Haemophilia

Bleeding economics

David Hendrey

The shortage of money in the National Health Service is adversely affecting its staff and patients alike. One group of patients deserving to be considered as a special case for better treatment, claim certain doctors, are those with haemophilia.

"When as a result of years of research life-saving therapeutic materials suddenly become available to a population of patients, previously under-treated, there should surely be some means of assimilating this welcome advance. Otherwise it is stupid to undertake the research in the first place."

This somewhat pessimistic view comes from Dr Rosemary Biggs, who is director at the Oxford centre for treating haemophilia, but undoubtedly lies at the back of many other medical researchers' minds. Dr Biggs and her colleague Dr Charles Rizza have been leading a campaign, asking the Department of Health to provide more money for the treatment of haemophilia. The latest treatment is very expensive and an estimated £2,000 should, in Biggs's opinion, be spent each year on the treatment of each of the three thousand haemophiliacs in the United Kingdom. To achieve this the forty-two haemophilia treatment centres spread throughout the country will need at least £2 million added to their combined annual budgets.

The Department of Health is reluctant to provide this extra money from the National Health Service budget, which is heading towards crisis point under the joint strain from inflation and wage claims. Biggs and Rizza believe, however, that in the leng term under-treatment can be just as expensive.

Before the 1950s there was no treatment at all for haemophilia. The young boys diagnosed for this blood disorder, which prevents timely clotting of bleeding, were not expected to survive to adult life. Some did survive but were invariably crippled, because years of uncontrolled internal bleeds would lead to arthritis and deformed muscles.

Then came a break-through in medical research. A first treatment for haemophilia was developed and, although it was crude to begin with, hopes were raised that one day boys with haemophilia would be able to lead a normal life. To some extent subsequent refinements of the treatment have realised these hopes. The original treatment entailed transfusing blood plasma from a normal person until the bleedinb stopped, but one doctor has described wards where a severe bleed was being treated as having a "battlefield atmosphere", with blood donors lining the corridor outside. The transfusions were not only difficult and risky because of the large increase in body fluids, but also were somewhat ineffective.

A big step forward was when a bleed could be stopped by a single injection of a pharmaceutical pro duct which had to be stored in very cold refrigerators. This is the general practice nowadays for treating haemophilia; whenever a bleed occurs the haemophiliac must be taken to the nearest treatment centre, which may be several miles away. Although bleeding is stopped quickly at the centre, a great deal of Llamage is done to joints and muscles during the journey.

The latest pharmaceutical for haemophilia can be easily stored in a kitchen refrigerator, with the advantage that it can be injected immediately by the boy, his parent or the family doctor. But home treatment is very expensive. Without it however crippling is likely to occur and even at a relatively young age a haemophiliac may be too handicapped to take on a worthwhile job, depending on sodal security pay-outs and often (though certainly not always) placing an intolerable strain on other members of the family.

Although it depends very much on how the calculation is done, the cost to the state for social security benefits due to under-treatment may turn out to be as expensive as home treatment. "The financial argument," says Dr Biggs, "takes no account of the misery and anxiety attached to painful episodes of bleeding and the inability to hold a normal place in school and society."

Haemophilia is a rare disorder, inherited by only one boy in ten thousand. There are 3,000 haemophiliacs throughout the United Kingdom and consequently it is not surprising that very few people, including doctors, have any idea of the nature of the disorder.

The blood defect is inherited by sons from their mothers, who, although apparently normal, carry an abnormality in their chromosomes. A boy is therefore more likely to be a haemophiliac if there is a history of the disorder in his family. But contrary to widespread belief a diagnosis of haemophilia often comes as a shattering surprise to parents. Surveys have revealed that at least 30 per cent of cases are in families with no known prior history. One explanation for this could be that females predominate in the family and the abnormal chromosomes are passed on by several generations of 'carrier' females before it becomes apparent in a male child.

The thing which makes haemophiliacs so vulnerable to bleeds is that their blood lacks totally or partially a substance called Factor VIII. This is just one component of a complicated biochemical system for clotting blood whenever bleeding occurs inside or outside the body. The system is complicated because it must quickly stop loss of blood or damage being done to joints and mugicles, but should not be so effective that it causes blood clots within undamaged veins and arteries; these clots can kill.

The fundamental aim of treating a haemophiliac's bleed is to temporarily raise the amount of Factor VIII in his blood (in the early days by transfusions, but now by injections of purified Factor VIII) until clotting occurs. The treatment centres currently inject a crudely purified form of Factor VIII called "cryo-precipitate", but this is nowhere near as satisfactory as the latest available "freeze-dried concentrate" of Factor VIII. This is a white powder and, as already mentioned can be easily stored in a kitchen refrigerator. Apart from its high cost, this form of Factor VIII could do for haemophiliacs what insulin has for diabetics.

Why is Factor VIII so very expensive? The main reason is that whereas insulin can be cheaply produced from animal tissue from slaughterhouses, Factor VIII can be safely extracted only from human blood. Factor VIII was recently withdrawn from use, after it was discovered that repeated injections had adverse effects. The Department of Health has licensed four pharmaceutical companies to import and sell their brands of Factor VIII, but lack of money has so far prevented the haemophilia treatment centres from making use of this supply.

The NHS runs its own central blood fractionating unit, which supplies a small amount of Factor VIII to the treatment centres. The unit is reported to be desperately short of staff and facilities, so much so that it cannot meet the demand for Factor VIII.

Some while ago the British Medical Journal took a critical view of all this, commenting in an editorial: "For the first time in Britain we have come to rely on professional donors, not from Leeds, Liverpool or London, but Puerto Rico, Chile and Columbia" (where the foreign pharmaceutical companies buy their blood) . . . . "The introduction of human plasma fractions from foreign sources could in the next ten years prove to be a serious drain on the financial resources of the NHS. It is also certain that the cost to the taxpayer of producing similar products would be distinctly less if undertaken by the NHS." The drain on NHS financial resources has been prevented simply by adequate money not being made available to the haemophilia treatment centres for commercial Factor VIII. Dr Biggs Oxford centre has got around this by exceeding its budget from the local health authority, which understandably is not too pleased. The other main treatment centres at Manchester and Sheffield have approached their health authorities for more money, but have been less militant. One doctor at a treattnent centre confided that he lived in fear of being dismissed after contraverl; ing the rules by ordering a smah amount of commercial Factor VIII. So it seems that the haemophilil success story is in danger ° becoming a farce; the quiet revola" tion in medical science during the 1950s is turning into a bitter struggle, with the haemophilia specialists on one side and the Department of Health bureaucrats on the other. Dr Biggs explainsh°,W, her Oxford centre is coping in tI7 meantime: "Much time and effort 1,S, put into spreading the inadeclaa.t`, amounts of therapeutic mater Id thinly, so that deprivation sh°u t be least damaging. Essential, ba non-urgent, surgical operations

have been postponed, and are still being postponed." Surgery and dental treatment are particularly hazardous for, haemophiliacs.

Should haemophiliacs be considered as a special case? In their favour are a number of points, which can be summarised as (1) an effective and fully developed treatment is available now, (2) Factor VIII is found naturally in blood and therefore is safer than most drugs used for other disorders, (3) the high cost of Factor VIII should offset (at least in part) the present demands made by haemophiliacs On the health and social services, and (4) perhaps the most important point of all, haemophilia afflicts Young boys mainly and effective home treatment with Factor VIII should enable them to live normal and pain-free lives. But whether the extra money needed for home treatment will come from special grants from the Department of Health or from other sources remains to be seen.

David Hendrey is director of Janis, the specialist medicine and biology news agency.