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01:19 GMT, Monday, 1 December 2008

Transplant first for gut failure

By Helen Briggs
Health reporter, BBC News

Brothers born with a rare disease that destroys the gut have had their lives transformed by a pioneering operation.

Michael Wenman, 12, became the world's first child to have a bone marrow transplant for gut failure, followed later by his younger brother Matthew.

Before the operation the boys spent much of their time in hospital but they can now lead normal lives.

Great Ormond Street Hospital says more transplants will take place next year following a successful pilot study.

Gut failure affects the body's digestive system, leaving it unable to absorb vital nutrients.

Children with the most severe form of the condition have to be fed intravenously to keep them alive.

"It's a new way of treating something that was untreatable before"
Dr Neil Shah

Before his transplant at the age of six, Michael, lived in constant pain, and had rashes all over his body.

His mother Lisa says he has had a 100% improvement in his quality of life since the surgery, and is now "a different child".

"He can now play football, he can go swimming, he goes to full-time school," she told the BBC.

"He used to sleep in the afternoon; now he'd stay up all night if I let him."

New approach

Lisa Wenman, from Cranleigh in Surrey, said she first realised there was something wrong with Michael when he was three weeks old. After numerous tests, he was eventually diagnosed with gut failure, at the age of one.

Gut failure



He was continuously unwell, and at one point was so ill he was given just 15 minutes to live.

Doctors at Great Ormond Street eventually suggested a bone marrow transplant operation, even though it had never been tried before for gut failure.

They hoped that by replacing Michael's defective immune system, they might be able to stop the inflammation that was destroying his gut.

Dr Neil Shah, consultant paediatric gastroenterologist at Great Ormond Street Hospital, said the decision was "incredibly difficult", but all other treatment options had failed.

"It's a new way of treating something that was untreatable before," he said.

Great Ormond Street has now carried out 11 such operations, with success in 10 cases.

'Normal lives'

Among them was Michael's brother Matthew, who also has the condition.

"When I had Matthew, he seemed absolutely perfect," said Lisa Wenman.

"We came home and on the fifth day I started to get a bit concerned - he didn't seem to want his feed."

 (Image: GOSH)

Matthew, now nine, was eventually diagnosed with gut failure, and had a bone marrow transplant when he was four.

"The improvement for Matthew has been just as dramatic as it was for Michael," the boys' mother explained.

"I have another son who is 14 and I really feel as though I have three strong healthy boys in my family now."

The boys have gone from being in and out of hospital almost every week, to visiting doctors for a follow-up only once a year.

"Michael and Matthew are now leading completely normal lives," said Dr Shah.

"They're doing what kids should do."

Following the pilot study, Great Ormond Street Hospital has secured funding from the NHS to become a recognised centre for carrying out bone marrow transplants for gut failure, starting next April.

Newcastle General Hospital will run a similar service, and together the hospitals hope to treat about 10 patients a year.

Dr Paul Travers, senior scientist at the MRC centre for regenerative medicine at Edinburgh University, said the work was a "useful advance" that might hold promise for treating other immune conditions.

"The use of stem cell transplants to reset the immune system is one that offers a lot of promise in a number of conditions," he said. "To see it applied in this case is very exciting."




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Related to this story:
New hope for bone marrow patients (15 Jul 08 |  Edinburgh, East and Fife )
Bone marrow 'cures HIV patient' (13 Nov 08 |  Health )

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