthDay News) -- For children born with immunodeficiencies, researchers may have found a better way for them to get the help they need from stem-cell transplants.

A regimen using antibodies instead of high-dose chemotherapy may help even in the sickest children, according to Dr. Persis J. Amrolia, a doctor in the department of bone marrow transplantation at Great Ormond Street Children's Hospital in London, and colleagues. Their findings appear online in advance of publication in an upcoming edition of The Lancet.

Children with primary immunodeficiencies have genetic defects in their immune system that leave them open to infection and other complications. Stem-cell transplants can replace the defective immune system with one derived from healthy donor bone marrow, but without a stem-cell transplant, many of these children might die, the researchers noted in a journal news release.

In order to create space for the donor stem cells and prevent rejection, the patient usually undergoes chemotherapy, radiotherapy or both. This chemoradiotherapy can cause severe liver or lung damage, as well as hair loss and sickness. It may also cause problems with growth, puberty and infertility in later life, according to the news release.

In this study, instead of using high-dose chemotherapy, the researchers used immune molecules made by white blood cells (called monoclonal antibodies). The method targeted molecules called CD45 and CD52, which are specific to bone marrow and blood cells. Therefore, the monoclonal antibodies targeted only the immune cells that cause rejection and did not affect other body tissues, the authors explained.

With this approach, the 16 children with primary immunodeficiencies in this study, who were too sick for a traditional stem-cell transplant, were able to avoid much of the toxicity caused by chemotherapy.

The approach was well-tolerated despite the fact that most patients were extremely sick at the time of transplant. The researchers reported no signs of sickness or hair loss normally associated with high-dose chemotherapy and less damage to the liver and lungs.

Patients recovered twice as fast as those given standard treatment, and almost all are doing well and are expected to have few problems associated with the treatment later in life, the study authors added.

More information

Learn more about childhood immune deficiencies from the Lucile Packard Children's Hospital at Stanford University.