Bone marrow breakthrough eases treatment
Updated on 02 September 2009
Children needing bone marrow transplants could be spared chemotherapy side effects after doctors develop a new technique to prevent rejection.
A new life saving technique has been developed to treat terminally ill children in need of bone marrow transplants.
Patients with Primary Immunodeficiencies (PID) can now be given antibodies instead of standard chemotherapy to prepare them for transplants.
The technique pioneered by doctors at Ormond Street Hospital and the UCL Institute of Child Health is more effective and has fewer side effects than traditional chemotherapy which can cause organ damage and infertility.
Dr Persis Amrolia, a consultant in bone marrow transplants at GOSH who led the research published today in The Lancet said: "Because this technique gives us an alternative to intensive chemotherapy, the treatment we can offer is safer, and provides a greater chance of allowing these children to grow up to lead normal healthy lives.
"We didn’t see any of the hair loss and sickness normally associated with intensive chemotherapy for BMT, there was much less damage to the liver, lungs and gut and we anticipate none of the harmful long-term side effects.
"This represents a major breakthrough in how we treat patients who have PID."
The technique has been used to save the lives of 13 out of 16 children too sick to undergo a traditional bone marrow transplant, over the last ten years.
As well as being cured of their underlying diseases, they also recovered twice as quickly as those given the standard treatment. Fifty children with PID receive a transplant each year in the UK.
Dr Amrolia from GOSH added: "Because this approach was experimental, we only used it on the sickest children, who we felt could not tolerate standard transplant chemotherapy.
"Given how sick these children were before transplant, the results are remarkable. What’s really encouraging is that pretty much all the children who survived now have a really good quality of life."
The significant improvement in outcome for PID patients has led to many European and US centre adopting this as standard care, but ultimately similar approaches could be used to treat children with other genetic diseases and even leukaemia.
