Money, or their lives...

Haemophiliac children are being denied treatment with an artificial, virus-free clotting agent. The problem? It costs more than the alternative - made from human blood. Dina Rabinovitch reports
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Indy Lifestyle Online
Last week, six-year-old Noam Roth had his grommets "done" - practically a childhood rite of passage these days, involving the insertion of tiny cotton-reel like objects into the ear to clear up hearing problems. It is an occurrence so minor and mundane, doctors don't describe it to parents as an operation, but merely a "procedure."

For Noam, there is no such thing as minor surgery, however, because he is haemophiliac. When he bleeds his blood does not clot and without medical intervention he can bleed to death. So before the operation the doctors boosted his blood by twice as much as they would ordinarily, and gave him another blood booster straight afterwards. Noam is a blond-haired boy with an old-fashioned cherub's face, who nevertheless tears round his school playground like a demon on speed. He is - and I have watched him in that playground for several years now - quite fearless, a tribute to his parents and carers who don't want him growing up hampered more than he has to be by his incurable condition.

But after Noam's grommets procedure, the consultant haematologist came over to talk to Sue Roth, Noam's mother, and what she said shook Mrs Roth from her resolutely calm approach to Noam's condition. It was straight after the operation, and time for Noam's blood to be boosted for the second time that day. Noam's blood lacks VIII, the chemical link that enables blood to clot. The factor VIII and syringe for injecting were standing ready as the consultant, Dr Christine Lee, began to talk. She told Noam's mother that she was not easy about the factor VIII about to be injected into Noam's veins.

There are two kinds of factor VIII - one type made from human blood, and the other a genetically produced artificial equivalent, called recombinant factor VIII. Because of the risk of infection from a human blood product, doctors prefer to treat haemophiliacs with the purer, artificial product - the UK Haemophiliac Centre Directors' Organisation is drawing up guidelines this week stating, according to a spokesman, that "we'd like to use recombinant for everybody".

The factor VIII about to be syringed into Noam Roth after his surgery last week, however, was plasma-produced - the human product. What concerned Dr Lee was that in the same hospital where Noam is treated, the Royal Free in London - second largest centre for treatment of haemophiliacs in the UK, and a recognised centre of excellence - some children were getting recombinant, while others were still on the human product. And the reason? Cost. Recombinant is about 52p a unit, while plasma-produced factor VIII sells from 20p-28p a unit. Since the restructuring of the NHS, the Royal Free has to get authorisation from individual purchasers to pay for recombinant. Though Dr Lee has been canvassing for two years for all patients to be on recombinant, she understood from her management that only boys at the the Royal Free whose health authorities had agreed to pay the larger cost were being treated with the artificial product.

Recombinant was licensed for use in this country in August 1994. It was developed in the wake of the 1980s panic when it became clear that the haemophiliac population was falling prey to HIV and hepatitis viruses from the very blood donations that were their gift of life. Although all blood now used in transfusions is heat-treated, the procedure is not enough to kill all viruses in the blood. Every six months Noam and all the children like him, are tested to check they haven't picked up a virus from their factor VIII donations. When the results come up clear, doctors and parents sigh with relief; the child is "virally negative".

In November 1994, HM Customs and Excise slapped 17.5 per cent VAT on recombinant. Blood and drugs are usually exempt from VAT, but Customs decided recombinant factor VIII didn't count as blood. The UK lags behind the rest of the Western world in using recombinant - despite the fact that recombinant is cheaper here than in Europe. In Germany, 50 per cent of factor VIII is recombinant, while in France it is 40 per cent - in the UK it was 4 per cent last year, possibly up to 10 per cent this year.

The Royal Free is not the only hospital where doctors are facing the dilemma of some children getting recombinant while others don't. It is happening right across the country. "This is a big problem across the UK," Dr Paul Giangrande, director of Oxford's Haemophilia Centre explained to me. "Now we've got a genetically engineered product, I believe strongly we should use it."

Oxford Haemophilia Centre is the largest in the country, just ahead of the Royal Free. Oxford has used recombinant on all boys under 10 - except one - since April 1995. One of Dr Giangrande's health authorities, Swindon, has balked at the cost. He has written to them saying, "we accept your decision but it's against my medical advice". He is now considering new ways of getting them to change their mind. In the meantime, the boy from Swindon being treated at Oxford is the only under-10 year-old there not on recombinant.

Dr Lee showed the Roths a letter she had written to the chief executive of the Royal Free in which she expressed her "discomfort" at having to give some children plasma-derived products. "Day after day we are signing prescriptions for plasma-derived concentrate which we know has the potential to transmit life-threatening viruses to our children."

She also revealed that "[after] the finding of hepatitis C genomic material in a clotting factor concentrate that is used to treat our patients, we have identified 16 individuals who have received the contaminated batches". One of them is a child who will now have to be under "active viral surveillance ... this comes fast on the heels of the episode in which three children in Manchester have been infected with hepatitis A, as a result of treatment with plasma-derived factor VIII."

Dr Lee said: "it's very difficult to explain the shadow HIV and hepatitis cast over all of us working with haemophiliacs, but it is just so difficult because it is a disease we have given to patients in our care. And the fact is that there are still two problem viruses that break through all sterilisation methods of human products - hepatitis A and parvo-virus infection."

The Roths were stunned at the news that was given them last week. They have known about recombinant factor VIII for some time, but trusting in the hospital, they simply assumed it would be given to Noam as soon as possible. To find a two-tier system operating around their boy's health added unnecessary suffering to a family who are dealing with enough already.

In their kitchen refrigerator the Roths have a largish tupperware box. It is filled with injection paraphernalia and small jars of factor VIII, the human product. When Noam bruises himself, which happens every six to eight weeks, to stop him from bleeding to death, he must be injected. Normally, his mother or rather will take him to the Royal Free to have the injection, but in an emergency they can do it at home. Because of the years of his illness, he is pale and it is hard to find veins in his skin. There is also a problem with using the same vein too often, since the vein itself can become bruised from the injection.

Noam hates the injections. He becomes tense as soon as he sees the bottles and needles. He doesn't understand why his mother - his father cannot bear to give the injections at all - will sometimes do something to him which he can't abide; why his parents insist on him having the repeated treatments "This," said Sue Roth, "is what we do as parents. We do this hated thing to him, because he needs it." What she could not tolerate was the thought that the hated thing could be actively harming her child, when it need not - when there is a better alternative.

The end of the story is good news. For, interestingly, within a day of the Roths beginning to lobby the Royal Free, within a day of the hospital's press office being informed that a journalist was going to write a story about Noam Roth, his parents were told permission had been given for Noam to receive recombinant factor VIII. Within a few more days, the news became even better. The hospital's press officer called me to say that all children at the Royal Free would now be receiving recombinant. A statement from Martin Else, the chief executive, had authorised her to say that health authorities had been told it was the preferred method of treatment and "we'd be looking to them for support". The hospital is also applying for special funding to pay for the extra cost. It seemed odd that it had happened so quickly - was there a connection between the press interest and the marvellous news? I asked her. "That strikes me as extremely unlikely," she replied.

Later that night I spoke to a delighted Dr Lee. "I have to tell you," she said, "that I have great respect for your newspaper. I am so happy to know that all our patients will now have recombinant and that there is a generation of children with haemophilia who won't be cursed by viral infection."