Friday, February 12, 1999 Published at 12:26 GMT
Transplant hope for arthritic children
Transplants could help children with severe arthritis
Bone marrow transplants could help hundreds of children who suffer from a potentially life-threatening form of arthritis.
Dutch researchers have found that children with systemic juvenile chronic arthritis (JCA) - a severe form of the disease which affects 10% of sufferers - could benefit from receiving a transplant.
Around 100 children with the disease are so badly affected that it is life-threatening because of its effect on the heart.
JCA occurs when a person's own immune cells attack the capsule that surrounds the joints, causing painful inflammation and, in severe cases, destruction of the joint cartilage and underlying bone.
Scientists do not know why it happens, but they suspect it could be linked to immune deficiencies.
Traditional anti-inflammatory and immunosuppressive drugs used to treat JCA sometimes do not work.
Dutch researchers used autologous haemopoletic stem-cell transplantation (AHSCT) on four children with systemic JCA.
Their aim was to kill the white blood cells that cause inflammation, basically wiping out the immune system and replacing it with one that works.
The results, followed up for a period of up to 18 months, are "very promising".
One patient was a six-year-old girl who had had chronic arthritis since she was one. She had severe pain in her joints, elbows, knees, wrists and ankles.
She was first treated with aspirin and immunosuppressive drugs. However, she only had a partial response to treatment.
As a result, she only grew two centimetres between the age of two and five.
She also suffered side effects such as hirsutism and a mild cataract.
X-rays showed she had bone erosion, osteoporosis and severe joint problems.
Although she contracted a mild virus and swelling around four months after having AHSCT, this soon cleared up.
During the 18 months of follow-up, she grew 10 centimetres.
All the four patients showed a marked reduction in joint swelling, pain and morning stiffness after having AHSCT.
The researchers from Utrecht say it is too early yet to know what the long-term effects of AHSCT are.
Writing in The Lancet magazine, they say: "Short-term results in our four cases are very promising, but follow-up at present is too short to permit firm conclusions to be drawn on the long-term effects."
AHSCT has been used to treat other immune disorders in adults, but long-term results have shown some relapses.
European doctors have drawn up guidelines on when the treatment should be used on children.
It will be published in the spring in the British Journal of Rheumatology.
The technique could be tried out in the UK soon. Hospitals in Birmingham, Newcastle and London are considering performing the operation.
But it is very expensive. It costs between £25,000 and £100,000 per patient.
Doctors say there are only between 10 to 12 patients a year who would qualify for the operation.
And cases would have to be selected very carefully. Ideally only children who have not responded to other treatment, who are fit and who have not yet been severely disabled by JCA would be suitable.
But it is difficult to identify children who will become severely disabled from the disease.
Professor Tauny Southwood of Birmingham Children's Hospital said the research results were "excellent".
But he added that the risks of dying from systemic JCA would have to be balanced against the risks of undergoing AHSCT.
Around 3% of patients are at risk of dying from AHSCT, mainly because of its suppression of the immune system.
Professor Pat Woo of Great Ormond Street Hospital also urged caution.
She said: "AHSCT is potentially exciting, but we have to be very cautious as the children could relapse."