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April 17 is World Hemophilia Day
Polish patients with hemophilia can envy Hungarian, Czech and Lithuanian patients. There, everyone is given prophylactic drugs to prevent strokes, in Poland only children.
Haemophilia is a genetic disease where one of the clotting factors is deficient or absent in the blood. Minor injuries and cuts are not dangerous for the sick, but the more serious ones can even be life-threatening. Internal bleeding, mainly into joints and muscles, is also dangerous. – You cannot see them, but they are very painful. The pain is then like putting a hand between a closed door – says one of the patients.
Repeated strokes irreversibly damage the joints. Therefore, patients who have been improperly treated for many years and have not received enough clotting factors today have enormous mobility problems: walking, boarding a bus, climbing stairs. Most of them must be on a pension.
Prophylaxis in the treatment of haemophilia
A preventive treatment program has been operating in Poland since 2008. Children and adolescents up to the age of 18 receive a clotting factor injection 2-3 times a week, which helps to avoid most strokes. – In the past, each stroke would disable my son from normal functioning for a few days. Today, thanks to preventive treatment, Janek can function normally, he goes to school and goes on vacation. And, most importantly, in the future we will not walk on crutches like older patients, says Agnieszka Cybulska, mother of an 8-year-old boy suffering from hemophilia.
A young person who turns 18 is no longer “entitled” to preventive treatment. He only gets medication when he has a stroke. Unfortunately, each of them causes joint damage. – Patients who turned 18 are admitted to my clinic and because of the fact that they could not continue prophylaxis, they had a stroke. Implanting endoprostheses into damaged joints is as expensive as prophylactic treatment – says Prof. Jerzy Windyga from the Institute of Hematology and Transfusion Medicine in Warsaw.
– Prophylactic treatment should also be continued after the age of 18, especially in people who have frequent bleeding. This is the case in other countries, such as Lithuania, the Czech Republic, Bulgaria, and Hungary. Thanks to this, young people can finish their studies, get a job – adds Bogdan Gajewski, president of the Polish Association of Patients with Hemophilia (PSCH). – Prophylactic treatment protects them against joint damage and disability. It is also profitable for the state, which will not have to pay disability pensions in the future, as such people will be able to work normally.
Safe recombinant drugs
The biggest problem for parents is that children – apart from a very small group of newly diagnosed ones this year – receive drugs derived from blood plasma. Meanwhile, small patients who have not received such preparations before – receive recombinant clotting factors produced by genetic engineering. These are drugs with the highest safety profile. Recombinant drugs are used in Lithuania, the Czech Republic, Bulgaria, Hungary and many other countries with a similar economic situation as in Poland. Not only for the youngest children, but also for patients who were previously treated with plasma-derived drugs. – I am glad that Jasiek gets the factor, but at the same time I have concerns, because I know that it is a drug derived from plasma collected from many thousands of people. Are they really all healthy? My dad got hepatitis C this way. I know drugs no longer transmit hepatitis C. However, just as the virus that caused the disease was not known in the past, so now we do not know many other infectious agents that can transmit other diseases. In most countries, even in our neighbors – the Czech Republic, Lithuania, Hungary – children receive the safest, recombinant clotting factors. It is outrageous and it hurts why children from Poland are deprived of the possibility of using the safest drugs – adds Agnieszka Cybulska.
Prof. Jerzy Windyga emphasizes that drugs made from plasma are safe and tested for all known infectious particles. However, the professor believes that the future belongs to recombinant preparations, because they will be cheaper in the future.
Clotting factor regulations
In Poland, there is also a lack of clear guidelines on what medications a parent may give to a child. According to the assumptions of prophylactic treatment, these should be the clotting factors that they get home. If, however, the child has bleeding, according to the regulations, the parent cannot give the agent he has at home, but should take the child to the doctor. The doctor, in turn, must write an order for the delivery of the drug to the regional blood donation station.
Difficult situations may arise for parents due to unclear regulations. In February, at the Gdańsk regional blood donation station (RCKiK), despite an order issued by the attending physician, a clotting factor was refused for an 8-year-old boy who had a painful ankle haemorrhage. The doctor from the Gdańsk RCKiK explained her refusal by the fact that she had small amounts of the agent at her disposal, intended only for saving lives and for elective surgical procedures. The parents were in despair in this situation: the stroke exposed the boy not only to pain, but also to the risk of damaging the joint. – The preventive program is financed by the National Health Fund, and the treatment of bleeding is financed by the Ministry of Health. Therefore, in the event of bleeding, parents are forbidden to give the factors that they have at home. But what are they to do in such a situation: expose the child to pain and go to the hospital, where they may be refused to administer the factor? – asks Bogdan Gajewski, who receives signals about irregularities. – Parents are not required to know who is funding the program. They just want their child to be treated well.
The Gdańsk branch of PSCH, to which the boy’s parents contacted, wrote a letter to the Ministry of Health on this matter. So far, however, the parents have not received an answer.
Another problem is the evaluation of the treatment of patients with hemophilia by the National Health Fund. Prof. Windyga informs that the Fund pays PLN 10 for a 561-day stay and treatment at his clinic, while the actual cost of treatment is PLN 4861. – That is why many institutions send patients to the Institute, because it is not profitable for them to treat them – says prof. Elevator.
Text: Halina Pilonis