The CGD Café

This is an interesting article for those considering a BMT. It might be good to get input from your doctor. Mary Hurley

www.thenorthernecho.co.uk The Northern Echo March 9, 2009 ‘Copy us’ urge transplant team’

PIONEERING North-East medics have shown that their approach to treating a rare and life-threatening condition is so successful it should be adopted worldwide.

A team at Newcastle General Hospital has demonstrated that the best chance of curing chronic granulomatous disorder (CGD) is by carrying out bone marrow transplants.

In a paper published online by the British Journal of Haematology, the Newcastle team say their record of curing 18 out of 20 children or young adults with CGD suggests this should be the standard approach to this group of patients.

Most hospital units around the world treating children with CGD tend not to carry out transplants unless the patient has a brother or sister who could provide bone marrow.

But the Newcastle study showed that it was possible to be just as successful using unrelated donors for transplant.

Dr Andy Gennery, a senior lecturer in paediatric immunology and bone marrow transplantation at Newcastle University, said: “Our message to other units treating younger CGD patients is, ‘What are you waiting for?’.”

Five out of the 20 patients transplanted by the Newcastle General Hospital team were from the North-East, including four-year-old Riley Mulvany, from Darlington, and 19- year-old Phillip Simpson, from Durham City.

Eighteen have gone on to make a full recovery. The two who died were young adults who had an advanced form of the disease.

Riley found a match after his family approached The Northern Echo to appeal for volunteer donors. Hundreds volunteered and were tested as potential volunteers, before a match was found.

Dr Gennery, who runs the Northern Unit for the Treatment of Children with Primary Immuno-deficiency, said: “We have done more of these transplants than anywhere else in the world and we think our results show that transplantation is a very successful way of curing many of our younger patients.”

Dr Gennery said the approach showed that patients – particularly children and young adults – should undergo transplantation as soon as possible, regardless of whether they have siblings.

“We have also shown that the success rate from unrelated donors is just as good as from sibling donors,” he said.

CGD severely damages the patient’s immune system, leading to regular hospitalisation and reducing life expectancy.

Medication to control the condition can also stunt growth.

“That is why it makes sense to manage the condition through transplantation,” said Dr Gennery.

Patients requiring bone marrow transplants spend weeks living in a virtual “bubble” of pressurised sterile air before and after surgery.