Bone marrow transplant findings to cut chemotherapy for youngsters

Published on Tuesday 1 September 2009 22:07

A NEW technique to prevent rejection in children needing bone marrow transplants means youngsters can avoid some of the worst side-effects of chemotherapy, researchers said yesterday.

The new method, revealed in medical journal the Lancet, uses antibodies which recognise and kill the patient's own bone marrow to create space for the donor cells. It reduces the need for intensive chemotherapy and also lessens the risk of rejection, according to the study.

The technique has been successfully used in children with genetic defects of their immune systems – known as primary immunodeficiencies or PID – who were too sick to undergo a traditional bone marrow transplant.

Doctors believe the technique, developed by Great Ormond Street Hospital and University College London Institute of Child Health, probably saved the children's lives.

Without a bone marrow transplant to replace the defective immune system with one from a healthy donor, children with PID often die from infection and other complications.

Previously, children needed high doses of chemotherapy to wipe out their own bone marrow and create space for the donor stem cells prior to a transplant.

This chemotherapy can result in severe damage to the liver, gut or lungs as well as hair loss and sickness.

The new technique uses an antibody directed against a molecule called CD45, to wipe out patients' own diseased bone marrow tissue and create a space for the bone marrow from the donor to grow.

The study showed 13 of 16 patients with PID who were treated between 2003 and 2007 survived and were cured of their underlying disease. They recovered twice as quickly as those given standard treatment and did not show damage to other organs linked with high doses of chemotherapy.

Dr Persis Amrolia, a consultant in bone marrow transplant who led the research, described the results as "remarkable".

"Because this approach was experimental, we only used it on the sickest children, who we felt could not tolerate standard transplant chemotherapy."

Katie Docherty, from the Anthony Nolan Trust bone marrow register in Scotland, said: "This new conditioning regime appears to offer great hope to those children who are the sickest and often the hardest to provide successful transplants for."