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Compared to the period from 5 years ago, the situation of patients with hemophilia in Poland has improved significantly. However, this does not change the fact that most of them still have limited access to modern drugs, which are standardly used in other European countries, both in the treatment and prevention of this disease.
In small steps
A beneficial change was certainly the introduction in 2008 of a prophylactic program for the treatment of children and adolescents up to 18 years of age with low doses of clotting factors. – Thanks to this program, young people with hemophilia can live almost as much as their healthy peers. They avoid painful bleeding that destroy the joints and lead to disability – says Bogdan Gajewski, president of the Polish Association of People with Hemophilia.
This year, the Ministry of Health has taken another step forward by providing children with newly diagnosed hemophilia who have not been treated with plasma-based preparations to date with recombinant clotting factors. These are drugs with the best safety profile, manufactured with the use of biotechnology.
The problem is that – as doctors say – the group of children covered by this therapy is only a few. The rest must be content with plasma-derived factors, from which many countries have already withdrawn or will soon be withdrawn. Reason? Plasma drugs can cause infections. Years ago, it was through them that approximately 90 hemophiliacs worldwide were infected with HIV. Today, due to the different technology of their production, they do not carry the risk of HIV, HCV or HBV infection. However, infections with non-enveloped viruses, prions causing Creutzfeldt-Jacob disease or pathogens that have not yet been identified cannot be prevented. – That is why, as an association, we fight to ensure that all patients in Poland have the possibility of being treated with recombinant coagulation factors – says Bogdan Gajewski.
Let us follow the example of the neighbors
According to the data of the European Haemophilia Consortium, recombinant coagulation factors are the drugs of choice, incl. in Great Britain, Ireland, Canada and Denmark. In each of these countries, they account for 100 percent. drugs for the treatment of haemophilia, while plasma concentrates are reserved only for the treatment of von Willebrand disease. In the United States, recombinant drugs constitute 85%, in France and Italy 70-80%, in Germany – 60%. drugs that are tendered for sick people.
Someone will say that these countries are not the best example, because Poland is not as rich as they are. So let’s look at our southern neighbors, the Czechs. Since 2006, newly diagnosed, previously untreated or minimally treated children have been treated with recombinant drugs of the 21nd and 10rd generation prophylactically. In Hungary, recombinant second- or third-generation factors are prophylactically administered to children and adolescents up to the age of XNUMX and patients with a clotting factor inhibitor, one of the most common complications of hemophilia. Even in Lithuania, which is a much poorer country than Poland, a much larger group of patients has access to recombinant drugs. They are given to all children with hemophilia up to the age of XNUMX, and also to older ones, if they have medical indications for their use (e.g. when their immune system reacts badly to drugs made from blood plasma).
Sentenced to a disability pension
Adult patients in Poland, who have been treated for many years contrary to European standards, are in the worst situation. For many years, the coagulation factor VIII consumption per capita in Poland was only 1,3 units, while the World Health Organization recognized 2 units as the minimum necessary to save lives. In recent years, the Polish index has increased to 2,97 units. However, it is still several times lower than in other European Union countries (e.g. in Hungary it is 7,2 units).
Due to the lack of prophylaxis and poor treatment, consisting in the use of insufficient amounts of coagulation factors after strokes, many patients had to quit their jobs and go on a disability pension. The disease led to many complications for them. – The most common is the so-called hemophilic arthropathy, i.e. progressive damage to the joints of the upper and lower extremities – says Dr. Grzegorz Dobaczewski from the Department of Bone Marrow Transplantation, Hematology and Pediatric Oncology, Medical University of Wroclaw.
Haemophilic arthropathy is manifested by joint stiffness, limited mobility, and severe pain when walking. Patients suffering from this disease have great problems with moving, climbing stairs, boarding a bus or tram. At an advanced stage, they require orthopedic surgery, for which they have to wait in a queue of several years. They also need long-term, intensive rehabilitation, which is even more difficult to access. The Hemostasis Group of the Polish Society of Hematologists and Transfusionists is fighting to establish a network of specialist centers in Poland that provide patients with hemophilia with comprehensive care – diagnostics, therapy and rehabilitation. In Western Europe and the United States, such centers have been operating for a long time.
Instead of desensitization – pain and disappointment
Many patients develop the so-called a clotting factor inhibitor. Their immune system, as a result of an abnormal reaction, makes antibodies against the coagulation factor they are taking. The patient’s organism treats him as an enemy that must be destroyed and destroys him. The medicine therefore stops working and the bleeding cannot be stopped.
The most effective method of treating this complication is desensitization, i.e. inducing immunotolerance to the administered clotting factor. All children in Poland have had access to desensitization since last year. Unfortunately, the remaining patients not only are not desensitized, but also do not have full access to less effective therapy with drugs that bypass inhibitors, which should be used in the event of strokes. And each subsequent stroke means pain and further damage to the joints for the patient.
What You Need to Know About Hemophilia
Hemophilia is a hemorrhagic diathesis, i.e. a disorder of blood clotting caused by the lack or deficiency of one of the blood clotting factors. It is a very rare disease, it affects one in 10 people. people. In Poland, about 3 thousand people suffer from it. patients. Most of them are people with haemophilia A, which is not enough factor VIII in the blood. Haemophilia B, which is deficient in factor IX, is less common. – The cause of this disease lies in genetics. It is carried by women, and men are ill – explains Dr. Grzegorz Dobaczewski.
The vast majority of patients inherited hemophilia from their parents. In those who did not inherit it (it is estimated that they constitute about 30% of all patients), the cause of hemophilia is a mutation of their own blood coagulation factor gene.
Text: Mariola Marklowska-Dzierżak
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